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.

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)

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

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

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

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).

Disease/Category-Specific EA Policies/Criteria

biomarck pharmaceuticals intends to support children with difficult to treat cancers such as Rhabdomyosarcoma and Neuroblastoma. This may be done by a compassionate use program under the Investigator IND of the treating hospital. This effort will be done in support of the concepts of non-profit organizations such as the Miami based Live Like Bella Foundation (Learn More) which supports children with cancer, their families and research into childhood cancer.

BIO-11006 is an investigational peptide medication delivered by nebulizer which has shown activity in the laboratory and in animal studies in certain types of cancer. It has been granted FDA acceptance to begin studies in adult lung cancer. BIO-11006 has been given to over 200 patients with COPD (chronic bronchitis and emphysema) and been shown to have a good safety profile at the recommended dose. It has not been given to children or cancer patients in controlled clinical studies.

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

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)

Disease/Category-Specific EA Policies/Criteria

Capricor Initiates Compassionate Use Program for Severe COVID-19 Patients using CAP-1002, its Novel Cell Therapy

CAP-1002 in Severe COVID-19 Disease - Compassionate Use Program

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. 

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

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

Available Therapies via Single-Patient EA

A Compassionate Use Study of Leronlimab in Breast Cancer

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

Available Therapies via Single-Patient EA

• Lonafarnib
• Pegylated Interferon Lambda
• Avexitide

Disease/Category-Specific EA Policies/Criteria

The Hutchinson-Gilford Progeria Syndrome and Progeroid Laminopathy Managed Access Program

Eiger BioPharmaceuticals, the distributor of the drug lonafarnib, is sponsoring a Managed Access Program (MAP) for eligible patients with Hutchinson-Gilford Progeria Syndrome (HGPS or Progeria) or a Progeroid Laminopathy (PL).  The protocol governing this program can be viewed at ClinicalTrials.gov. The MAP study number is NCT03895528.  Clinigen is the administrator of the lonafarnib MAP for HGPS and PL.

Patients or family members desiring more information about this program should speak to their doctors  or contact The Progeria Research Foundation at 978-535-2594 or e-mail info@progeriaresearch.org.  Patients and family members should not contact Clinigen.  Clinigen is only authorized to speak with doctors.

For treating doctors with questions about the MAP, please contact Clinigen’s Medicine Access Team at +44 (0) 1932 824123 or email medicineaccess@clinigengroup.com.  The doctor may also e-mail Eiger at ProgeriaMA@eigerbio.com or Eigeraccess@eigerbio.com.

COVID-19

Eiger is supplying Peginterferon Lambda (Lambda) and is assisting in the protocol design of multiple Investigator-Sponsored Studies that are being initiated to evaluate the safety and efficacy of Lambda in patients diagnosed with COVID-19.  These studies will assess a 180 mcg, once-weekly, subcutaneous dose of Lambda, and will enroll at academic centers across the U.S., as well as international sites.

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

Disease/Category-Specific EA Policies/Criteria

Emergency Access to Remdesivir Outside of Clinical Trials

Expanded Access Treatment Protocol: Remdesivir (RDV; GS-5734) for the Treatment of SARS-CoV2 (CoV) Infection

Expanded Access Remdesivir (RDV; GS-5734™) - for DOD/military personnel only

Remdesivir Clinical Trials

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

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

Any request for access to Immunomedics’ investigational drug, sacituzumab govitecan must be sponsored by a duly licensed U.S. healthcare professional. One of the qualifying criteria is that a patient must have metastatic triple-negative breast cancer (mTNBC) who has received at least two (2) prior therapies for metastatic disease.

Available Therapies via Single-Patient EA

sacituzumab govitecan

Single-Patient EA Policies/Criteria

Questions or inquiries regarding the availability of ruxolitinib for compassionate use or independent research should be made to:

U.S. Medical Information
1-855-4MED-INFO (1-855-463-3463)
medinfo@incyte.com.

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.

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.

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.

Available Therapies via Single-Patient EA

fusidic acid (CEM-102)

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/

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.

Disease/Category-Specific EA Policies/Criteria

https://www.earlyaccesscare.com/images/Mirati%20Expanded%20Access%20Policy.pdf

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

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

Disease/Category-Specific EA Policies/Criteria

Developing Therapeutics and Vaccines for Coronaviruses

https://www.niaid.nih.gov/diseases-conditions/coronaviruses-therapeutics-vaccines

NIAID has also supported the clinical testing of two promising antibody-based therapeutics, which prevent the virus from infecting and entering cells.  NIAID conducted a phase 1 clinical trial of  SAB-301, an experimental MERS treatment developed from cattle that make human antibodies.  This was shown to be safe and well tolerated in healthy adults. More recently, NIAID supported a Phase I clinical trial of a combination of two monoclonal antibodies, REGN3048 and REGN 3051, and demonstrated this combination was also safe and well tolerated.  Planning for follow on Phase 2/3 efficacy studies using SAB-301 is currently ongoing with partners where MERS is endemic, including the Kingdom of Saudi Arabia.

NIH clinical trial of investigational vaccine for COVID-19 begins

https://www.nih.gov/news-events/news-releases/nih-clinical-trial-investigational-vaccine-covid-19-begins

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

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

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

www.pfizercares.com

Available Therapies via Single-Patient EA

There is an Expanded Access Program (EAP) for lurbinectedin to treat patients in the United States (U.S.) with relapsed Small Cell Lung Cancer (SCLC), who are unable to enter clinical trials and there are no appropriate alternative treatments.

Healthcare professionals wishing to request access to lurbinectedin under the EAP or who would like to find out more should do so by emailing Lurbinectedin.EAP@Bionical-Emas.com.

See also A Multicenter Expanded Access Treatment Protocol of Lurbinectedin in Previously Treated SCLC in the USA

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

Available Therapies via Single-Patient EA

COVID-19 - Yeliva (opaganib)

Expanded Access for the Treatment of Advanced Cholangiocarcinoma With ABC294640 (Yeliva ®)

Single-Patient EA Policies/Criteria

https://www.roche.com/dam/jcr:035f3847-505e-484c-b5f6-f666790791de/24_Position%20on%20Pre%20Approval%20Access%20on%20Investigational%20Medicinal%20Products_reviewed_March%202019.pdf

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

On the 18^th March Synairgen announced its plans to initiate a pilot clinical trial of SNG001 in COVID-19 patients. The trial will provide data on the efficacy of inhaled interferon beta in the treatment of ambulatory and hospitalised patients infected with SARS-CoV-2. Since the announcement, we have received a number of requests for SNG001 supplies to treat individuals outside the trial. As SNG001 is an experimental drug, untested as to safety and efficacy in this patient group, we regret that we are unable to fulfil any such request at this time. Synairgen’s team is working very hard to establish these measures in a well-controlled environment as quickly as possible. https://www.synairgen.com/

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.

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

http://www.ucb.com/_up/ucb_com_patients/documents/UCB_Policy%20Access%20Investigational%20Drugs%20u…

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

Venatorx 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. Venatorx will consider requests so that patients 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. Use of an investigational product outside the scope of a clinical study is often referred to as expanded access (sometimes called compassionate use).
Expanded access is allowed in some countries under local laws and regulations and must be conducted in compliance with applicable requirements. The requirements are different across countries and the type of use that is allowed (i.e., for individual patients or groups of patients) varies.
Venatorx will consider making available, for expanded access use for individual patients, an investigational medicine in response to an unsolicited written request from a licensed physician who is in a treatment relationship with the patient/s. Expanded access requests must be sent to medinfo@venatorx.com.
Venatorx considers the following factors when reviewing expanded access requests:
1. The investigational medicine is being studied in humans under an appropriate regulatory authorization or all investigational studies of the product have been completed.
2. The patient is suffering from a serious disease or condition, or whose life is immediately threatened by their disease or condition.
3. Whether potential alternative treatment options are available or have been used previously with the patient.
4. Patient ineligibility for participation in any ongoing clinical study of the investigational product.
5. An assessment of the potential patient benefit justifies the potential risks of treatment.
6. Available clinical information on the drug’s use.
7. Meets any other relevant medical criteria for expanded access of the investigational product, as established by Venatorx.
8. Providing the investigational medicine for expanded access or compassionate use will not interfere with development of the product.
9. There are enough supplies of the investigational medicine to support the request as well as the ongoing and planned clinical trials.
10. The physician attending to the patient/s for whom expanded access is sought agrees to comply with all applicable legal and regulatory requirements in relation to the request and any requirements in terms of medical criteria (e.g., clinical monitoring of the drug use), safety reporting or other data provision which Venatorx may require.
Certain expanded access and treatment studies, which involve the collection of safety and efficacy data, must be registered on ClinicalTrials.gov or other clinical trial listing website/s. The results of those studies must be made publicly available in accordance with the Venatorx standards on public disclosure.

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.

Single-Patient EA Policies/Criteria

Vitaeris’ objective is to provide access to clazakizumab at the appropriate time and in the correct manner for patients.
At this time, participation in a controlled clinical trial is the most appropriate way to access the use of clazakizumab. Clinical trials are controlled research studies in humans designed to determine if an investigational drug is safe and effective. Since the benefit:risk profile of clazakizumab has not been fully established, Vitaeris is not supporting expanded access to clazakizumab outside of clinical trials. Vitaeris is maintaining a focus on conducting well-controlled clinical trials that can support global health authority approvals.
Vitaeris is currently conducting a pivotal trial investigating the efficacy and safety of clazakizumab for the treatment of chronic active antibody-mediated rejection (CABMR) in kidney transplant subjects.

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.