Company Directory

Available Therapies via Single-Patient EA

The mission of Achillion is to provide better treatments for people with serious diseases. We are a science-driven company committed to putting patients first. In our endeavor to bring innovative medicines to patients as fast and safely as possible, we conduct clinical trials to establish the safety and efficacy of an ‘investigational drug’. An ‘investigational drug’ is a potential medicine that is in active clinical development, but has not yet received marketing approval by regulatory authorities. Clinical trials and the subsequent timely generation of safety and efficacy data are the most effective way of ensuring review and decision making by Regulatory Authorities. This ultimately has resulted in access to new, safe and effective approved medicines for patients.

Achillion supports the need for Early Access to investigational drugs for patients ineligible to enroll in clinical trials.

An Early Access program for a specific investigational drug may be opened if Achillion determines that all the following criteria are met:
1. The investigational drug is the subject of an active clinical development program and is not approved in any indication in the country concerned
2. An adequate supply of the investigational drug exists to perform necessary clinical studies as well as to provide Early Access to patients who do not have alternative treatment options
3. Early Access does not impede or compromise the clinical development or regulatory approval of the drug under investigation
4. There are sufficient clinical data available with respect to both the investigational drug and the disease condition for which the application is being sought, to anticipate that any potential benefits from treatment are likely to outweigh any potential risks to the patient

Achillion continues to assess the eligibility requirements and criteria for Early Access to the investigational drug danicopan (ACH-4471). At this time, an Early Access Program (EAP) is not available. We will re-evaluate this policy from time to time.

Disease/Category-Specific EA Policies/Criteria

AiCuris develops Pritelivir oral tablets for the treatment of acyclovir-resistant mucocutaneous herpes simplex virus (HSV) infections. A phase 2 trial in immunocompromised patients is ongoing in the US. AiCuris supports expanded access requests for Pritelivir oral tablets for the treatment of eligible patients and collaborates with myTomorrows to facilitate early access to Pritelivir for acyclovir-resistant mucocutaneous HSV  infections in immunocompromised patients.

To get access, patients need to consult with their treating physician to explore all treatment options. If the physician agrees to a treatment with Pritelivir oral tablets and to oversee the patient's treatment, the physician should  contact the myTomorrows medical team for more information on this EAP. When providing medical information about the patient, please note that only anonymized data shall be included in any patient information. Due to EU data protection law this shall not be accompanied by personal data.

Single-Patient EA Policies/Criteria

Akeso Biopharma, Inc. (Akeso) is a biopharmaceutical company committed to bringing new, innovative therapies to patients by conducting rigorous clinical trials and obtaining marketing approval by the U.S. Food and Drug Administration (FDA), China National Medical Products Administration (NMPA) and other regulatory authorities. Akeso conducts clinical trials to study investigational medicines in patients in which the studies are designed to obtain data on safety and efficacy that may be used to support approval of the product and subsequent wider accessibility to patients. Investigational medicines are drugs or biologics that have not been approved or cleared by regulatory authorities.

Akeso seeks to retain the ability to manufacture and supply investigational medicines in a fair and equitable manner and in a volume that assures adequate supply for ongoing clinical trials and development programs. Hence, at this time, Akeso is unable to provide its investigational medicines on an expanded access or right to try basis. For patients seeking access to our investigational medicines before they are approved by a regulatory authority, participation in one of Akeso’s clinical trials is the most appropriate way to access these investigational medicines. To learn more about available clinical trials by Akeso, please visit https://www.akesobio.com, or visit https://clinicaltrials.gov and search by company, disease or medicine.

If you have additional questions, please speak with your physician or contact Akeso at clinicaltrials@akesobio.com.

Consistent with the 21st Century Cures Act, Akeso may revise this policy at any time.

Disease/Category-Specific EA Policies/Criteria

Emergency Treatment for COVID-19 https://alexion.com/our-commitment/global-access-to-medicines

Alexion has provided SOLIRIS as an experimental emergency treatment for a small number of patients with COVID-19 infection and severe pneumonia. Requests are assessed on a case-by-case basis, with priority given to those with clinical trial experience and the oversight of a physician who has extensive experience using SOLIRIS.

For more information, contact us at covid.requests@alexion.com.

Eculizumab (Soliris) in Covid-19 Infected Patients  (Expanded Access Study)

ULTOMIRIS^® (ravulizumab-cwvz) - the first and only long-acting C5 complement inhibitor

Single-Patient EA Policies/Criteria

http://www.alkermes.com/assets/content/files/Expanded%20Access%20Policy.pdf

Single-Patient EA Policies/Criteria

AlloVir is committed to developing safe and effective therapies addressing severe and potentially life-threatening virus-associated diseases and their potential sequelae. Our goal is to provide access to our investigational therapies at the appropriate time and in the correct manner for patients.
Expanded access refers to the use of an investigational therapy outside a clinical trial when the primary purpose is to diagnose, prevent, or treat a serious condition in a patient. This is different from a clinical trial, where more comprehensive safety and efficacy data are collected. We understand the need for expanded access programs, and we recognize the importance of having an appropriate policy.
A number of factors consistent with the US Food and Drug Administration (US FDA) and other regulatory agencies’ guidelines should be taken into account when considering expanded access. They include:
•    The illness must be serious or immediately life-threatening with no other satisfactory treatment options (such as approved products or enrolling clinical trials).
•    The patient must have undergone appropriate standard treatments without success and no comparable or satisfactory alternative treatment is available or exists to treat the disease or condition.
•    The patient is ineligible for participation in any ongoing clinical study of the investigational product, which includes lack of access due to geographic limitations.
•    The patient has a disease for which there is sufficient evidence of a projected benefit from the use of the investigational product and the benefit outweighs the known or anticipated risks.
•    There is an ability to provide the product in a fair and equitable manner and there is adequate manufacturing capacity for ongoing programs.
•    Whether granting expanded access would potentially compromise the scientific validity of broader development programs or interfere with or delay current clinical trials or regulatory filings designed to make the therapy available to many more patients.

At this time, AlloVir believes that participation in one of our clinical trials is the most appropriate way to access our investigational therapies. If you have additional questions, please speak with your physician or contact expandedaccess@allovir.com. We anticipate acknowledging receipt of requests sent to this email within five business days.

Disease/Category-Specific EA Policies/Criteria

http://www.alnylam.com/medical-professionals/expanded-access-program/

Single-Patient EA Policies/Criteria

Alpine will evaluate an expanded access request based on a variety of factors, which include but are not limited to:
• The request for expanded access is submitted by a treating physician, who is appropriately licensed;
• The patient’s condition is serious or life-threatening and there is a reasonable potential that the investigational drug has the potential to provide a benefit to the patient with an acceptable level of risk;
• Participation in a clinical trial is not an option, either because the patient is not eligible to enroll or because there are no appropriate ongoing clinical trials;
• The proposed dose of the investigational drug is within the existing dose range for which human safety data are available;
• The investigational product is available for treatment use without compromising supplies that have been designated for other uses.

Requests will be considered on a case-by-case basis. If a request for expanded access is granted, the requesting physician must agree to obtain appropriate regulatory and ethics committee approvals and comply with regulatory obligations, including obtaining patient consent, patient monitoring, and safety reporting.

Available Therapies via Single-Patient EA

ALPN-101

Single-Patient EA Policies/Criteria

At ALX Oncology, our focus is on the discovery and development of novel anti-cancer compounds that block the CD47 - SIRPα pathway. The goal of our current clinical study program is to enroll patients and obtain clinical data on ALX148 that is required by the US Food and Drug Administration (FDA) and other regulatory authorities for review and approval of marketing applications for this investigational agent. We believe that focusing on clinical studies is the most appropriate way to achieve this goal; therefore, we do not currently offer access to ALX148 outside of clinical studies and, at this time, we have no Expanded Access program (sometimes referred to as “compassionate use” or “pre-approval access”).

For information on participating in and status of ALX148 clinical trials, please see: https://clinicaltrials.gov/ct2/home

If you have additional questions regarding this policy, please speak with your health care provider or contact: info@alxoncology.com

Single-Patient EA Policies/Criteria

Requests for Early Access cannot be made directly by an individual patient or a patient’s parent/legal guardian or caregiver. Patients and caregivers seeking general information may reach out to Amicus’ Global Patient & Professional Advocacy Department at patientadvocacy@amicusrx.com or call toll-free in the United States at 1-866-9AMICUS (1-866-926-4287), or +44-1753-888-567 for International inquiries.

Single-Patient EA Policies/Criteria

The Company has not implemented an expanded access program (EAP) in the US for use of AP101 in epidermolysis bullosa (EB). There is an ongoing Phase 3 randomised controlled study evaluating the safety and efficacy of AP101 for the treatment of patients with junctional and dystrophic EB. While the study is ongoing and equipoise remains with respect to the benefits and risks for AP101 in this patient population, the Company’s policy is to encourage all potentially eligible patients in the US to be considered for enrolment in the clinical trial

Single-Patient EA Policies/Criteria

At this time, Angiocrine’s investigational therapies are not available on an expanded access or right-to-try basis for new patients. In the event Angiocrine decides to consider expanded access or right-to-try use, the Company will update its policy at that time and then evaluate and respond to each request that it receives on a case-by-case basis. For more information on our investigational therapies and ongoing clinical trials, please visit clinicaltrials.gov.

Single-Patient EA Policies/Criteria

Aravive, Inc. is a clinical-stage biopharmaceutical company developing treatments designed to halt the progression of life-threatening diseases, including cancer. The following is Aravive’s policy for evaluating and responding to requests for individual patient access to its investigational drugs that are intended to treat serious diseases.

Aravive believes that investigational drugs should be studied in patients as part of clinical trials designed to obtain data on safety and efficacy that may be used to support approval of the product and subsequent wider accessibility to patients. We encourage patients to speak with their physicians and to participate in clinical trials. In rare cases when patients with serious diseases are unable to participate in clinical trials and have exhausted all available therapies, Aravive may consider providing an investigational drug outside of a clinical trial.

General Criteria

We consider a number of factors when determining whether or not to support expanded access for a given investigational drug:

Will the potential benefit potentially outweigh the collective potential risks to the patient?
Is there adequate drug supply for both the clinical development program (current and future trials) and broad expanded access?
Is there enough evidence to reasonably assess that the drug will be safe and effective so that it can be provided to patients under expanded access in an environment that is less controlled than a sponsored clinical trial?
To meet regulatory requirements, will the safety data be adequately provided to Aravive by a local physician outside of a sponsored clinical trial?
Is there a good understanding of the indication for which use is requested?
Will it jeopardize the ongoing development work that Aravive is conducting to bring a therapeutic to market as quickly as possible and to as many patients as possible?
In addition, the program must be compliant with local rules and laws and the treating physician has to be willing to open a single-patient (Investigator) IND with the FDA.

Contact Information

A treating physician may submit questions or requests regarding expanded access to the following: clinicaltrials@aravive.com

Additional information may be obtained from the U.S. Food and Drug Administration at https://www.fda.gov/downloads/newsevents/publichealthfocus/expandedaccesscompassionateuse/ucm504494…

Request Procedures

Requests should be submitted to clinicaltrials@aravive.com by the treating licensed physician and should include sufficient supporting detail to enable Aravive to evaluate the expanded access request. Please include contact information so Aravive can follow-up with the physician directly (i.e., address, phone number, e-mail). The requesting physician would be expected to submit an Investigator IND, if appropriate, only after Aravive review of the provided information and approval of the request.

Aravive may revise this expanded access policy at any time. Additionally, the posting of the policy by Aravive shall not serve as a guarantee of access to any specific investigational drug for any patient.

Available Therapies via Single-Patient EA

AVB-500 is a therapeutic recombinant fusion protein that has been shown to neutralize GAS6 activity by binding to GAS6 with very high affinity. In doing so, AVB-500 selectively inhibits the GAS6-AXL signaling pathway. Aravive reported positive data from the first 31 patients enrolled in the Phase 1b portion of a Phase 1b/2 clinical trial of AVB-500 in platinum-resistant recurrent ovarian cancer. AVB-500 continues to be well tolerated. Investigator-sponsored Phase 1/2 trials of AVB-500, in combination with durvalumab in patients with platinum-resistant recurrent epithelial ovarian cancer and with avelumab in patients with advanced urothelial Carcinoma (COAXIN), are also ongoing.

Disease/Category-Specific EA Policies/Criteria

https://aravive.com/patients/

Single-Patient EA Policies/Criteria

At this time, Arcellx's investigational therapies can only be accessed through participation in a clinical trial. Additional details regarding Arcellx's active clinical trials can be found at www.clinicaltrials.gov.

Available Therapies via Single-Patient EA

Currently, we have no therapies available via single-patient EA.

Disease/Category-Specific EA Policies/Criteria

http://arcellx.com/#pipeline

Single-Patient EA Policies/Criteria

Arrowhead requires the following criteria be met for a request to be considered:
• The investigational medicine must be part of an active clinical development program,
• Access to the medicine will not compromise clinical trials or the regulatory pathway,
• There is substantial scientific evidence to support the benefit risk profile of the investigational product for its intended use,
• Sufficient supply of the investigational medicine is available, and
• It is logistically possible to safely administer the investigational medicine outside of a clinical trial setting
The patient and the treating physician must also meet the following criteria to be considered by Arrowhead for access to an investigational medicine:
• He/she has a serious or immediately life-threatening disease
• He/she lacks other currently available therapeutic options
• He/she is unable to join an active clinical trial of the investigational product
• His/her treating physician believes there is potential for the patient under consideration to reasonably expect benefit from the treatment

Available Therapies via Single-Patient EA

ARO-AAT Injection
For the treatment of alpha-1 antitrypsin deficiency associated liver disease.

Disease/Category-Specific EA Policies/Criteria

https://arrowheadpharma.com/wp-content/uploads/2019/03/Compassionate-Use-Expanded-Access.pdf

Single-Patient EA Policies/Criteria

At this time, we do not have an expanded access program that allows patients to have access to our investigational products prior to FDA approval.

Single-Patient EA Policies/Criteria

Atossa Genetics is a clinical-stage biopharmaceutical company developing novel, proprietary therapeutics and delivery methods for breast cancer and other breast conditions. 
Atossa Genetics’ development resources are focused on conducting clinical studies to fully answer important scientific questions about the potential risks and benefits of the investigational products, and to obtain marketing approval by the FDA and other regulatory health authorities. 
Atossa Genetics is committed to making investigational products available to seriously ill patients who have exhausted other treatment options. A treating physician, who is able to comply with the requirements that are stated in this document, may request information about how to apply for access to Atossa Genetics’ investigational products by contacting the Company. 
The purpose of this policy is to describe the requirements for Expanded Access to Atossa Genetics investigational products to patients outside of a clinical study. 
ATOSSA GENETICS’ APPROACH TO REQUESTS FOR ACCESS TO EXPERIMENTAL THERAPIES
•    Participation in clinical trials is the first and most preferable route.
•    If participation in clinical trials is not an option, physicians may consider other options, which may include expanded access programs managed by the company or single-patient expanded access.
•    Requests are considered on a case-by-case basis in a fair and equitable manner.
GENERAL CRITERIA USED TO EVALUATE ACCESS REQUESTS FOR INDIVIDUAL PATIENTS
The patient has a serious or life-threatening condition with no satisfactory alternative.
•    Assessment that benefits outweigh the risks to the patient.
•    Assessment that the company has an adequate supply of the investigational medicine.
•    A determination that expanded access will not interfere with the company’s ability to complete clinical trials in a timely fashion or which might otherwise delay marketing approval and ultimately availability to all patients.

Available Therapies via Single-Patient EA

Oral endoxifen (for pre-surgical and adjuvant treatment of Luminal A ER+ breast cancer; mammographic breast density)

Disease/Category-Specific EA Policies/Criteria

https://www.atossagenetics.com/wp-content/uploads/2019/07/AG-Expanded-Access-Policy.pdf

Single-Patient EA Policies/Criteria

Atox Bio is not accepting requests for expanded access at this time. The ongoing clinical trial is the most appropriate and expeditious way to advance therapies to patients. The ongoing trial can be viewed at clinicaltrials.gov.

Disease/Category-Specific EA Policies/Criteria

http://www.atoxbio.com/pipeline/aki/overview/

Single-Patient EA Policies/Criteria

The purpose of our compassionate use program is to make AVM0703 available to those patients who have exhausted other treatment options and who do not meet the criteria to enroll in a clinical trial. It is intended to improve access to AVM0703 for patients with serious or immediately life-threatening diseases or conditions who lack other therapeutic options and may benefit from it.

Available Therapies via Single-Patient EA

AVM0703 Injection is a novel and proprietary formulation of high concentration (24 mg/mL) of dexamethasone.

It is the subject of two clinical investigations:
• Leukemia and Lymphoma
• COVD-19 or Influenza (A or B) mediated ARDS (Acute Respiratory Distress Syndrome)

Disease/Category-Specific EA Policies/Criteria

https://avmbiotech.com/wp-content/uploads/2020/09/AVMExternal-EAP.pdf

Single-Patient EA Policies/Criteria

BerGenBio ASA is a clinical-stage oncology company focused on developing transformative drugs targeting AXL as a potential cornerstone of therapy for aggressive diseases, including immune-evasive, therapy resistant cancers. BerGenBio is currently developing a novel investigational medicinal drug product, bemcentinib (BGB324). Bemcentinib is in the early stage of clinical development, being evaluated as a treatment option in various oncology settings, including acute myeloid leukaemia (AML) and non-small cell lung cancer (NSCLC).

At BerGenBio, we are focused on enrolling and conducting clinical trials aimed at gaining regulatory approval, making bemcentinib available to patients as quickly as possible. As such, we believe participation in our clinical trial is the most appropriate way to access bemcentinib at this stage of development. Information about our clinical trials, including eligibility criteria and locations, is available at www.clinicaltrials.gov.

BerGenBio understands the interest of patients in accessing bemcentinib outside of clinical trials and prior to regulatory approval for potentially life-threatening diseases, however we do not currently offer any Expanded Access programs for bemcentinib.

BerGenBio recognizes the need for Expanded Access programs and will re-evaluate the status of our policy based on data from ongoing and future clinical trials.

Single-Patient EA Policies/Criteria

Requests for Early Access must be made by a physician on behalf of their patient.   A licensed physician overseeing the patient’s care, who is able to comply with Biohaven’s requirements, may contact Biohaven at BHV0223.ExpandedAccess@earlyaccesscare.com or call toll-free in the United States at 1-888-315-5797 (Option 6).

Single-Patient EA Policies/Criteria

Biosight is a clinical stage biotech company, developing innovative therapeutics for hematological malignancies and disorders. Biosight’s lead product, BST-236 (INN aspacytarabine), is an innovative proprietary anti-metabolite designed to enable high-dose therapy with reduced systemic toxicity. BST-236 is currently being investigated as a single agent in a phase 2b clinical trial as a first-line treatment of acute myeloid leukemia (AML) in patients unfit for standard chemotherapy. Additional studies in patients with relapsed or refractory AML and myelodysplastic syndrome (MDS) are under preparation.
At Biosight, we are focused on enrolling and conducting clinical trials aimed at gaining regulatory approval, making BST-236 available to patients as quickly as possible. As such, we believe participation in our clinical trials is the most appropriate way to access BST-236 at this stage of development. Information about our clinical trials, including eligibility criteria and locations, is available at www.clinicaltrials.gov.

Biosight understands the interest of patients in accessing BST-236 outside of clinical trials and prior to regulatory approval for potentially life-threatening diseases, however we do not currently offer any Expanded Access programs for BST-236.
Biosight recognizes the need for Expanded Access programs and will re-evaluate the status of our policy based on data from ongoing and future clinical trials.

Single-Patient EA Policies/Criteria

Biothera is evaluating the safety and efficacy of its proprietary innate immune modulator, Imprime PGG, in combination with various immune checkpoint inhibitors and monoclonal antibodies for the treatment of multiple cancers. At this time, Biothera does not have a compassionate use program; however, it will consider providing Imprime PGG for individual patients (Single Patient IND) on a case-by-case basis. This opportunity applies only to patients with serious or life-threatening conditions who are unable to participate in a clinical trial and for whom there are no comparable or satisfactory alternative therapy options.

In determining whether single patient access is appropriate outside of a clinical trial, Biothera will consider many factors, including: the strength of available efficacy and safety data; the patient’s medical condition and history; the benefit-risk profile of our drug in relation to the patient’s case; the potential impact on the clinical development program; and the ability and willingness of the patient’s physician to sponsor the treatment application.

If you are a potential patient and your physician believes you may be a candidate for single patient access to Imprime PGG, please ask your physician to contact Biothera at earlyaccess@biothera.com.

Available Therapies via Single-Patient EA

Imprime PGG

Single-Patient EA Policies/Criteria

This is Blaze Bioscience's expanded access policy for its investigational drug tozuleristide intended for patients with life-threatening diseases or conditions who have exhausted approved treatment options and are unable to participate in a clinical trial involving the investigational drug tozuleristide.

1. Contact Information: A licensed treating physician may submit questions or requests on behalf of a patient regarding expanded access to tozuleristide to be evaluated in accordance with Blaze Bioscience company policies. Physician Expanded Access Requests should be submitted in writing to expandedaccess@blazebioscience.com and include “Expanded Access Request” in the subject.

2. Request Procedures:
a. General Criteria: Blaze Bioscience will evaluate and respond to each Expanded Access Request individually and on a case-by-case basis. Criteria Blaze Bioscience will use in its evaluation of whether to grant Expanded Access Request include:
1) Adequate supply of the investigational drug tozuleristide must be available above and beyond the supply needed for Blaze Bioscience clinical trials;
2) There is sufficient clinical data to identify an appropriate dose of the investigational drug;
3) There is a good understanding of the patient’s clinical situation and investigational drug proposed use for surgery including the proposed fluorescence detection device;
4) All available therapeutic approaches for the patient’s disease have been exhausted by the patient and their physicians;
5) The investigational drug is considered an “eligible investigational drug” under Section 561(B)(2) of the FDCA at the time of the Expanded Access Request;
6) Providing the investigational drug is compliant with all applicable rules and laws;
7) Appropriate Institutional Review Board/Ethics Committee and FDA authorization requested expanded access has been obtained;
8) Treating physician understands and is willing to be responsible for ensuring that the patient informed consent requirements are met; and
9) Treating physician understands and is willing to be the holder of a treatment IND with FDA.

b. Timing of acknowledgement: Blaze Bioscience endeavors to acknowledge requests within ten (10) business days of receipt of an Expanded Access Request.
c. Clinical trials: Blaze Bioscience lists its active clinical trials on clinicaltrials.gov. Before granting an Expanded Access Request for tozuleristide, written confirmation by the treating physician that the patient is not eligible for an active Company clinical trial is needed prior to consideration of the Expanded Access Request.

Available Therapies via Single-Patient EA

tozuleristide

Single-Patient EA Policies/Criteria

Please see our Pre-Approval Access Policy on our website

Available Therapies via Single-Patient EA

Investigational medicine, avapritinib (formerly known as BLU-285)
See also:  https://clinicaltrials.gov/ct2/show/NCT03862885?term=avapritinib&draw=2&rank=4

Investigational medicine, pralsetinib (formerly known as BLU-667)
See also:  https://clinicaltrials.gov/ct2/show/NCT04204928?term=pralsetinib&draw=2&rank=1

Disease/Category-Specific EA Policies/Criteria

Avapritinib: Advanced gastrointestinal stromal tumors (GIST), advanced or smoldering systemic mastocytosis (SM), or another tumor type with a mutation in exon 17 of the KIT gene or in exon 18 of the PDGFRA gene

Pralsetinib: Advanced Non-Small Cell Lung Cancer (NSCLC) or Medullary Thyroid Cancer (MTC)

Available Therapies via Single-Patient EA

None

Disease/Category-Specific EA Policies/Criteria

https://celldex.com/docs/Compassionate_Use_Policy_MAY2018.pdf

Single-Patient EA Policies/Criteria

Cellectar Biosciences is a clinical stage biopharmaceutical company focused on the discovery, development and commercialization of drugs for the treatment of cancer. Our core objective is to leverage our proprietary phospholipid drug conjugates™ (PDCs™) delivery platform to develop PDCs that specifically target cancer cells to deliver improved efficacy and better safety as a result of fewer off-target effects. 

Consistent with Cellectar Biosciences’ mission to bring our PDCs™ to patients with cancer, we are focused on enrolling and conducting the clinical trials necessary to gain regulatory approvals to make our products available broadly to patients as quickly as possible. We are privileged to collaborate with clinical investigators and with patients who participate in our studies to develop new, safe and effective therapies. We believe this approach will serve patients who could be helped by the therapies we are developing. At the same time, we understand that there are seriously ill patients who will not be eligible for our clinical trials and may not have options for alternative therapies, including investigational therapies in trials being conducted by other sponsors. In these circumstances, Cellectar Biosciences will consider providing a requesting physician with pre-approval access to a specific Cellectar Biosciences investigational drug, for the treatment of an individual patient outside of a clinical trial, when certain conditions are met. These conditions include the following: 

•    The patient has a serious or life-threatening illness or condition and is either no longer responsive to or no longer able to tolerate any available treatment option; 
•    The investigational drug is in active clinical development with sufficient data available to determine an appropriate dose and schedule for the patient’s specific condition; 
•    A benefit-risk analysis, based on both the available clinical data as well as the requesting physician’s assessment of the individual patient’s condition and history, supports making the investigational drug available; 
•    Making the investigational drug available will not negatively impact or delay the conduct of clinical trials or regulatory review or approval of the investigational drug for broader patient access; and 
•    Adequate supply of the investigational drug is available. 

We continually evaluate the benefit-risk profile of each of our investigational drugs based on evolving clinical data. Each compound under development is different and the fact that one investigational drug is made available for the treatment of a particular patient does not mean it will be made available in response to other requests on behalf of other patients whose circumstances and medical histories may be different, or that a different investigational drug will be made available under our policy. Requests will be considered on a case-by-case basis. 

Cellectar Biosciences is committed to evaluating all requests in a fair and equitable manner. All requests must be submitted by the patient’s treating physician; Cellectar Biosciences may require more detailed information in order to fully evaluate a request. The requesting physician must agree to obtain appropriate regulatory and ethics committee approvals and comply with regulatory obligations, including obtaining patient consent, patient monitoring and safety reporting. Each request will be given careful consideration by Cellectar Biosciences whose decisions are final. Currently available therapies include CLR 131 for 4th line or later relapsed or refractory multiple myeloma and patients with relapsed or refractory diffuse large B-cell lymphomas (DLBCL) who have received at least two prior treatment regimens; and patients with relapsed or refractory lymphoplasmacytic lymphoma (LPL) or Waldenstrom’s Macroglobulinemia (WM) who have received at least two prior treatment regimens.

Physicians seeking pre-approval access for patients with no alternative treatment options should submit their requests to clinical@cellectar.com. We regularly monitor this mailbox and will use our best efforts to acknowledge each submitted request within 5 business days after receipt.

Available Therapies via Single-Patient EA

CLR 131 4th line or later relapsed or refractory multiple myeloma and patients with relapsed or refractory diffuse large B-cell lymphomas (DLBCL) who have received at least two prior treatment regimens; and patients with relapsed or refractory lymphoplasmacytic lymphoma (LPL) or Waldenstrom’s Macroglobulinemia (WM) who have received at least two prior treatment regimens.

Single-Patient EA Policies/Criteria

Cellphire Policy: Expanded Access Use of Thrombosomes®

Overview

This Policy regarding Expanded Access to Cellphire’s Investigational New Drug Thrombosomes (TBX), an activated freeze-dried platelet, is aimed at addressing the limited availability of platelets that are the result of the COVID-19 pandemic. Blood products are in critical need during this time frame and blood banks are faced with extremely limited supplies.

In the situation where a patient presents with WHO Grade 3 bleeding or higher and is in critical need of platelet/blood products, this Policy facilitates the use of TBX to treat the acute bleed and possibly mitigate the risk of additional critical bleeding. The benefit of the use of TBX in this critical set of circumstances is believed to outweigh the risks of the use of this investigational product.

Policy Statements

In cases where a clinical trial with Thrombosomes is not an option, Cellphire may elect to provide the Sponsor/Investigator expanded access to its investigational product, TBX. Treating Sponsor/Investigators and patients should note that clinical safety and efficacy of investigational products has not been fully established, so all potential risks and benefits should be carefully evaluated before seeking expanded access to this product. It is envisioned that requests could be made by Sponsor/Investigators as either an Individual Patient IND or Emergency Use Individual Patient IND. For more information on submitting requests to the FDA, click here.

This policy is aimed at addressing both types of requests. Cellphire will consider requests for access to TBX, according to internal Cellphire SOPs and as permitted by applicable law, in very specific circumstances, when certain criteria are met. For more detailed information, see Cellphire’s full policy here.

Requesting Access

Sponsor/Investigators seeking single patient expanded access (either emergency or non-emergency use) to TBX on behalf of their patient should call Cellphire at 301-545-2528 or submit an inquiry to expandedaccess@cellphire.com.

The request will promptly be addressed, generally within 24 hours of receipt by a member of either the Cellphire Clinical Affairs or Clinical Study Teams. Sponsor/Investigators should provide a written summary of the specific medical circumstances that require single patient expanded access and the need for treatment with TBX which briefly includes responses to each of the eligibility requirements listed above.

It should be noted that there is no guarantee that an expanded access request will be granted by FDA. Sponsor/Investigators who receive a TBX dose for their patients through the expanded access program must comply with all applicable FDA regulations, contractual conditions, safety reporting required by FDA, and protection of intellectual property.

This policy is subject to change. Cellphire will revisit the policy periodically and amend it as appropriate. This policy is not a guarantee of access to any of Cellphire’s investigational products.

Available Therapies via Single-Patient EA

Thrombosomes(R) - where a patient presents with WHO Grade 3 bleeding or higher and is in critical need of platelet/blood products, this Policy facilitates the use of TBX to treat the acute bleed and possibly mitigate the risk of additional critical bleeding.

Single-Patient EA Policies/Criteria

Due to the unique nature of personalized cell manufacturing and the high-level of monitoring required for patients treated with this product, an expanded access program to provide this therapy to patients at their local institution is not feasible at this time. For patients who may fit the indication to receive this therapy, and are between the ages of 3-30 with CD22 positive B-cell Acute Lymphoblastic Leukemia that is refractory or in second or later relapse, and either CD19 negative or relapsed/refractory to CD19 targeting, enrollment may be possible on the ongoing National Institutes of Health CD22 CAR T-cell clinical trial “Anti-CD22 Chimeric Receptor T Cells in Pediatric and Young Adults With Recurrent or Refractory CD22-expressing B Cell Malignancies” (NCT02315612).

Available Therapies via Single-Patient EA

https://clinicalstudies.info.nih.gov/ProtocolDetails.aspx?A_2015-C-0029.html

Single-Patient EA Policies/Criteria

Chiesi USA’s stated goals include the development of pharmaceutical solutions to improve the quality of human life and to combine commitment to result with integrity, operating in a socially responsible manner. In line with the Chiesi Mission and our core values, Chiesi USA will consider granting access to patients whenever possible who meet the following criteria:

- The patient has a serious or life-threatening illness with no comparable or satisfactory alternative therapies.
- The patient is ineligible for, or otherwise unable to, participate in a clinical trial related to the Investigational Product requested. Geographical limitations related to investigational product access will not necessarily render expanded access subjects ineligible.
- In the treating physician’s judgement, the potential patient benefit justifies the potential risks of the treatment use and those potential risks are not unreasonable in the context of the disease or condition to be treated.
- The Investigational Product is currently being studied in humans.
- Adequate supply exists or can be produced to support both the ongoing clinical investigations and compassionate use.
- Providing the Investigational Product for the requested use will not interfere with the initiation, conduct, or completion of clinical investigations that could support FDA approval of the expanded access use or otherwise compromise the potential development of the expanded access use.

Available Therapies via Single-Patient EA

Pegunigalsidase alfa for Fabry disease

Single-Patient EA Policies/Criteria

At Crestone, we are committed to developing CRS3123 as a first-in-class treatment for patients with Clostridioides difficile infection. We are conducting clinical studies to demonstrate safety and efficacy to obtain regulatory approval and ultimately make CRS3123 available. Currently, Crestone does not offer an expanded access program and does not accept expanded access requests outside of clinical trials.

Prior to regulatory approval by the U.S. Food and Drug Administration (FDA) and foreign regulatory authorities, patients gain access to investigational treatments, such as CRS3123, by participating in clinical trials. Expanded access is also referred to as “compassionate use”, “Early Access”, and “Emergency Use”. Patients who seek access to investigational medicines outside of an established clinical study and prior to health authority marketing authorization may wish to do so because standard treatments have failed, they cannot tolerate already approved medicines, because they are unable to participate in a clinical study or because there are no comparable or satisfactory therapy options available outside of clinical trials. The primary purpose of expanded access is to use the investigational drug for patient treatment purposes rather than to gather data on safety, tolerability, and effectiveness.

We believe that access to CRS3123 should be limited to clinical trials until such time as its safety, tolerability and effectiveness for a particular indication has been determined and confirmed by regulatory authorities. Patient access to CRS3123 outside of a clinical trial may interfere with the conduct of our ongoing and future clinical trials, and may also disrupt the progress of our development program, which would, in turn, delay access to many patients in need.
Accordingly, while clinical trials are ongoing, we believe participation in our clinical trials is the only safe and appropriate way to access CRS3123.
If you have questions about Crestone’s expanded access policy, please contact Crestone at drugsafety@crestonepharma.com. Please expect a response within five business days.

In line with the 21st Century Cures Act, Crestone may revise this policy at any time.

Single-Patient EA Policies/Criteria

CStone Pharmaceuticals (Suzhou) Co., Ltd. (CStone) is a biopharmaceutical company committed to bringing innovative therapies to patients by conducting clinical trials and obtaining marketing approval by the US Food and Drug Administration (FDA), China National Medical Products Administration (NMPA) and other regulatory authorities. Sugemalimab, an anti-PD-L1 monoclonal antibody, is currently investigated in a Phase 2 clinical trial for subjects with relapsed or refractory extranodal natural killer/ T cell lymphoma (R/R ENKTL), as well as other Phase 1-3 trials indicated for a variety of hematologic malignancies and solid tumors.

Expanded Access, which is sometimes known as “compassionate use”, is a potential pathway for a patient with an immediately life-threatening condition or serious disease or condition to gain access to an investigational therapy for treatment outside of clinical trials when no comparable or satisfactory alternative therapy options are available. For more information about expanded access in the US, please visit the FDA website at https://www.fda.gov/news-events/public-health-focus/expanded-access.

CStone focuses on conducting clinical trials aimed at gaining regulatory approval, making sugemalimab available to patients as quickly as possible. We seek to retain the ability to manufacture and supply sugemalimab in a fair and equitable manner and in a volume that assures adequate supply for ongoing clinical trials and development programs. As such, CStone believes participation in clinical trials is the most appropriate way to receive sugemalimab for patients seeking access before it is approved by a regulatory authority, and we do not offer any Expanded Access programs for sugemalimab at this time. Please visit https://clinicaltrials.gov and search by company, disease or medicine.

Consistent with the 21st Century Cures Act, CStone may revise this policy at any time.

Single-Patient EA Policies/Criteria

At Daiichi Sankyo, we recognize that there are instances when a patient has a serious or life-threatening disease or condition, for which all currently available treatment options have been exhausted and enrollment into a clinical trial is not possible. In these cases, in particular, a treating physician can request the use of an investigational Daiichi Sankyo product prior to regulatory approval for the particular condition or indication, provided it is allowed by the applicable local laws. 

Daiichi Sankyo strives for an equitable balance between the public interest in securing the approval of a new drug and allowing access to certain investigational medicinal products that may have the potential to treat seriously ill patients who cannot be satisfactorily treated with commercially available products.  The following criteria are typically required before Daiichi Sankyo would consider patients eligible for expanded access to a Daiichi Sankyo investigational product. All criteria are subject to local laws and regulations:
--The patient suffers from a life threatening or serious disease or condition, where there is an unmet medical need that cannot be met by existing products.
--The patient is ineligible or unable to participate in any ongoing clinical study of the investigational product.
--The investigational product is under active development (a dose and regimen has been defined and the product is typically in Phase 2 development or beyond) and has not been placed on regulatory or any other hold.
--The benefit-risk profile of the drug in the indication and the patient population is deemed, to date, to be positive.
--Daiichi Sankyo has decided to seek marketing approval in at least one major market globally.
--The expanded access use of the investigational product will not jeopardize the development of the drug (for instance, granting access to an investigational unapproved drug through expanded access should not delay, interfere with, or compromise the completion of clinical trials that are intended to support approval by regulatory authorities).
--The use of the investigational product in an expanded access protocol is compliant with local rules and regulations.

Daiichi Sankyo cannot approve expanded access use requests in cases where doing so would violate company policy, local laws or regulations, or if access would present an unacceptable benefit/risk profile, as deemed by medical personnel of the company.  We commit to a careful and fair evaluation of each request by the appropriate medical experts at Daiichi Sankyo within the boundaries of local laws and regulations.

Available Therapies via Single-Patient EA

We have a formal program for quizartinib (relapsed/refractory FLT3-ITD AML)

Daiichi Sankyo does not currently have formal expanded access programs for our other investigational products. We consider granting expanded access to products other than quizartinib on a case-by-case basis, as long as such provision will not delay, interfere with or compromise the completion of clinical trials that are intended to support approval by regulatory authorities, which, in turn, provides access to the medication for the greatest number of individuals.

Disease/Category-Specific EA Policies/Criteria

https://www.daiichisankyo.com/rd/pipeline/index.html

Single-Patient EA Policies/Criteria

Requesting Expanded Access
All requests for Expanded Access must be made by a treating physician on behalf of the child and the child's parent or adult guardian. DBV Technologies has opened an Expanded Access Program for Viaskin® Peanut post-trial treatment. A treating physician may submit questions or requests regarding Expanded Access by emailing dbvtechnologies.eap@earlyaccesscare.com or by calling Early Access Care at 203-441-7939 and speaking with an Early Access Coordinator. Patients must provide informed consent for Expanded Access. No patient is guaranteed entry into the program solely by applying. Requests will be acknowledged within one business day.

Disease/Category-Specific EA Policies/Criteria

https://www.earlyaccesscare.com/companies/dbv+technologies/viaskin+peanut

Available Therapies via Single-Patient EA

Debio 1143

Head & neck cancer

Debio 1143 is a potent, orally available, inhibitor of IAPs (Inhibitor of Apoptosis Proteins) that displays immunomodulatory properties making it a natural candidate for combination with Immune Checkpoint Inhibitors. In addition, like other members of the class, Debio 1143 promotes apoptosis of cancer cells by mimicking the activity of the natural Second Mitochondrial-derived Activator of Caspases (SMAC). Through this dual mode of action, Debio 1143 is expected to improve cancer patient treatment outcomes in combination with immunotherapy, chemotherapy and/or radiotherapy respectively investigated in NSCLC, H&N cancer and Ovarian Cancer.

Single-Patient EA Policies/Criteria

Epirium Bio is committed to developing safe and effective therapies for intractable neuromuscular and neurodegenerative diseases associated with mitochondrial depletion, as well as primary mitochondrial disorders, and providing those therapies to the broadest group of patients as quickly as possible. We also recognize that there are many diverse conditions in which mitochondrial depletion or dysfunction are a key component of the disease process. As part of our commitment to the rare disease community, we will support compassionate use / expanded access programs* when we have substantial scientific evidence to support both the safety and the potential efficacy of an investigational medical product for a given indication, and when it is logistically practicable.

Epirium has a developed a process for determining whether the company will provide an experimental therapy under compassionate use.
In the first step, the company will evaluate whether:
• there is substantial scientific evidence to support both the safety and the efficacy of an investigational medical product for a particular indication;
• it has been established that access on a compassionate use basis will not compromise clinical trials or the regulatory pathway for an investigational medical product;
• there is adequate supply of the investigational medical product; and
• the investigational medical product can be administered – and it is logistically feasible to make it available – outside of the clinical trial setting.
If the company decides that, under the first step, availability of the investigational medical product on a compassionate use basis is possible, then the company will evaluate an individual’s request for access.
This second step uses the following criteria:
• the patient has a serious disease or condition, or whose life is immediately threatened by their disease or condition;
• there is no comparable or satisfactory alternative therapy to diagnose, monitor, or treat the disease or condition;
• patient enrollment in a clinical trial is not possible;
• potential patient benefit justifies the potential risks of treatment;
• providing the investigational medical product will not interfere with investigational trials that could support a medical product’s development or marketing approval for the treatment indication, and;
• all necessary regulatory/institutional approvals have been obtained to allow the administration of the investigational medical product.
Requests for access to investigational medical products must be made by a qualified and licensed physician and will be evaluated by Epirium. Patients interested in seeking expanded access to an Epirium investigational medical product should talk to their physician. Qualified and licensed physicians may make compassionate use / expanded access requests by contacting Epirium by e-mail at info@epirium.com. Epirium anticipates it will acknowledge receipt of such requests within five business days of their receipt
* Compassionate use programs include requests for access under section 561(b) of the Federal Food, Drug, and Cosmetic Act, as well as ‘Right to Try’ legislation.

Available Therapies via Single-Patient EA

EPM-01 ((+)-epicatechin)

Disease/Category-Specific EA Policies/Criteria

https://epirium.com/wp-content/uploads/2020/05/Epirium_Bio_Statement_On_Compassionate_Use.pdf

Single-Patient EA Policies/Criteria

Erytech’s Public Policy – Compassionate Use (Expanded Access)

Erytech has no marketing approvals for any of its products throughout the globe.

Erytech is a science-led organization researching and developing new medicines through its ERYCAPS® platform. We are currently operating in North America and Europe.

This policy sets out the general principles for Erytech providing compassionate use. It is intended as global guidance, although Erytech will act in strict accordance with all local country laws and regulations on providing compassionate use.

Erytech’s lead product, eryaspase, is supported by clinical evidence from late stage clinical development¹.

Erytech is currently conducting late stage clinical trials in second-line pancreatic cancer (Trybeca-1) in Europe and the US, and in first-line Triple Negative Breast Cancer (Trybeca-2) in Europe. More details, including participating investigator centres, can be found on CT.gov or by sending an email to:

medaffairs@erytech.com

Erytech recognises that not all cancer patients will be suitable candidates for these studies. Where enrolment into a clinical trial is not an option for them, it may be in the interest of patients to have access to eryaspase. Under these circumstances, and where patients are in a life-threatening situation with no satisfactory alternative treatment options, Erytech may provide a treating healthcare professional (HCP) with an investigational medicine such as eryaspase.

As general guidance, Erytech will consider providing Compassionate Use according to the following:

• Erytech considers the appropriateness of compassionate use for all our investigational medicines early on in the planning of our research programmes. At an early stage in a medicine’s development there could be only limited understanding. For example:

• Uncertainty on the best way to provide the medicine to patients, such as the exact dose to use and frequency.
• Establishing the medicine’s efficacy and safety profile.

- Erytech provides compassionate use in two ways:

• Formal programmes
  • Patient populations meeting specific criteria managed under a compassionate use protocol.
• Named (or individual) patients where appropriate and subject to fulfillment of our criteria.

All requests for compassionate use, which must be made by an HCP, will be considered by Erytech and according to the following:
o Patients are in a life-threatening situation.
o There are no satisfactory alternative treatments (confirmed by the HCP).
o There is sufficient evidence to believe the potential benefit to the patient justifies the risk.
• Compassionate use decisions are made by Erytech’s Chief Medical Officer and are final.

Other key considerations include:

• Any concern that compassionate use of the investigational medicine might somehow compromise any related clinical trial or regulatory pathway.
• Restricted to countries where Erytech is able to provide its product. Currently, this is North America and Europe.
• That use in formal progammes will only take place in countries where Erytech intends to seek regulatory approval and to make the medicine available; the same limitation will not necessarily apply for named (or individual) patients.
• That the proposed compassionate use complies with local laws and regulations.

These criteria ensure Erytech has a consistent approach for our decisions on compassionate use.


To investigate further options for the appropriate access to Erytech’s medicines, any HCP can contact Erytech by sending an email to:

medaffairs@erytech.com

Erytech will acknowledge receipt of a compassionate use request submitted by an HCP within 48 hours.

Patients interested in accessing an Erytech medicine for compassionate use should talk to their doctors.

Erytech is committed to transparency in its interactions with HCPs and health care organizations/institutions consistent with applicable laws and/or codes of practice applicable to the pharmaceutical industry. Erytech will fulfil all regulatory requirements to make public information about our compassionate use activities.



1. Hammel P, Fabienne P, Mineur L, et al. Erythrocyte-encapsulated asparaginase (eryaspase) combined with chemotherapy in second-line treatment of advanced pancreatic cancer: An open-label, randomized Phase IIb trial. Eur J Cancer. 2019;124:91- 101.

Available Therapies via Single-Patient EA

Eryaspase

Currently under clinical study in:

- Pancreatic cancer
- Triple Negative Breast Cancer
- Acute Lymphoblastic Leukaemia (ALL)

Disease/Category-Specific EA Policies/Criteria

https://erytech.com/contact/compassionate-use/

Single-Patient EA Policies/Criteria

ER-004 COMPASSIONATE USE/EXPANDED ACCESS POLICY in XLHED

Expanded access/Compassionate use refers to the use of an investigational therapy, that have not yet been approved by government regulatory agencies, outside a clinical trial, for patients with serious or life- threatening diseases or conditions who lack therapeutic alternatives.

Esperare’s mission is to advance treatments for rare diseases and to promote treatment accessibility through a patient-centered approach.
Accordingly, a Patient Advisory Council dedicated to the XLHED program has been established and provides the opportunity to Esperare and patient groups representatives to work in close collaboration. In particular, clinical development plans are discussed in order to refine development and generate robust and necessary data for an application and to contribute to the common goal of giving the greatest chance of success to this program, with the ultimate objective of maximizing the benefits and minimizing the risks for XLHED patients and their families.

Requests for compassionate use/expanded access will be considered on a case by case basis , in accordance with applicable regulations and in alignment with our mission and values and with the safety of the patient and childbearing mother as a priority. Of note, ER-004 is delivered to the patient before birth by injection into the amniotic fluid, an untried route of administration that requires careful consideration in each individual case.

Inquiries regarding the expanded access may be sent to info.er004@esperare.org.
Esperare will endeavor to acknowledge receipt of any expanded access questions or requests within 5 business days.

Available Therapies via Single-Patient EA

ER-004 (also known as EDI200) is a protein replacement therapy designed as a substitute for endogenous EDA, a protein missing in XLHED.

Single-Patient EA Policies/Criteria

Expanded access, also called “compassionate use”, enables some patients with an immediately life-threatening condition or serious disease to gain access to an investigational new drug for treatment outside of clinical trials when no alternative therapy options are available. Currently, Forte does not offer an expanded access program. Forte may revise this policy at any time.

Disease/Category-Specific EA Policies/Criteria

https://www.fortebiorx.com/pipeline/default.aspx

Single-Patient EA Policies/Criteria

The IND has been deemed safe-to-proceed and the fast track designation has been granted. The registration in ClinicalTrials.gov will be done before the trial starts and a link will be provided then.

Single-Patient EA Policies/Criteria

https://www.gene.com/patients/investigational-medicines/criteria

Single-Patient EA Policies/Criteria

http://www.gsk.com/media/3368/compassionate-use.pdf

Go to GSK Compassionate Use Request Portal at https://gsk-cu-portal.idea-point.com/ to make a request.

Single-Patient EA Policies/Criteria

GMTUSA development resources are focused on conducting clinical studies required by regulatory authorities to fully answer important scientific questions about the potential risks and benefits of our investigational device treatment, and to obtain regulatory approval.

GMTUSA is committed to making investigational products available to seriously ill patients who have exhausted other treatment options. A treating physician, who is able to comply with the requirements that are stated in this document, may request information about how to apply for access to GMTUSA's investigational PCI-01 device treatment by contacting the Company.

The purpose of this policy is to describe the requirements for Expanded Access to GMTUSA investigational PCI-01 device treatment to patients outside of a clinical study.

Scope

This policy applies to provision of access to a GMTUSA investigational device product that is not approved for any purpose in the country from which the request is intended to be used. This also includes the time period between regulatory approval of an investigational product and its commercial availability in a country.

Policy Statements

Any use of a GMTUSA investigational product outside a clinical study in a country must be in accordance with local laws and regulations governing such programs, including COGMTUSA policies and procedures.

In general, where permitted by local regulation, the investigational product supplied via Expanded Access may no longer be provided by GMTUSA when it becomes available via the local healthcare system.

GMTUSA may decide not to provide an investigational product under this policy if the Company does not intend to market the product in the country.

A. Patient Eligibility Criteria

To be eligible for access to an investigational product, patients must meet the
following criteria:

• Suffer from a serious or immediately life-threatening disease or condition (COVID-19).
• Have undergone appropriate standard treatments without success and no comparable or satisfactory alternative treatment is available or exists to treat the disease or condition.
• Are ineligible for participation in any ongoing clinical study of the investigational product, which includes lack of access due to geographic limitations.
• The patient has a disease (COVID-19) for which there is sufficient evidence of a projected benefit from the use of the investigational product and the benefit outweighs the known or anticipated risks.
• There is adequate information to support appropriate dosing for a special population patients such as pediatric, elderly, renal or hepatic disease, etc.
•Any other pertinent medical criteria for access to the investigational product, as established by the GMTUSA clinically or medically responsible individual.

B. Investigational Product Criteria

In addition to the patient eligibility requirements, the investigational product must meet the following criteria:
• The product is under investigation in one or more clinical studies.
• There is sufficient evidence to expect that the investigational product will have an acceptable safety profile for the intended patient population.
• The provision of the investigational product will not interfere with or compromise the clinical development of the product.

C. Treating Physician Criteria and Responsibilities

The physician(s) attending to the patient(s) who is/are receiving an investigational product through compassionate use access is (are) properly licensed and fully qualified to administer the product. The physician must agree in writing to comply with:
• Any applicable country-specific legal and regulatory requirements related to providing an investigational product under Expanded Access.
• Any CGMTUSA requirements in terms of medical criteria, safety reporting, drug supply/use and protection of intellectual property. A treating physician may submit questions or requests regarding expanded access to drfernandez@gmtpci.com

Single-Patient EA Policies/Criteria

Gyroscope Therapeutics’ mission is to preserve people’s sight and fight the devastating impact of blindness around the world. We embarked on this journey because each year millions of people lose their vision due to diseases that we believe may one day be treatable. Our current focus is to discover and develop pioneering gene therapies for one of the leading causes of vision loss – age-related macular degeneration (AMD).

To realise this mission, it is our duty to conduct the research and clinical trials necessary to evaluate the safety and effectiveness of our investigational medicines. We believe that rigorous clinical trials are the most appropriate way to do this. We are committed to working closely with researchers, doctors, clinical trial site teams, patients and families to enrol, conduct and analyse our studies. If successful, data from these studies may support submissions to regulatory authorities (such as the FDA and EMA) for potential approval – with the ultimate goal of making our medicines available to as many eligible patients in need as safely and quickly as possible.

At this time, we are not making our investigational gene therapy, GT005, available on an expanded access basis anywhere in the world (expanded access is sometimes also referred to as compassionate use or pre-approval access). For now, participation in one of our clinical trials is the only way to receive our investigational gene therapy. This decision was made after careful evaluation of many factors, including: the safety and effectiveness of GT005 is still being evaluated in early stage clinical trials; our manufacturing capacity for GT005; considerations related to one-time gene therapies; and, what we believe is in the best interest of patients.

We understand there are currently no approved treatments for dry AMD, which is why we are working hard to develop our gene therapies as quickly as we can. There are many clinical trials evaluating potential new therapies for this devastating condition. We recommend that you talk to your doctor about what is right for you.

If you are interested in participating in one of Gyroscope’s clinical trials and would like more information, or if you have any questions about this expanded access policy, please talk to your doctor or contact us. We anticipate acknowledging receipt of expanded access questions or requests sent to us within five (5) business days of receipt.

More information about Gyroscope’s ongoing clinical trials can be found by visiting the links below.

We will continue to evaluate the possibility of expanded access as we advance development of our investigational medicines. As authorised by the 21st Century Cures Act, Gyroscope may revise this posted expanded access policy at any time.

The availability of this policy or any revised version shall not serve as a guarantee of access to GT005 or any other investigational medicines by any individual patient.

Current trials of GT005 in geographic atrophy secondary to dry AMD:

FOCUS Phase I/II Trial

EXPLORE Phase II Trial

Available Therapies via Single-Patient EA

At this time, none of our therapies are available via single-patient EA. 

Disease/Category-Specific EA Policies/Criteria

https://www.gyroscopetx.com/expanded-access/

Single-Patient EA Policies/Criteria

Any use of Hanmi’s investigational products outside clinical trials should be made in accordance with local regulations and guidelines as well as Hanmi’s procedures and policies.
Hanmi will not grant access to Hanmi’s investigational products if the expanded access will interfere with the company’s ability to complete clinical trials as planned or will delay the drug development program.
Hanmi will grant access to Hanmi’s investigational products when there is an adequate supply of the investigational products to meet the needs of the expanded access without impairing clinical trials for drug development.
Hanmi will provide investigational product with 3 months’ treatment supply per case. In case treating physician judges that it is beneficial for patients to continue Hanmi’s investigational products, new apply should be made.
In general, where permitted by local regulation, an investigational product provided via expanded access according to local regulations and guidelines will no longer be provided by Hanmi once it becomes available on the market.
Hanmi may decide not to provide an investigational product under this policy if Hanmi decides to stop the drug development or not to launch in the concerned country(ies).

Available Therapies via Single-Patient EA

LAPS-Triple Agonist (also known as HM15211) is a long-acting Glucagon/GIP/GLP-1 triple agonist chemically conjugated with constant region of human immunoglobulin via non-peptidyl flexible linker. The therapeutic indication is non-alcoholic steatohepatitis (NASH).

Disease/Category-Specific EA Policies/Criteria

http://www.hanmipharm.com/ehanmi/handler/Rnd-FocusedPipelineB

Single-Patient EA Policies/Criteria

HiberCell, Inc. is committed to developing novel therapeutics that address the most common cause of cancer mortality: relapse and metastasis. HiberCell has multiple compounds in development focused on modulating the immunosuppressive tumor microenvironment and adaptive stress biology. Currently one of its therapies, Imprime PGG, an innate immune modulator, has advanced into clinical testing (Phase 2). Clinical studies evaluating the safety and efficacy of Imprime PGG in combination with immune checkpoint inhibitors, tumor-targeting, and anti-angiogenic antibodies for the treatment of multiple cancers have been conducted. At this time, HiberCell does not have a compassionate use program; however, it will consider providing Imprime PGG for individual patients (Single Patient IND) on a case-by-case basis. This opportunity applies only to patients with serious or life-threatening conditions who are unable to participate in a clinical trial and for whom there are no comparable or satisfactory alternative therapy options.

In determining whether single patient access is appropriate outside of a clinical trial, HiberCell will consider many factors, including: the strength of available efficacy and safety data; the patient’s medical condition and history; the benefit-risk profile of our drug in relation to the patient’s case; the potential impact on the clinical development program; and the ability and willingness of the patient’s physician to sponsor the treatment application.

If you are physician or a potential patient and your physician believes you may be a candidate for single patient access to Imprime PGG, please ask your doctor to contact Michele Gargano, VP Clinical Operations, HiberCell, at mgargano@hibercell.com, who will respond within 5 working days.

Available Therapies via Single-Patient EA

Imprime PGG, for multiple oncology indications, in combination with checkpoint inhibitor, tumor-targeting- or anti-angiogenic antibodies

Disease/Category-Specific EA Policies/Criteria

https://www.hibercell.com/programs/

Single-Patient EA Policies/Criteria

Imara is dedicated to developing novel therapeutics for patients with sickle cell disease and other hemoglobinopathies. Sickle cell disease represents a critical unmet medical need globally in which it is considered as a rare disease in many parts of the world, including in the United States, and as an endemic condition in several African countries. Imara’s lead product candidate, IMR-687, is under development for the treatment of sickle cell disease.

Expanded access may provide an avenue to use an investigational product, such as IMR-687, outside a clinical trial to diagnose, monitor, or treat a serious condition or disease in a patient. Imara has used guidelines from the US Food and Drug Administration (US FDA) and other regulatory agencies to develop the following criteria for when expanded access may be made available on a case-by-case basis for individual patients: 

•    Ongoing or planned clinical studies are not available to the patient, including lack of access due to geographical location of potential clinical trial sites, 
•    Significant evidence exists that supports both the safety and the efficacy of the investigational drug for the indication, 
•    The potential benefits to the particular patient seeking access to the investigational drug outweigh the potential risks to the patient,
•    An adequate supply of the investigational drug exists,
•    All necessary regulatory/institutional approvals have been obtained to allow drug administration, and 
•    The request for expanded access has been made by a qualified health care provider with expertise appropriate for the administration of the drug and for monitoring and managing the patient.

Imara supports expanded access programs and the need for a suitable policy, and it intends to provide patients with sickle cell disease access to IMR-687 at a suitable time and in the correct method when used outside a clinical trial. At this time, Imara believes that the most appropriate way to use our investigational lead product candidate, IMR-687, is by participation in one of our clinical trials (https://clinicaltrials.gov/).

Available Therapies via Single-Patient EA

IMR-687 for sickle cell disease

Single-Patient EA Policies/Criteria

http://immixbio.com/pipeline/compassionate-use-policy/

Available Therapies via Single-Patient EA

imx-110 - all advanced solid tumors

Single-Patient EA Policies/Criteria

Immunicum is currently unable to offer an Expanded Access

Available Therapies via Single-Patient EA

Currently, Immunicum does not have any investigational cell therapy products
available for Expanded Access

Single-Patient EA Policies/Criteria

Intensity Therapeutics’ (the Company) is the developer and drug manufacturer for a novel investigational drug product, INT230-6. This new product is in early clinical development and uses intratumoral injection for its delivery modality. Recently the FDA granted Fast Track Status to INT230-6 for development in patients with triple negative breast cancer who have failed 2 lines of therapy.

The drug is being used to treat solid tumor cancers. Intensity Therapeutics has chosen not to offer EA and will not accept EA requests for its products at this time.

Available Therapies via Single-Patient EA

There are no therapies available for single-patient EA at this time.

Single-Patient EA Policies/Criteria

Iterum Therapeutics was founded with one over-arching goal: to develop an antibiotic that addresses the growing crisis of multi-drug-resistant pathogens and meets the specific needs of physicians, their patients and other important stakeholders. Among the most important demands were new treatments for common, often serious conditions, including urinary tract infections (UTI), and intra-abdominal infections (IAI). Iterum Therapeutics is committed to conducting the clinical trials required to gain regulatory approvals to allow our medicines to be available to patients. Patients are encouraged to participate in one of our clinical trials whenever possible. For those patients with an unmet need who have exhausted all available therapy options and who are unable or ineligible to participate in a clinical trial, we may be able to provide access to our investigational medicine(s) in certain circumstances through our expanded access program.
Iterum Therapeutics may provide access for patients to our investigation medicine(s) outside of a clinical trial provided the patient meets Iterum Therapeutics’ evaluation criteria for such access. At a minimum, requests must meet the following criteria in order to be considered:
•    The patient has a serious or immediately life-threatening illness when there are no comparable or satisfactory alternative treatments available and the patient is ineligible or unable to participate in a clinical trial;
•    The benefit-risk analysis of the patient’s condition and medical history support the use of an investigational drug and justifies the potential risks of the treatment;
•    Making the investigational therapy available for expanded access will not interfere with the initiation, conduct, or completion of clinical trials being conducted by Iterum Therapeutics that could support marketing approval for a particular treatment indication;
•    The investigational drug is in active clinical development with sufficient data available to determine an appropriate dose and schedule for the patient’s specific condition;
•    There is an adequate supply of the investigational medicines;
•    Provision of such access to investigational medicines is compliant with local regulations and laws.

All requests meeting the above criteria must be submitted by the patient’s treating physician to EAProgram@iterumtx.com. Iterum Therapeutics may request additional information from the patient’s treating physician to support the request. The treating physician must also agree to obtain appropriate regulatory and Institutional Review Board approvals and patient informed consent and to carry out patient monitoring and safety reporting. 

Iterum Therapeutics is committed to assessing every early access request with the highest degree of fairness and equality, however, Iterum Therapeutics cannot guarantee access to any investigational medicine. Iterum Therapeutics regularly monitors this email box and will attempt to acknowledge each submitted request within 3 business days after receipt. 

Available Therapies via Single-Patient EA

Sulopenem sodium (intravenous)
Sulopenem etzadroxil/probenecid (tablet)

Single-Patient EA Policies/Criteria

http://www.janssen.com/sites/www_janssen_com/files/janssen_pre-approval_access_policy_022017.pdf

Single-Patient EA Policies/Criteria

At this time, we do not have an expanded access program that allows patients to have access to our investigational products prior to completing clinical phase II study. The IND of Meplazumab for Injection (IND 143872) has been deemed partical safe-to-proceed and the fast track designation has been granted on Feb 14th, 2020. The registration in ClinicalTrials.gov will be done before the trial starts and a link will be provided then.

Single-Patient EA Policies/Criteria

To serve patients, KalVista Pharmaceuticals (KalVista) engages in clinical research with the goal of obtaining regulatory approval of its products.

Clinical trials allow KalVista to evaluate new treatments in patients in order to generate the safety and efficacy information needed to obtain approval of those treatments and make them available to the broader patient population. Outside of a clinical trial, access to KalVista’s investigational products would be considered only under limited circumstances, and as permitted by applicable law, in the following situations:
   - KalVista may provide continued access to its investigational products to participants in a clinical trial once the trial is complete.
   - KalVista may provide physician-requested expanded access (also known as compassionate use) to its investigational products to patients who cannot join an active clinical trial of the investigational product providing the criteria below are met.

Available Therapies via Single-Patient EA

Paxalisib (formerly known as GDC-0084)
 

Disease/Category-Specific EA Policies/Criteria

https://www.kaziatherapeutics.com/researchpipeline/paxalisib

Single-Patient EA Policies/Criteria

KC Pharma DFMO EXPANDED ACCESS POLICY

KC Pharma (KCP) is committed to serving patients by providing safe, fair, and sustainable patient access to its medicines. KCP may be able to provide patients access to its investigational drug, DFMO (also called eflornithine or difluoromethylornithine), outside of a clinical trial if certain conditions are met and if permitted by local law and regulation. The use of an investigational medical product (i.e., one that has not been approved by the Food and Drug Administration [FDA]) outside of a clinical trial is known as Expanded Access (sometimes referred to as “compassionate use”). Generally, there are two types of Expanded Access—Group Expanded Access and Individual Patient Expanded Access. For more information on the different types of Expanded Access, visit the following FDA website: https://www.fda.gov/news-events/public-health-focus/expanded-access.

KCP collaborates with Beat Childhood Cancer Research Consortium (BCC) and Sponsor-Investigator, Dr. Giselle Sholler and makes investigational product DFMO 192 mg tablets available for pediatric oncology indications through multiple clinical trials and a Group Expanded Access Program (also called Intermediate Population Expanded Access). KCP does not currently provide Individual Patient Expanded Access.

Expanded Access may not always be available. Information about the Group Expanded Access Program, including a full list of eligibility criteria and site contact information can be found here: https://clinicaltrials.gov/ct2/show/NCT03581240. In addition to meeting the eligibility criteria outlined at the link above, the following criteria must be met for entry into the Group Expanded Access Program:

• KCP must have sufficient supply of DFMO to reasonably accommodate the anticipated duration of treatment.

• The patient’s licensed physician must be willing to collaborate with the enrolling center.

• The patient’s physician and enrolling physician determine there is no comparable or satisfactory therapy available to treat the patient’s disease or condition and they agree that the patient is clinically stable and able to receive this medication.

• The patient’s parent/guardian is willing to travel with the patient to BCC at study entry and every three months while on treatment.

Licensed physicians may contact KCP with general requests or questions regarding Expanded Access at DFMOExpandedAccess@usworldmeds.com. KCP will endeavor to acknowledge general requests and questions within 5 business days. In line with the 21st Century Cures Act, KCP cannot guarantee access to investigational product to all patients. KCP may revise this policy at any time; at such time, the revised policy will be made publicly available.

Single-Patient EA Policies/Criteria

We are privileged to collaborate with clinical investigators and patients who participate in our studies to develop new, safe and effective therapies. At the same time, we understand that there are patients who will not be eligible for our clinical trials and may not have options for effective alternative therapies. In these circumstances, Krystal will consider providing a requesting physician with pre-approval access to a specific Krystal investigational drug for the treatment of an individual patient outside of a clinical trial when certain conditions are met. These conditions include, but are not limited to, the following:
•    The patient’s serious or life-threatening condition limits their ability to comply with certain clinical trial requirements, such as travel;
•    The investigational drug is in active clinical development with sufficient data available to determine an appropriate dose and schedule for the patient’s specific condition;
•    A benefit-risk analysis, based on both the available clinical data as well as the requesting physician’s assessment of the individual patient’s condition and medical history, supports making the investigational drug available;
•    Making the investigational drug available will not negatively impact or delay the conduct of clinical trials, regulatory review, or approval of the investigational drug for broader patient access; and
•    Adequate supply of the investigational drug is available. 
We continually evaluate the safety and efficacy profile of each of our investigational drugs based on evolving clinical data. Each disease, patient, and investigation drug under development is unique, and as such, requests will be considered on a case-by-case basis. 
Krystal is committed to evaluating all requests in a fair and equitable manner. All requests must be submitted by the patient’s treating physician; Krystal may require more detailed information in order to fully evaluate a request. The requesting physician must agree to obtain appropriate regulatory and ethics committee approvals and comply with regulatory obligations, including obtaining patient consent, patient monitoring and safety reporting. Each request will be given careful consideration by Krystal, whose decisions are final.

Available Therapies via Single-Patient EA

Bercolagene Telserpavec (KB103) - Dystrophic Epidermolysis Bullosa

Disease/Category-Specific EA Policies/Criteria

https://www.krystalbio.com/wp-content/uploads/2019/07/Krystal-Expanded-Access-Policy.pdf

Single-Patient EA Policies/Criteria

Kymab is dedicated to developing novel human antibody-based therapeutics across a broad range of indications including immune mediated diseases.

Expanded access permits the use of an investigational therapy in patients with a serious or life-threatening illness when no satisfactory preventive or treatment options are available, and the patient is ineligible to participate in a clinical trial. Kymab understands and respects the need for appropriately managed expanded access program(s), and accordingly, this policy has been generated.

The following criteria are informed by regulatory guidelines for when expanded access may be made available to an investigational product on a case-by-case basis for individual patients:
• Ongoing or planned clinical studies are not available to the patient, including lack of access due to geographical location of potential clinical trial sites;
• Significant evidence exists that supports both the safety and the efficacy of the investigational drug for the indication;
• The potential benefits to the particular patient seeking access to the investigational drug outweigh the potential risks to the patient;
• An adequate supply of the investigational drug exists;
• All necessary regulatory/institutional approvals have been obtained to allow drug administration, and
• The request for expanded access has been made by a qualified health care provider with expertise appropriate for the administration of the drug and for monitoring and managing the patient.

KY1005 is currently in early clinical development. Kymab believes that the most appropriate way to ensure appropriate use of KY1005 at this stage in development is via approved clinical trials. The initial clinical trial(s) will permit the effective generation of safety and efficacy data for KY1005 which, when shared with the Regulatory Authorities will further guide the decision making regarding its safe and effective use in an expanded access program.

At this time, therefore, Kymab will not offer expanded access and will not currently accept expanded access requests for KY1005. Information regarding KY1005’s clinical trials will be made available at https://clinicaltrials.gov/.

If you have additional questions, please speak with your health care provider or contact KY1005ExpandedAccess@Kymab.com.

In accordance with the US 21st Century Cures Act, Kymab may revise this policy at any time.

Single-Patient EA Policies/Criteria

Poteligeo was approved by the FDA on August 8, 2018 for the treatment of adult patients with relapsed or refractory mycosis fungoides (MF) or Sézary syndrome (SS) after at least one prior systemic therapy. Only patients who have relapsed or refractory MF or SS after at least one prior systemic therapy will be eligible to participate.
The main purpose of the EA program is to facilitate the access of Poteligeo to patients who lack therapeutic alternatives and in which there is a reasonable expectation that the provision of access to Poteligeo will have a positive benefit/risk for the patient. The intention was for the EA program to fill the time between approval by the FDA and the date of availability of commercial supply to patients. Commercial supply is now available and the EA program ended on November 1, 2018.

Disease/Category-Specific EA Policies/Criteria

https://www.leaptx.com/for-patients

Single-Patient EA Policies/Criteria

Expanded Access Program Policy for LNS8801
LNS8801 is currently being developed for the treatment of advanced cancer and has fast track designation for the treatment of patients with metastatic or unresectable melanoma who have progressed on or after anti–programmed cell death receptor or ligand (anti–PD-1/L1) therapy. Linnaeus Therapeutics (Linnaeus) understands that advanced cancer patients with limited therapeutic options may desire to try promising experimental therapies such as LNS8801. The best way for patients to get access to investigational medicines is by taking part in clinical trials. Please search ClinicalTrials.gov for publicly available information related to Linnaeus’s ongoing clinical trials.

It is recognized that not all patients are eligible to enroll in clinical trials. Linnaeus is unfortunately unable to consider requests for expanded access (sometimes called compassionate use) at this time due to limited drug supply.

Linnaeus hopes that in the near future it will be able to consider requests so that patients in the United States with an immediately life-threatening condition or serious disease or condition who have exhausted other appropriate treatment options may, under the conditions described below and in accordance with applicable local law, have appropriate access to its investigational medicines in development before they are commercially available.

For more information regarding Linnaeus’ current and future expanded access policies please contact, medinfo@linnaeustx.com. Linnaeus anticipated being able to response to inquiries within one week.

Available Therapies via Single-Patient EA

LNS8801 - advanced cancers

Single-Patient EA Policies/Criteria

At Lumicell, our focus is on the development of pegulicianine. The goal of our current clinical study program is to enroll patients and obtain clinical data on pegulicianine that is required by the US Food and Drug Administration (FDA) and other regulatory authorities for review and approval of marketing applications for this investigational agent. We believe that focusing on clinical studies is the most appropriate way to achieve this goal; therefore, we do not currently offer access to pegulicianine outside of clinical studies and, at this time, we have no Expanded Access program (sometimes referred to as “compassionate use” or “pre-approval access”).
For information on participating in and status of pegulicianine clinical trials, please see: https://clinicaltrials.gov/ct2/home
If you have additional questions regarding this policy, please speak with your health care provider or contact: info@lumicell.com

Single-Patient EA Policies/Criteria

MacroGenics designs its clinical studies for development of new potential therapies to determine the safety and efficacy for medical use consistent with approval requirements of the US FDA and similar regulatory authorities in other countries. Treating physicians should consider approved therapies for a patient’s disease as well as on-going clinical studies before seeking expanded access use with an investigational agent. However, MacroGenics may, on a case-by-case basis, consider requests by treating physicians to file an investigational new drug application for expanded access to MacroGenics investigational products in Phase 2 or Phase 3 of development and for MacroGenics to supply such investigational product. There is no guarantee of expanded access to an investigational product.

Available Therapies via Single-Patient EA

MacroGenics currently has an Expanded Access program open for margetuximab for patients with pretreated HER2+ breast cancer for whom potential benefit justifies potential treatment risks.

Single-Patient EA Policies/Criteria

Meissa Vaccines Inc. is developing an investigational vaccine MV-012-968 designed to prevent disease caused by respiratory syncytial virus (RSV) and the vaccine is in very early stages of clinical development.
Since at this time there is insufficient safety and efficacy data to determine the risk:benefit profile of the vaccine, and the vaccine is designed for prophylactic not treatment use, Meissa Vaccines Inc. (https://www.meissavaccines.com/) has chosen not to offer Expanded Access (EA) to MV-012-968, and will not accept EA requests. MV-012-968 will only be provided in the course of ongoing clinical trials.
Meissa Vaccines Inc. Expanded Access Policy, Rev. December 27, 2019.

Single-Patient EA Policies/Criteria

Consistent with Melinta Therapeutics’s mission to discover, develop, and commercialize a continual stream of novel antibiotics, we are focused on enrolling and conducting the clinical trials necessary to gain regulatory approvals and promptly make our medicines available to patients.  At Melinta Therapeutics, we believe that wherever possible, the use of an investigational medicine for a patient as part of a clinical trial is preferable; however, when patients with an unmet medical need are unable to access our investigational products through a clinical trial and all other options have been exhausted, we may be able to provide access through our compassionate use or expanded access program.

The evaluation criteria to determine if Melinta Therapeutics can provide expanded access to a specific Melinta Therapeutics investigational agent outside of a clinical trial include all of the following:
•    The patient has a serious or life-threatening illness or condition and is either no longer responsive to or no longer able to tolerate any available treatment option;
•    The investigational drug is in active clinical development with sufficient data available to determine an appropriate dose and schedule for the patient’s specific condition;
•    A benefit-risk analysis, based on both the available clinical data as well as the requesting physician’s assessment of the individual patient’s condition and history, supports making the investigational drug available;
•    Access is compliant with local rules and laws;
•    Making the investigational drug available will not negatively impact or delay the conduct of clinical trials or regulatory review or approval of the investigational drug for broader patient access; and
•    Adequate supply of the investigational drug is available.

All requests must be submitted by the patient’s treating physician and Melinta Therapeutics may require more detailed information in order to fully evaluate a request. The treating physician must agree to obtain appropriate regulatory and ethics committee approvals and comply with regulatory obligations, including obtaining patient consent, patient monitoring and safety reporting. Treating physicians seeking expanded access, meeting the above-mentioned evaluation criteria, should submit their requests to medinfo@melinta.com. We regularly monitor this mailbox and will use our best efforts to acknowledge each submitted request within 3 business days after receipt. Melinta Therapeutics is committed to carefully evaluating requests on a case-by-case basis and in a fair and equitable manner; however, Melinta Therapeutics cannot guarantee access to any specific investigational drug by any individual patient.

If you are a patient with questions concerning access to our investigational products, either through a clinical trial or through expanded access, please consult with your treating physician.

Single-Patient EA Policies/Criteria

Criteria for Evaluation

Merus considers many factors when evaluating a request for early access of an investigational medicine, such as (but not limited to) the strength of the currently available data, including clinical data together with patient condition and the benefit-risk profile, the impact of providing access to an investigational medicine on its clinical development program, including the ability to complete patient enrollment and other necessary aspects of the clinical trials, the phase of development, and probability and timing of regulatory approval.

A patient may be considered for early access to an investigational medicine based upon evaluation of the following minimum requirements (and other requirements may apply based on the circumstances):

*There must be adequate data supporting use of the investigational medication in the treatment of the patient’s disease.
*There are no adequate standard or investigational therapies available.
*Sufficient safety and efficacy data must exist for use of the medicine at the time of request.
*Sufficient information must be made available to make a benefit-risk analysis consistent with the establishment of an early access program.
*Sufficient clinical data is available to identify an appropriate patient-specific dose.
*A patient’s treating physician and Merus’ clinical team both believe there is the potential for the specific patient under consideration to reasonably expect benefit from the treatment.
*Adequate supply exists to support both the ongoing clinical trials and approved early access, until and if a product becomes commercially available.
*The patient is not eligible for or cannot participate in a Merus-sponsored study for the therapy being requested.
*Early access will not adversely impact the clinical development program or the regulatory approval process.
*The request must be made, unsolicited, by the patient’s treating physician. The physician must be appropriately licensed.
*The treating physician must also agree to comply with the safety and monitoring requirements.
*Where appropriate, demonstration of efficacy and safety may be required for continued treatment.
*Early access is permitted or approved in the country, region, and/or state where the investigational medicine will be administered.
*Any condition, in addition to those listed above, that in the opinion of Merus may preclude appropriate use of the investigational medicine, will be considered.
Given the unique nature of individual requests for early access to investigational medications, there is no guarantee a request will be fulfilled.

Merus may revise, suspend or terminate the early access program at any time.

All early access requires approval by the Chief Medical Officer and Merus management and decisions will be made based on scientific and other evidence available to Merus at the time of the request.

Available Therapies via Single-Patient EA

MCLA-128 is available for patients with solid tumors that harbor an NRG1 fusion

Disease/Category-Specific EA Policies/Criteria

https://nrg1.com/

Single-Patient EA Policies/Criteria

At this time, Metacrine believes access to its products should be limited to clinical trials and does not offer an expanded access program or accept expanded access requests.

Single-Patient EA Policies/Criteria

Met Vital, Inc. will consider providing pre-approval access to our investigational drug, outside of the clinical trial setting, when certain conditions are met. These conditions are as follows:
• The patient to be treated has a serious or immediately life-threatening illness and there is no satisfactory alternative therapy.
• The patient is not eligible for, or cannot access, any ongoing clinical trials.
• The potential benefit of the investigational medicine to the patient, outweighs the potential risk. This should be evaluated by your physician and discussed in detail with the patient.
• There is an adequate supply of the investigational drug, meaning that MetVital, Inc has surplus investigational drug over and above what is required for the ongoing clinical trial.
• Providing the investigational drug will not interfere with the clinical trial that could support a marketing approval.

Requests for our investigational drug must come from a patient’s treating physician. The requesting physician must be willing to work on designing a treatment protocol and obtain the appropriate regulatory and ethics committee approvals from their Institutional Review Board (IRB). MetVital, Inc will not be responsible for the physician gaining IRB approval. The physician must also comply with various regulatory obligations, including obtaining patient consent, patient monitoring and safety reporting.

Available Therapies via Single-Patient EA

Anhydrous enol-oxaloacetate for newly diagnosed Glioblastoma multiforme

Disease/Category-Specific EA Policies/Criteria

http://metvital.com/

Available Therapies via Single-Patient EA

Currently, none.

Single-Patient EA Policies/Criteria

Minoryx is a clinical stage biotech company committed to the development of safe and effective novel therapies for severe and life-threatening orphan CNS diseases with high unmet medical needs. We are a science-driven company committed to putting patients first. Our goal is to provide access to our investigational therapies at the appropriate time and in the correct manner for patients.
Clinical trials and the subsequent timely generation of safety and efficacy data are the most effective way of ensuring review and decision making by Regulatory Authorities. This ultimately has resulted in access to new, safe and effective approved medicines for patients.
Expanded access refers to the use of an investigational therapy outside a clinical trial when the primary purpose is to diagnose, prevent, or treat a serious condition in a patient. We understand the need for expanded access programs, and we recognize the importance of having an appropriate policy.

Disease/Category-Specific EA Policies/Criteria

FDA Orphan indication was granted for the company’s lead program, leriglitazone (MIN-102), a novel, selective peroxisome proliferator-activated receptor gamma (PPARγ) agonist, for the treatment of X-ALD. On 4 April 2018, Minoryx opened the IND for leriglitazone (company code MIN-102) with a double-blind, randomized, placebo-controlled study to assess the effect of leriglitazone on the clinical progression in male patients with Adrenomyeloneuropathy (AMN). Recently, the FDA granted Fast Track Status to leriglitazone for development in patients with X-ALD.
An Early Access program for leriglitazone may be opened if Minoryx determines that all the following criteria are met:
• The investigational drug is the subject of an active clinical development program and is not approved in any indication in the country concerned
• An adequate supply of the investigational drug exists to perform necessary clinical studies as well as to provide Early Access to patients who do not have alternative treatment options
• Early Access does not impede or compromise the clinical development or regulatory approval of the drug under investigation
• There are sufficient clinical data available with respect to both the investigational drug and the disease condition for which the application is being sought, to anticipate that any potential benefits from treatment are likely to outweigh any potential risks to the patient
At this time, Minoryx believes that participation in one of our clinical trials is the most appropriate way to access our investigational therapies. Minoryx continues to assess the eligibility requirements and criteria for Early Access to the investigational drug leriglitazone and will re-evaluate this policy from time to time.
If you have additional questions, please speak with your physician or contact clinical@minoryx.com. We anticipate acknowledging receipt of requests sent to this email within five business days.

Single-Patient EA Policies/Criteria

Minovia will make every attempt to respond to EAP request as soon as possible. An acknowledgement of receipt of request will be made within one week. Responses as to relevance of patient for EAP treatment may vary, based on the nature of the request and details of rationale for patient not being eligible for the currently open clinical trial.
Written requests from the patient’s healthcare provider should be submitted by email. All requests will remain confidential.

Disease/Category-Specific EA Policies/Criteria

Patients will be considered for EAP if they are not eligible for the currently open clinical trial of MNV-BM-BLD, carry a mitochondrial DNA deletion, and the mother is not a carrier of this deletion. Patient ineligibility for the open clinical trial may be due to their not meeting all inclusion/exclusion criteria (eg patient is below minimum age) or having a mitochondrial deletion syndrome other than Pearson Syndrome. 
Healthcare providers interested in information about the MNV-BM-BLD product for their patients can contact Minovia to request expanded access at expandedaccess@minovia.com

Single-Patient EA Policies/Criteria

Single-Patient Expanded Access Policies and Criteria - Mirati does not currently provide access to its investigational medicines outside of enrollment in clinical trials. Patients and Physicians seeking general information may contact Early Access Care by emailing Mirati.ExpandedAccess@earlyaccesscare.com or by contacting the Mirati Call Center 1-475-522-2200.

Single-Patient EA Policies/Criteria

Momenta is committed to developing safe and effective medicines to treat rare immune-mediated diseases with high unmet medical needs. Our goal is to ensure access to our investigational drug, at the appropriate time and in a clinically appropriate manner for patients.

Momenta’s Early/Expanded Access Policy (EAP) refers to the use of an investigational drug outside of a clinical trial. This is initiated when the primary purpose is to diagnose, prevent, or treat a serious condition in a patient, which is different from a clinical trial where more comprehensive safety and efficacy data are collected. At Momenta, we recognize and understand the need for an early/expanded access policy.

A number of factors consistent with the US Food and Drug Administration (FDA) and other regulatory agencies' guidelines should be taken into account when considering this kind of access.

Momenta’s Key Eligibility Requirements
To be eligible to access Momenta's investigational drugs under the EAP, participants must meet certain requirements, including, but not limited to:
- The illness must be serious or life-threating, with no other satisfactory treatment options (such as approved products or enrolling clinical trials)
- There is sufficient evidence that the potential benefit to the patient would likely outweigh the potential risks, based on all available safety and efficacy information
- Providing the investigational drug will not interfere with clinical trials that could support the investigational drug's development or marketing approval for the treatment indication

At this time, Momenta believes that participation in one of our clinical trials is the most appropriate way to access our investigational drugs. We do not currently provide our investigational drugs for use through expanded access.

Single-Patient EA Policies/Criteria

If you are a patient and interested in obtaining access to an investigational medicinal product, you should discuss available options with your treating physician. If a MorphoSys clinical trial may be a good treatment option for you, we recommend that your treating physician contacts us on your behalf via the contact information provided in ClinicalTrials.gov (search for Tafasitamab).

If you are not eligible for a clinical trial that is currently active, or if no appropriate clinical trials exist, please contact your treating physician to determine if an EAP may be the best or only treatment option for you. Your physician will find details on the EAP at ClinicalTrials.gov with the identifier NCT04300803.

Treating physicians should use the following contact (tafasitamab@clinigengroup.com) to receive the necessary request form, eligibility criteria and instructions for the EAP process. Receipt of requests will be acknowledged within two business days of receipt, approval or denial will usually be finalized within 2-3 business days. Before treatment can be started, the treating physician has to obtain IRB review.

MorphoSys is committed to a fair and impartial evaluation of each request for access to our EAP. Therefore, all decisions are based solely on clinical evidence and guided by the principles outlined below and by eligibility criteria for the EAP. In addition, MorphoSys intends to work in cooperation with local health authorities for the provision of an EAP.

Available Therapies via Single-Patient EA

Tafasitamab

Tafasitamab is used in combination with lenalidomide for the treatment of adult patients with relapse or refractory diffuse large B cell lymphoma (R/R DLBCL)

Single-Patient EA Policies/Criteria

NeoMatrix Therapeutics (the Company) is the developer of a novel investigational drug product, cP12. The investigational drug product is being evaluated for the treatment of serious burn injuries and is still in the early stages of clinical development.
At the current stage of clinical development, NeoMatrix Therapeutics has chosen not to offer Expanded Access (EA) and will not accept EA requests for the investigational drug product. The justification of the restriction on EA is as follows:
•    There is insufficient safety and efficacy data to determine the risk-benefit profile for the treatment of serious burn injuries.
•    The Company’s ability to conduct clinical trials is limited. Offering EA under any setting at this time will impact the Company’s ability to execute its trials.
•    The organization does not have the personnel and other resources to offer EA on a fair and equitable basis.
•    The Company’s investigational drug product cP12 requires special handling and shipping.
As the company is not currently accepting EA requests, the specified policy information is not provided at this time. The status of EA restriction will be re-evaluated if early data from future clinical trials suggest that cP12 investigational treatment can offer benefits for patients facing serious or life-threatening conditions.

NeoMatrix Therapeutics, Inc. Expanded Access Policy, Rev: May 17, 2019

Single-Patient EA Policies/Criteria

At Neuraptive, we are committed to developing first-in-class treatments for patients with peripheral nerve injuries (PNI) and physicians who treat them.

NTX-001 has been designed and is being developed as an adjunctive treatment for patients with nerve injuries and may restore and improve the recovery of neurons following nerve repair surgery. We will be conducting clinical studies to demonstrate the safety and efficacy of NTX-001, and to obtain regulatory approval and ultimately make NTX-001 available.

Before regulatory approval by the U.S. Food and Drug Administration (FDA) and foreign regulatory authorities, patients gain access to investigational treatments, such as NTX-001, by participating in clinical trials and by ‘Expanded access,’ also referred to as compassionate use, ‘Early Access,’ and ‘Emergency Use’. Patients who seek access to investigational medicines outside of an established clinical study and before health authority marketing authorization may wish to do so because standard treatments have failed, because they cannot tolerate already approved medicines, because they are unable to participate in a clinical study or because there are no comparable or satisfactory therapy options available outside of clinical trials. The primary purpose of expanded access is to use the investigational drug for patient treatment purposes rather than to gather data on safety, tolerability, and effectiveness.

Currently, Neuraptive does not offer an expanded access program and does not accept expanded access requests outside of clinical trials. We believe that access to NTX-001 should be limited to clinical trials until its safety, tolerability, and effectiveness for a particular indication has been determined and confirmed by regulatory authorities. Patient access to NTX-001 outside of a clinical trial may interfere with the conduct of our ongoing and future clinical trials, and may also disrupt the progress of our development program, which would, in turn, delay access to many patients in need.

Accordingly, while clinical trials are ongoing, we believe participation in our clinical trials is the only safe and appropriate way to access NTX-001. 

If you have questions about Neuraptive’s expanded access policy, please contact Neuraptive at info@Neuraptive.com. Please expect a response within five business days.

In line with the 21ST Century Cures Act, Neuraptive may revise this policy at any time.

Available Therapies via Single-Patient EA

Ruxolitinib Managed Access Program (MAP) for Patients Diagnosed With Severe/Very Severe COVID-19 Illness

Alpelisib – PIK3CA-Related Overgrowth Spectrum (PROS)
Asciminib – CML
Capmatinib – MET mutated non-small cell lung cancer (NSCLC) and MET amplified NSCLC
Clofazimine– Pulmonary mycobacterium abscessus infection
Dabrafenib/Trametinib - metastatic or unresectable melanoma, adjuvant (resected) melanoma, non-small cell lung cancer (NSCLC) - (liquid/powder formulation only)
Midostaurin – AML, advanced systemic mastocytosis (liquid/powder formulation only)
Pazopanib – Advanced renal cell carcinoma, advanced soft tissue sarcoma (liquid/powder formulation only)
Ribociclib – CDK4/6inhibitor - (liquid/powder formulation only)

Single-Patient EA Policies/Criteria

Single-Patient Expanded Access Policies and Criteria - If a treating physician believes that expanded access may be the only option for a patient, the physician should contact NS Pharma to make a formal request on behalf of a patient. Physicians may contact Early Access Care by emailing nspharma.expandedaccess@earlyaccesscare.com. We may request additional information to assist with patient eligibility for an investigational medication.

Available Therapies via Single-Patient EA

Viltolarsen Expanded Access for Duchenne Muscular Dystrophy - Confirmed DMD mutation in the dystrophin gene that is amenable to skipping of exon 53

Disease/Category-Specific EA Policies/Criteria

https://www.earlyaccesscare.com/companies/ns+pharma+inc./viltolarsen

Single-Patient EA Policies/Criteria

Pafolacianine sodium injection (OTL38) can be accessed through On Target’s Expanded Access program by contacting On Target Laboratories directly. The patient would be required to travel to an approved clinical site, where the investigator has completed the necessary training to administer the drug and has access to the required Near-Infrared Camera device. This would include any clinical investigator who have been approved to participate and enrolled subjects in either the Phase 2 or Phase 3 Lung trials with the drug. The patient may qualify to be enrolled in an open study, if available, and On Target would not include this subject in the randomization plan and their data would not be included in the efficacy analysis of the open study. In the event this is not possible, an Expanded Access Submission would be required to be submitted under the open IND 118215 as a Protocol Amendment. On Target Laboratories would work with the selected clinical investigator to generate the submission, which would include the required information defined in CFR 312.305, including the rational for the intended use of the drug, criteria for the patient selection, and the benefit risk of utilizing the investigational drug and device. The patient selection criteria would match the inclusion criteria defined in the Lung Cancer Phase 3 trial.

Available Therapies via Single-Patient EA

Pafolacianine sodium injection (OTL38) for use as an adjunct imaging agent in the detection of lung cancer during surgery.

Single-Patient EA Policies/Criteria

Oncoceutics will consider single - patient expanded access for patients who meet the criteria for the company's existing expanded access program, as outlined on clinicaltrials.gov

Available Therapies via Single-Patient EA

ONC201 - H3 K27M mutant gliomas

Available Therapies via Single-Patient EA

Melphalan Flufenamide (melflufen)

An Expanded Access Program (EAP) Protocol for Melphalan Flufenamide (melflufen) in Combination with Dexamethasone in Patients with Triple Class Refractory Multiple Myeloma

EA Policies/Criteria

To provide early treatment access and evaluate the safety of melflufen and dexamethasone in patients with triple class refractory multiple myeloma prior to commercial availability of the medication.

Single-Patient EA Policies/Criteria

Named Patient Access (Single Patient IND in the U.S.) – where a qualified healthcare professional requests an investigational medicine for use for a single patient because, in their judgement, currently available therapies are not satisfactory. Provision of the investigational medicine by Ondine may or may not require approval by FDA as well as an Institutional Review Board.

Available Therapies via Single-Patient EA

Non-antibiotic nasal decolonization. See https://www.ondinebio.com/solutions/steriwave/ . This is a light-based therapy that can decolonize the nose of bacteria, viruses and fungi within 4 minutes. Typically used for pre-operative patients as well as for vulnerable populations or immunocompromised patients.

Single-Patient EA Policies/Criteria

Orinove Inc. is committed to developing novel and selective medicines to patients with serious or life-threatening conditions. 
We are focused on enrolling and conducting the clinical trials necessary to gain regulatory approval to make our medicines available broadly to patients as quickly as possible. Orinove believes that participation in our clinical trial is the most appropriate way to access our investigational product therefore, at this time, Orinove is not making its investigational product available on expanded access.
We understand the need for expanded access programs, and we recognize the importance of having an appropriate policy. In line with the 21st Century Cures Act, Orinove may revise this policy at any time. 
If you have questions about our investigational product or expanded access, please contact wangweiai1@orinove.com. Orinove anticipates that it will acknowledge receipt of any expanded access questions or requests within two weeks of receipt. 
For active clinical trials with Orinove’s investigational agents, please search “Orinove” at www.clinicaltrials.gov.

Single-Patient EA Policies/Criteria

In general, Orphazyme will consider granting access to an investigational drug on a case-by-case basis if an Early Access Program is open in the country of residence of the patient and the following criteria are met:
• The patient has a serious or life-threatening medical condition within the disease area and the patient population that Orphazyme is investigating and planning to pursue approval by the national regulatory authorities.
• The patient has no access to a suitable approved treatment for the disease condition or no adequate treatments are available.
• The patient does not qualify to participate in any ongoing or planned clinical trials in a reasonably accessible geographical location.
• The patient has a disease for which there is sufficient clinical evidence of benefit from the use of the investigational drug, and the benefits likely outweigh the known or anticipated risks.
• Providing Early Access does not in any way disrupt the clinical development program and/or the regulatory pathway towards marketing authorization leading to broader access.
• There is an adequate and sustainable supply of the investigational drug, beyond what is required for all ongoing and planned clinical trials.
• The request for Early Access is received from a licensed physician with expertise in treating the disease and understands the potential risks and benefits of the investigational drug.
• The requesting physician agrees to comply with Orphazyme’s Early Access Program specifications as well as with the applicable laws and regulations governing the use of the investigational drug. Early Access
ends once the investigational drug is commercially available or reimbursed in a country. If Orphazyme decides to discontinue clinical development of the investigational drug for a specific disease area, Early Access will be discontinued.

Available Therapies via Single-Patient EA

Arimoclomol for Niemann-Pick type C (NPC)

Disease/Category-Specific EA Policies/Criteria

Early Access Policy

Single-Patient EA Policies/Criteria

We are unable to offer single patient expanded access at this time.

http://www.parion.com/pipeline/clinical-trials/

Single-Patient EA Policies/Criteria

www.pfizercares.com

Single-Patient EA Policies/Criteria

ACCESS TO PHARNEXT INVESTIGATIONAL MEDICINES PRIOR TO REGULATORY APPROVAL
(FOR US PATIENTS ONLY)
Pharnext develops innovative new medicines designed to address unmet medical needs of patients in major therapeutic areas. We are focused on creating new medicines that address important, real world concerns of patients and their families. We currently have investigational treatments in clinical trials for a number of different diseases, as described in the PRODUCTS section of our website at https://www.pharnext.com/en/products/r-d-pipeline
Pharnext’s goal is to efficiently bring safe and effective medicines to all appropriate patients by conducting rigorous clinical trials and obtaining marketing approval by regulatory authorities, including the U.S. Food and Drug Administration (FDA). Prior to such approval, participation in one of our clinical trials is the optimal way for patients to gain access to our investigational medicines. However, we understand that some patients may not be able to participate in a clinical trial and may seek access to investigational treatments before they are approved. These situations are often referred to in various ways, including expanded access, early access, pre-approval access, compassionate use and emergency use.
If early data from clinical trials suggest that an investigational treatment can offer benefits for patients facing serious or life-threatening conditions, Pharnext may consider requests to provide access outside of a clinical trial. Our top priority when evaluating requests for expanded access to investigational medicines is to ensure that we are not putting patients at risk of unnecessary harm.
Pharnext will consider an expanded access program, or a single request for expanded access of an investigational medicine, only if all of the following criteria are met:
• The patient’s licensed physician submits an unsolicited request for expanded access
• The patient to be treated has a serious or immediately life-threatening illness and there is no satisfactory alternative therapy
• There are no comparable or satisfactory alternative therapies approved and available to treat the disease or condition
• There is preliminary clinical evidence of effectiveness and acceptable safety of the investigational treatment for that patient’s condition
• The potential benefit of the investigational medicine to the patients is greater than the potential risk
• Staff and other required resources are available to provide the product and there is an adequate supply of the investigational medicine
• Providing the investigational medicine will not interfere with the timely completion of clinical trials that could lead to marketing approval and thereby could delay its availability to other patients
• Expanded access will only occur in a country where Pharnext expects to file for regulatory approval of the product
Patients interested in obtaining access to a Pharnext investigational therapy must do so via their physician. Physicians should contact Pharnext Medical Information Center at medical@pharnext.com. All requests will be carefully considered, and a written or oral response will be provided to the treating physician within two weeks of receiving a request. For further information on available expanded access programs, visit www.clinicaltrials.gov and search ‘expanded access programs’.

Available Therapies via Single-Patient EA

NA

Disease/Category-Specific EA Policies/Criteria

NA - see above

Single-Patient EA Policies/Criteria

There are seriously ill patients who will not be eligible for our clinical trials and may be in a high risk to experience an infection post a surgical intervention. In these circumstances, POLYPID will consider providing the requesting surgeon with pre-approved access to D-PLEX, for the prevention of infection of an individual patient outside of a clinical trial, when certain conditions are met. These conditions include the following:
• The patient is scheduled for an abdominal or a median sternotomy surgery for any reason;
• The patient is not eligible to participate in POLYPID clinical trials;
• The patient has a serious or life-threatening illness or condition and his/her likelihood to develop a serious infection due to surgical intervention is high;
• The patient has a disease or condition for which there is sufficient evidence of a projected benefit from the use of the D-PLEX and the benefit outweighs the known or anticipated risks;
• A benefit-risk analysis, based on both the available clinical data as well as the requesting surgeon’s assessment of the individual patient’s condition and history, supports making the investigational drug available;
• Making the investigational drug available will not negatively impact or delay the conduct of clinical trials or a regulatory review or an approval of the investigational drug for broader patient access; and
• Adequate supply of the investigational drug is available and can be supplied in the appropriate timeline of request.

Available Therapies via Single-Patient EA

D-PLEX, a new formulation of Doxycycline for local administration that is intended for prevention of Surgical Site Infections (SSIs) post cardiac surgery with median sternotomy and post abdominal surgery.

Disease/Category-Specific EA Policies/Criteria

https://www.polypid.com/wp-content/uploads/2020/08/PolyPid-Expanded-Access-Policy-FINAL.pdf

Single-Patient EA Policies/Criteria

At PRG S&T, we commit to bringing new, innovative therapies to patients by conducting rigorous clinical trials and obtaining marketing approval by the FDA and other regulatory authorities. At this point of time, PRG S&T believes that participation in one of our clinical trials is the most appropriate way to access our investigational medicine. However, we may also provide our investigational medicine for use through expanded access program.

Requests for expanded access must be made by a physician on behalf of their patient. The eligibility for a patient to participate in the expanded access program will be reviewed and evaluated based on the patient’s medical information. The request for access to a PRG S&T investigational medicine will only be considered if the patient's treating physician is committed to, and supportive of, the requested treatment.

Questions with regards to the expanded access to PRG S&T investigational medicine may be sent to us via the contact information listed on our website. Should the circumstance permits change to our expanded assess policy, updates will be posted to this website.

Available Therapies via Single-Patient EA

Progerinin for the treatment of Hutchinson-Gilford Progeria Syndrome (HGPS) and Werner Syndrome (WS)

Single-Patient EA Policies/Criteria

The following criteria are typically required before RemeGen would consider patients eligible for expanded access to a RemeGen investigational product. All criteria are subject to local laws and regulations.
• The patient suffers from a life threatening or serious disease or condition, where there is an unmet medical need that cannot be met by existing products.
• The patient is ineligible or unable to participate in any ongoing clinical study of the investigational product.
• The investigational product is under active development (a dose and regimen has been defined and the product is typically in Phase 2 development or beyond) and has not been placed on regulatory or any other hold.
• The benefit-risk profile of the drug in the indication and the patient population is deemed, to date, to be positive which typically occurs when RemeGen has decided to seek marketing approval in at least one major market globally.
• RemeGen has decided to seek marketing approval in at least one major market globally.
• The expanded access use of the investigational product will not jeopardize the development of the drug (for instance, granting access to an investigational unapproved drug through expanded access should not delay, interfere with, or compromise the completion of clinical trials that are intended to support approval by regulatory authorities).
• The use of the investigational product in an expanded access protocol is compliant with local rules and regulations.

Available Therapies via Single-Patient EA

Telitacicept for patients with moderate or severe SLE

Single-Patient EA Policies/Criteria

Renibus Therapeutics will evaluate and respond to a request that it receives on a case-by-case basis.

Available Therapies via Single-Patient EA

RBT-9 for the treatment of COVID-19 at the early stage (Stage I) of infection in patients who are at high risk for disease progression.

Available Therapies via Single-Patient EA

Procedure for Submitting Requests to Savara:
Savara will consider expanded access requests from treating physicians subject to US laws and regulations. All requests should be submitted via e-mail to EAP.US@savarapharma.com. 

Process for Review of Requests:
Savara is committed to a fair and impartial evaluation of each request for access to its investigational products. Therefore, all decisions are based solely on clinical circumstances and are guided by the criteria outlined below. Patients will be referred to ongoing clinical trials as the primary way to access investigational products. 

When evaluating requests for expanded access, Savara considers all of the following criteria: 
1. The patient for whom expanded access is requested suffers from a disease or condition that is serious or life-threatening. 
2. There are no comparable or satisfactory alternative therapies or clinical trials available. 
3. Sufficient preliminary efficacy and safety data exist to support an assessment that the benefit for the patient outweighs the potential risks and that the potential risks are not unreasonable in the context of the disease or condition being treated. 
4. Sufficient clinical data are available to identify an appropriate dose (amount and frequency) of the investigational drug. 
5. There is adequate drug supply to support the ongoing and necessary clinical trials as well as to support approved expanded access in a sustainable and equitable manner, until and if product becomes commercially available.
6. The patient is not eligible to participate in any ongoing clinical trials of the investigational drug. 
7. Expanded access will not adversely affect the clinical development program, in particular, the initiation, conduct, or completion of the clinical trials that are required for regulatory approval. 
8. The unsolicited request is made by a U.S. qualified and licensed physician who will take primary responsibility for supervising use of the investigational product from Savara and will comply with all applicable FDA regulatory requirements associated with treatment and use of an investigational product. 
9. All required regulatory and institutional approvals have been obtained. The patient must provide written informed consent. 

Requests for expanded access will be individually reviewed in accordance with these criteria. Savara is committed to evaluating all requests for expanded access in a fair and equitable manner. All requests will be evaluated by medical professionals and decisions will be based on available scientific evidence at the time of the request. 

Questions regarding Savara's ongoing U.S. expanded access program(s) can be forwarded to: EAP.US@savarapharma.com 

This policy is not applicable to countries outside the U.S. If you have questions on expanded access in a non-US country, please submit your request to info@savarapharma.com. 

Single-Patient EA Policies/Criteria

SCYNEXIS is committed to making Investigational Products available to seriously ill patients who have exhausted other treatment options.  A treating physician, who is able to comply with the SCYNEXIS requirements, may request information about how to apply for access to SCYNEXIS’ Investigational Products by contacting the company.

Available Therapies via Single-Patient EA

SCY-078 is a triterpenoid, glucan synthase inhibitor, antifungal agent undergoing investigation for:
•    The treatment of invasive candidiasis, including candidemia (IC). 
•    The treatment of invasive aspergillosis (IA), including IA that is refractory to or intolerant of other antifungal therapies.
•    The treatment of invasive candidiasis in patients who are refractory to or intolerant of other antifungal therapies.

Available Therapies via Single-Patient EA

At this time Silk Road Therapies, Inc./Ipekyolu Ilac Ltd. is testing a topical pentoxifylline formulation under US IND, but exclusively conducted in a trial in Istanbul, Turkey. At this time, we are unable to import our topical formulation from Turkey and do not have sufficient supply to offer expanded access to this drug. Therefore, until such time as the proof of concept trial is completed, Silk Road Therapies, Inc. is not offering expanded access beyond participation in the Istanbul trial.

However, we anticipate completing the proof of concept trial 3Q2019 and having reformulated material available in the US 1Q2020. At that time, we will revise this expanded access policy and may permit access to our product for patients who are ineligible for entrance into our later phase III pivotal trial.

Single-Patient EA Policies/Criteria

Expanded access is used to describe treatment with an investigational product for a patient with a serious or life-threatening illness or condition when there are no comparable or satisfactory alternative treatments available and the patient is not eligible for a clinical trial. These investigational products are not approved by the U.S. Food and Drug Administration (FDA). The expanded access pathway is often used by commercial pharmaceutical companies to provide their products before they are approved.

St. Jude Children’s Research Hospital (St. Jude) is dedicated to its mission of advancing cures for pediatric catastrophic diseases through research and treatment. St. Jude provides the best possible care for our patients. We also make discoveries that will lead to better treatment for children and young adults around the world. We accomplish this by offering patients participation in clinical trials such as the LVXSCID-ND study (NCT01512888). 

St. Jude Children’s Research Hospital is not a commercial pharmaceutical company and does not have the capability to manufacture products at an industrial level. Supplies of the investigational product for the LVXSCID-ND study are limited and earmarked for the completion of a prospective research guided clinical trial. At this time, the safety and efficacy of the product is under investigation. St. Jude is not able to provide the investigational product in the LVXSCID-ND study for expanded access. If your child has been diagnosed with X-Linked Severe Combined Immunodeficiency (SCID-X1), please contact the physician below to discuss possible treatment options.

Disease/Category-Specific EA Policies/Criteria

https://www.stjude.org/lvxscid-nd

Disease/Category-Specific EA Policies/Criteria

EXPANDED ACCESS TO INVESTIGATIONAL DRUGS

https://www.takedaoncology.com/medicines/expanded-access-to-investigational-drugs/

Single-Patient EA Policies/Criteria

TG Therapeutics is committed to bringing our investigational therapies for B-cell malignancies and autoimmune diseases to patients as fast as possible. We believe the best way for patients to access our investigational therapies is through participation in clinical trials, with the goal of obtaining regulatory approval of our products and allowing access to the broadest group of patients possible.

At this time, TG Therapeutics does not have a program to provide its investigational therapies to patients on an expanded access basis pursuant to section 561(b) of the U.S. Federal Food, Drug, and Cosmetic Act (FDCA). Because we do not currently have an expanded access program, we have not yet established procedures for making such requests or criteria for evaluating them. TG Therapeutics will update this policy if the company decides to make its investigational therapies available on an expanded access basis.

Patients may be able to access TG Therapeutics investigational therapies by participating in ongoing clinical trials. Whether or not a patient is eligible to enroll in any of our trials depends on a number of factors, including the specific requirements of each trial and the status of that trial. For more information about our trials, please visit Our Trials Overview or https://clinicaltrials.gov/ct2/results?cond=&term=%22tg+therapeutics%22&cntry=&state=&city=&dist.

TG is committed to responding to questions about its expanded access policy within five (5) business days of receipt. As noted above, at this time, we are not providing our investigational therapies on an expanded access basis.

Available Therapies via Single-Patient EA

G207 for pediatric high grade gliomas

Single-Patient EA Policies/Criteria

Triumvira Immunologics, Inc. is dedicated to developing new therapies that have a positive impact on patient health, and to serving patients, patient families and patient communities through education, empathy, and awareness. Consistent with Triumvira, Inc.’s mission to bring innovative medicines to patients with serious or life-threatening illnesses or conditions, we are focused on enrolling and conducting the clinical trials necessary to gain regulatory approvals to make our medicines available broadly to patients as quickly as possible. We are privileged to collaborate with clinical investigators and with patients who participate in our studies to develop new, safe and effective therapies. We believe this approach will serve patients who could be helped by the therapies we are developing.

At the same time, we understand that there are seriously ill patients who will not be eligible for our clinical trials and may not have options for alternative therapies, including investigational therapies in trials being conducted by other sponsors. In these circumstances, Triumvira, Inc. will consider providing a requesting physician with pre-approval access to a specific investigational drug, for the treatment of an individual patient outside of a clinical trial, when certain conditions are met. These conditions include the following:

• The patient has a serious or life-threatening illness or condition and is either no longer responsive to or no longer able to tolerate any available treatment option;
• The investigational drug is in active clinical development with sufficient data available to determine an appropriate dose and schedule for the patient’s specific condition;
• A benefit-risk analysis, based on both the available clinical data as well as the requesting physician’s assessment of the individual patient’s condition and history, supports making the investigational drug available;
• Making the investigational drug available will not negatively impact or delay the conduct of clinical trials or regulatory review or approval of the investigational drug for broader patient access; and
• Adequate supply of the investigational drug is available.

We continually evaluate the benefit-risk profile of each of our investigational drugs based on evolving clinical data. Each compound under development is different and the fact that one investigational drug is made available for the treatment of a particular patient does not mean it will be made available in response to other requests on behalf of other patients whose circumstances and medical histories may be different, or that a different investigational drug will be made available under our policy. Requests will be considered on a case-by-case basis.

Triumvira is committed to evaluating all requests in a fair and equitable manner. All requests must be submitted by the patient’s treating physician; Triumvira may require more detailed information in order to fully evaluate a request. The requesting physician must agree to obtain appropriate regulatory and ethics committee approvals and comply with regulatory obligations, including obtaining patient consent, patient monitoring and safety reporting. Each request will be given careful consideration by Triumvira whose decisions are final. Physicians seeking pre-approval access for patients with no alternative treatment options should submit their requests to Partners@Triumvira.com. We regularly monitor this mailbox and will use our best efforts to acknowledge each submitted request within 3 business days after receipt.

Available Therapies via Single-Patient EA

TAC01-CD19, autologous TAC (T cell antigen coupler) T cells, single infusion, multiple dosage levels.

Indication: B-Cell Lymphoma

Single-Patient EA Policies/Criteria

TVAX Biomedical, Inc. (TVAX) is committed to developing immunotherapies for patients with serious cancers. Our goal is to provide access to our investigational therapies primarily through clinical trials. At this time, TVAX does not have any investigational immunotherapy product available for expanded access.

“Expanded Access” refers to the use of an investigational therapy outside of a clinical trial for potential treatment of a serious condition in a patient. The US Food and Drug Administration has set forth guidelines when considering expanded access.

TVAX is not currently making its investigational immunotherapy product candidates available on an expanded access basis anywhere in the world. Requests for expanded access to TVAX Biomedical’s investigational immunotherapy product candidates must come from the patient’s treating physician because only physicians can enroll patients in a clinical trial or the expanded access program.

Certain therapies, like TVAX Immunotherapy, are made through complex manufacturing processes. TVAX seeks to retain the ability to make and supply product in a fair and equitable manner and in a volume that assures adequate manufacturing capacity for clinical trials and development programs. TVAX believes that participation in one of its’ clinical trials is the most appropriate way to access these investigational therapies.

At this time, TVAX is not currently making its unapproved therapies available on an expanded access or right to try basis. In the event TVAX decides to consider expanded access or right to try use, TVAX will evaluate and respond to each request that it receives on a case-by-case basis.

If you have questions, please speak with your physician or contact TVAX at info@tvaxbiomedical.com.

Consistent with the 21st Century Cures Act, TVAX may revise this policy at any time.

Single-Patient EA Policies/Criteria

Requests for early access to investigational therapies must be made by a qualified physician.

Single-Patient EA Policies/Criteria

Licensed physicians should submit expanded access requests to Usona at EA@usonainstitute.org using the Usona Expanded Access Request Form. Requests for Expanded Access will be acknowledged within 5 business days of receipt. All Expanded Access use requests will be decided on a case-by-case basis at the sole discretion of Usona. All requests received will be reviewed anonymously by an internal Usona Committee to ensure patient safety and equality. The licensed treating physician must obtain, where applicable, all Ethics Review Board (ERB) or Institutional Review Board (IRB) approvals along with approval from the overseeing regulatory authority (e.g. FDA, EMA, Health Canada, etc.) prior to submission.

Expanded Access requests to Usona must meet ALL of the following criteria in order to be considered:
• The disease or condition for which use is requested is serious or life-threatening;
• The patient is ineligible or not able to participate in a clinical trial for the requested use and all approved treatment options have been exhausted without success and no satisfactory alternative treatment is available as determined by the requesting licensed physician;
• The requesting physician is a licensed physician and is authorized to deliver treatment as outlined in the request;
• There is sufficient clinical evidence to inform the safe use of the investigational drug under the requested use (at the requested dose and frequency of treatment);
• There is sufficient clinical evidence to suggest the requested use is expected to provide a potential clinical benefit to the patient (at the requested dose and frequency of treatment);
• The requested use would not negatively impact or interfere with active clinical trials or drug development programs of the applicable investigational drug;
• The treating physician has received approval by their overseeing regulatory authority (e.g. FDA, EMA, etc.) and where applicable, any Ethics Review Board (ERB) or Institutional Review Board (IRB) approvals
• The requested use complies with all applicable laws, rules and regulations;
• The requested use complies with Usona’s policies, including strict adherence to the ‘Usona Expanded Access Set and Setting Manual’;
• There is an adequate supply of the requested investigational drug available for the requested use.

Please note that Usona is only able to respond to Expanded Access requests from licensed physicians. Additional requirements apply to Expanded Access use of controlled substances, including specific personnel and facility requirements for expanded access use of drugs of the psychedelic drug class.

Available Therapies via Single-Patient EA

Psilocybin capsules (25 mg)

Disease/Category-Specific EA Policies/Criteria

https://www.usonainstitute.org/expandedaccess/

Single-Patient EA Policies/Criteria

VelosBio will review all requests for investigational drug access on a case-by-case basis, taking into consideration regulations and guidance issued by applicable health authorities. Among other criteria, VelosBio will consider whether:

1. The patient has a serious, debilitating, or life-threatening medical condition for which no other medically appropriate treatment is available;
2. The patient’s healthcare provider can provide an assessment of the benefits and risks of the proposed expanded access use that suggests that the potential benefits of access to the investigational therapy likely outweigh known potential risks in the context of the risks posed by the patient’s underlying medical conditions;
3. The patient’s healthcare provider can provide relevant data (eg, medical records, etc.) to support the assessment of benefits and risks, considering standards of patient confidentiality appropriate for the situation;
4. The patient’s healthcare provider is currently licensed with the appropriate practitioner licensing board;
5. The patient’s healthcare provider is willing and able to support any regulatory procedures required by the relevant regulatory authority;
6. The patient’s healthcare provider is willing to enter into an expanded access agreement with VelosBio;
7. The patient’s healthcare provider will obtain informed consent from the patient or his/her legal guardian(s) for use of the investigational therapy;
8. The patient’s healthcare provider will supervise administration of the investigational therapy in accordance with any procedures specified by VelosBio (which in some countries may include a protocol for use of the investigational therapy);
9. The patient’s healthcare provider is willing and able to report any side effects of the investigational therapy and any other results as requested by VelosBio to assess benefits and risks of the expanded access use;
10. The patient does not qualify to participate in any ongoing clinical trial sponsored by VelosBio in an accessible geographical location;
11. Sufficient data exist to support the proposed expanded access use and the assessment of the benefits and risks of the expanded access use;
12. Access would not impede or compromise the ongoing clinical development program or regulatory approval of the investigational therapy; and
13. There is adequate supply of the investigational therapy to support access and such supply will not negatively affect a clinical trial or anticipated product launch

Requests for access to a VelosBio investigational therapy must be made by the patient’s treating physician. Whenever possible, the patient, the patient’s parent/legal guardian, or patient caregiver should work through the treating physician rather than contacting VelosBio directly.

To initiate a request for access to a VelosBio investigational drug, the physician should email info@velosbio.com. Such requests should aim to address Criteria 1-10 above. VelosBio will strive to respond to requests for access of an investigational product within five (5) business days of receiving a complete request that includes all necessary documentation.

Single-Patient EA Policies/Criteria

Venatorx Pharmaceuticals (the “Company”) is focused on conducting clinical studies and obtaining regulatory approval of investigational medicines in support of its mission to develop safe and effective treatments for infectious diseases.  The best way for patients to get access to investigational medicines is by taking part in clinical trials.  Please search ClinicalTrials.gov for publicly available information related to Venatorx’s ongoing clinical trials. 

It is recognized that not all patients are eligible to enroll in clinical trials. However, at this time, Venatorx is not able to offer Expanded Access (“EA”) and will not accept EA requests. 

Venatorx recognizes the need for EA programs.  The Company is currently establishing the necessary processes and procedures, as well as putting in place resources and supplies so that an EA program may be established in the future.

If you have questions about Venatorx’s EA policy, please contact medinfo@venatorx.com.

Available Therapies via Single-Patient EA

Cefepime/VNRX-5133 (taniborbactam)

Single-Patient EA Policies/Criteria

At Viela Bio, we work every day to improve the lives of people impacted by serious, underserved, inflammatory and autoimmune diseases. We share the urgency of patients seeking new treatments for potentially life-altering diseases, and understand the interest in accessing our therapies outside of clinical trials and prior to regulatory approval.

Expanded access, also referred to as compassionate use, is a channel through which the US Food and Drug Administration (FDA) allows physicians to request investigational medicines for patients. We do not currently offer any expanded access programs, as we are focusing our resources – including supply of complex investigational medicines – on clinical trials and regulatory approval.

We encourage patients who are interested in accessing therapies in our pipeline to talk to their doctor about participating in a clinical trial. Information about all of our trials, including eligibility criteria and locations, is available at ClinicalTrials.gov.

If we are able to offer expanded access in the future, we will update this policy. If you have further questions, please email eap@vielabio.com.

Disease/Category-Specific EA Policies/Criteria

https://enroll.xcures.com/static/ManagedAccessPolicy.pdf

Single-Patient EA Policies/Criteria

Xynomic Pharmaceuticas, Inc. (Xynomic) is dedicated to developing new therapies that have a positive impact on patient health, and to serving patients, patient families and patient communities through education, empathy, and awareness.
Xynomic development resources are focused on conducting clinical studies required by regulatory authorities to fully answer important scientific questions about the potential risks and benefits of our investigational products, and to obtain regulatory approval.
Xynomic is committed to making investigational products available to seriously ill patients who have exhausted other treatment options. A treating physician, who is able to comply with the requirements that are stated in this document, may request information about how to apply for access to Xynomic investigational products by contacting the Company.
Any use of a Xynomic investigational product outside a clinical study in a country must be in accordance with local laws and regulations governing such programs, including Xynomic policies and procedures.

Available Therapies via Single-Patient EA

Abexinostat tablets for treatment of advanced renal cell carcinoma in combination with pazopanib. Xynomic will only provide abexinostat tablets.