Company Directory
Use this Company Directory to identify investigational drugs, link to clinical trials, and search for expanded access policies, company contact information, and program listings.
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If you know the name of a drug manufacturer, you can easily search using the alphabetical menu below. Or you can search in free text – simply type the disease, drug, company name, or other detail into the Search bar below and click on the magnifying glass. For broadest results, type the terms without quotation marks; to narrow your search to an exact match, put your terms in quotation marks (i.e.; “breast cancer”). See Search Tips for tips and definitions to make your searches more effective.
The web links in this directory are public information provided to the Foundation and the acknowledgement times listed are the number of days or hours for a company to acknowledge receipt of request, not to make a decision. Data contained in this directory is provided either directly by the company or by ClinicalTrials.gov for the companies listed in this directory.
The Expanded Access listings provided on this website are limited to those companies currently in the Company Directory and include listings for expanded access which are Available, Temporarily Not Available and Approved for Marketing, which in some cases includes treatments which have not yet reached the market. In addition, many companies are willing to consider expanded access requests even if they do not show active Expanded Access Listings in this directory. Contact the company to explore the possibility of individual access to an investigational therapy.
To learn more about clinical trials and expanded access, please visit our patient and physician guides on this website.
Abbvie
2 business daysExpanded Access Listings
Conditions:
Relapsed or Refractory Mantle Cell Lymphoma
Conditions:
Multiple Myeloma
Conditions:
Primary Tauopathy, Corticobasal Degeneration Syndrome (CBD)
Conditions:
Glioblastoma or Solid Tumors, Epidermal Growth Factor Receptor (EGFR) Diagnosis
Conditions:
Hepatitis C Virus Infection
Conditions:
Myelofibrosis, Acute Lymphocytic Leukemia (ALL), Lymphoblastic Lymphoma, Thyroid Cancer
Conditions:
Crohn's Disease
Conditions:
Small Cell Lung Cancer
Conditions:
Atopic Dermatitis (AD)
Conditions:
Solid Tumors With Documented BRCA, BARD, or PALB or Other Acceptable DNA Mutations or Anomalies That Are Scientifically Sound, Triple Negative Breast Cancer (TNBC), High Grade Serous Ovarian Cancer
Conditions:
Chronic Lymphocytic Leukemia (CLL), Multiple Myeloma, Acute Myeloid Leukemia (AML), Non-Hodgkin's Lymphoma, Acute Lymphoblastic Leukemia (ALL), Amyloidosis
Conditions:
Metastatic Breast Cancer With BRCA 1 or BRCA 2 Genetic Mutation, Triple-Negative Breast Cancer
Achillion Pharmaceuticals
N/AAdditional Information
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.
Alexion
3 business daysExpanded Access Listings
Conditions:
Hypophosphatasia
Alkermes
2 weeksAdditional Information
SINGLE-PATIENT EA POLICIES/CRITERIA
http://www.alkermes.com/assets/content/files/Expanded%20Access%20Policy.pdf
AlloVir, Inc.
5 business daysAdditional Information
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.
Alnylam Pharmaceuticals
3 business daysExpanded Access Listings
Conditions:
Acute Hepatic Porphyria
Conditions:
TTR-mediated Amyloidosis, Amyloidosis, Hereditary, Amyloid Neuropathies, Familial, Familial Amyloid Polyneuropathies, Amyloid Neuropathies, Amyloidosis, Hereditary, Transthyretin-Related
Conditions:
Primary Hyperoxaluria
Additional Information
DISEASE/CATEGORY-SPECIFIC EA POLICIES/CRITERIA
http://www.alnylam.com/medical-professionals/expanded-access-program/
Amgen
3 business daysExpanded Access Listings
Conditions:
Celiac Disease
AMICUS THERAPEUTICS
3 business daysExpanded Access Listings
Conditions:
Pompe Disease
Physician Initiated Expanded Access Request for Migalastat in Individual Patients With Fabry Disease
Conditions:
Fabry Disease
Additional Information
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.
Amryt Pharmaceuticals
N/AAdditional Information
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
Arrowhead Pharmaceuticals, Inc.
5 business daysAdditional Information
Available therapies via single-patient EA
ARO-AAT Injection
For the treatment of alpha-1 antitrypsin deficiency associated liver disease.
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
DISEASE/CATEGORY-SPECIFIC EA POLICIES/CRITERIA
https://arrowheadpharma.com/wp-content/uploads/2019/03/Compassionate-Use...
Astellas Pharma US
7 daysExpanded Access Listings
Conditions:
Locally Advanced or Metastatic Urothelial Carcinoma (UC)
Conditions:
Acute Myeloid Leukemia (AML), FMS-like Tyrosine Kinase-3 (FLT3) Mutations
AstraZeneca Pharmaceuticals LP
Phone Number & Email
Expanded Access Listings
Conditions:
Relapsed/Refractory Hairy Cell Leukemia
Conditions:
Non Small Cell Lung Cancer, Cancer of the Head and Neck
Conditions:
NF type1 With Inoperable Plexiform Neurofibromas
Conditions:
Non Small Cell Lung Cancer (NSCLC)
Atossa Genetics
1-5 daysAdditional Information
Available therapies via single-patient EA
Oral endoxifen (for pre-surgical and adjuvant treatment of Luminal A ER+ breast cancer; mammographic breast density)
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.
DISEASE/CATEGORY-SPECIFIC EA POLICIES/CRITERIA
https://www.atossagenetics.com/wp-content/uploads/2019/07/AG-Expanded-Ac...
Atox Bio
2 business daysAdditional Information
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
Bayer
Two working daysExpanded Access Listings
Conditions:
Tumors Harboring NTRK Fusion
Conditions:
Solid Tumors Harboring NTRK Fusion
BerGenBio ASA
N/AAdditional Information
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.
Biogen
1 business dayExpanded Access Listings
Conditions:
Infantile-onset Spinal Muscular Atrophy
Biohaven Pharmaceuticals
2 business daysExpanded Access Listings
Conditions:
Migraine
Conditions:
Amyotrophic Lateral Sclerosis, ALS, Lou Gehrig Disease, Lou Gehrig's Disease, Lou-Gehrigs Disease, Motor Neuron Disease, Amyotrophic Lateral Sclerosis
Additional Information
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).
Biothera Pharmaceuticals, Inc.
7 business daysAdditional Information
Available therapies via single-patient EA
Imprime PGG
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.
BioXcel Therapeutics
5 Business DaysBlueprint Medicines Corporation
5 business daysExpanded Access Listings
Conditions:
GIST
Additional Information
Available therapies via single-patient EA
Investigational medicine avapritinib (formerly known as BLU-285)
SINGLE-PATIENT EA POLICIES/CRITERIA
Please see our Compassionate Use Policy on our website
DISEASE/CATEGORY-SPECIFIC EA POLICIES/CRITERIA
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
Boehringer Ingelheim Pharmaceuticals
5 business daysExpanded Access Listings
Conditions:
Lung Diseases, Interstitial
Conditions:
Carcinoma, Non-Small-Cell Lung
Bristol-Myers Squibb
48 hoursExpanded Access Listings
Conditions:
Multiple Myeloma
Conditions:
Multiple Myeloma
Conditions:
Pediatric Cancer
Celgene
24 hoursExpanded Access Listings
Conditions:
Myelodysplastic Syndrome
Conditions:
Myelodysplastic Syndrome (MDS)
Conditions:
Myelofibrosis
Conditions:
Myelofibrosis
Conditions:
Multiple Myeloma
Celldex Therapeutics
5 business daysAdditional Information
Available therapies via single-patient EA
None
DISEASE/CATEGORY-SPECIFIC EA POLICIES/CRITERIA
https://celldex.com/docs/Compassionate_Use_Policy_MAY2018.pdf
Cellectar Biosciences
5 business daysAdditional Information
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
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.
Center for Cancer Research, National Cancer Institute, National Institutes of Health
5 business daysAdditional Information
Available therapies via single-patient EA
https://clinicalstudies.info.nih.gov/ProtocolDetails.aspx?A_2015-C-0029....
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).
Daiichi Sankyo
5 business daysExpanded Access Listings
Conditions:
Acute Myeloid Leukemia With Gene Mutations
Additional Information
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.
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.
DISEASE/CATEGORY-SPECIFIC EA POLICIES/CRITERIA
Eisai
2 business daysExpanded Access Listings
An Extended Access Program (EAP) for Participants Who Have Completed Rufinamide Study E2080-G000-303
Conditions:
Lennox Gastaut Syndrome
Conditions:
Primary Generalized Tonic-Clonic or Partial Onset Seizures
Conditions:
Partial Onset Seizures
Expanded Access Program With Lenvatinib for the Treatment of Differentiated Thyroid Cancer in Brazil
Conditions:
Thyroid Cancer
Eli Lilly
5 business daysExpanded Access Listings
Conditions:
Melanoma, Prostate Cancer, Ovarian Cancer, Renal Cell Carcinoma, Colorectal Carcinoma, Pancreatic Carcinoma, Non-small Cell Lung Carcinoma, Solid Tumors, Breast Cancer
Conditions:
Melanoma, Prostate Cancer, Ovarian Cancer, Renal Cell Carcinoma, Colorectal Carcinoma, Pancreatic Carcinoma, Non-small Cell Lung Carcinoma, Solid Tumors, Breast Cancer
Conditions:
Non Small Cell Lung Cancer, Medullary Thyroid Cancer, Colon Cancer, Breast Cancer, Pancreatic Cancer, Papillary Thyroid Cancer, Other Solid Tumors With Evidence of Activating RET Alteration
Conditions:
Cancer
Conditions:
Metastatic Breast Cancer
Conditions:
Soft Tissue Sarcoma
EMD Serono
5 business daysExpanded Access Listings
Conditions:
Metastatic Merkel Cell Carcinoma
Entasis Therapeutics
3 business daysFrontier Biotechnologies Inc.
10 business daysAdditional Information
SINGLE-PATIENT EA POLICIES/CRITERIA
The IND has been deemed safe-to-proceed and the fast frack designation has been granted. The registration in ClinicalTrials.gov will be done before the trial starts and a link will be provided then.
Genentech
3 business daysExpanded Access Listings
Conditions:
Hemophilia A
Additional Information
SINGLE-PATIENT EA POLICIES/CRITERIA
https://www.gene.com/patients/investigational-medicines/criteria
GlaxoSmithKline
Expanded Access Listings
Conditions:
Solid Tumours
Conditions:
Multiple Myeloma
Conditions:
Hypereosinophilic Syndrome
Additional Information
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.
Imara, Inc.
5 business daysAdditional Information
Available therapies via single-patient EA
IMR-687 for sickle cell disease
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/).
Immix Biopharma
24hr to 48hrAdditional Information
Available therapies via single-patient EA
imx-110 - all advanced solid tumors
SINGLE-PATIENT EA POLICIES/CRITERIA
Intensity Therapeutics, Inc.
1 weekAdditional Information
Available therapies via single-patient EA
There are no therapies available for single-patient EA at this time.
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.
Inventiva
15 business daysIterum Therapeutics plc
3 business daysAdditional Information
Available therapies via single-patient EA
Sulopenem sodium (intravenous)
Sulopenem etzadroxil/probenecid (tablet)
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.
Janssen
ImmediateExpanded Access Listings
Conditions:
Crohn Disease
Conditions:
Relapsed or Refractory Mantle Cell Lymphoma
Conditions:
Multiple Myeloma
Conditions:
Advanced Cancers and FGFR Genetic Alterations
Conditions:
Depressive Disorder, Treatment-Resistant
Conditions:
Diabetes Mellitus, Type 1
Conditions:
H7N9 Subtype of Influenza A Virus
Conditions:
HIV
Additional Information
SINGLE-PATIENT EA POLICIES/CRITERIA
http://www.janssen.com/sites/www_janssen_com/files/janssen_pre-approval_...
Johnson & Johnson
N/AKrystal Biotech, Inc.
5 business daysAdditional Information
Available therapies via single-patient EA
Bercolagene Telserpavec (KB103) - Dystrophic Epidermolysis Bullosa
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.
DISEASE/CATEGORY-SPECIFIC EA POLICIES/CRITERIA
https://www.krystalbio.com/wp-content/uploads/2019/07/Krystal-Expanded-A...
Kyowa Kirin Pharmaceutical Development, Inc.
N/AAdditional Information
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.
Melinta Therapeutics, Inc.
3 business daysAdditional Information
Available therapies via single-patient EA
fusidic acid (CEM-102)
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.
Merck & Co.
N/AExpanded Access Listings
Conditions:
Mycoses
Merus NV
5 business daysAdditional Information
Available therapies via single-patient EA
MCLA-128 is available for patients with solid tumors that harbor an NRG1 fusion
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.
DISEASE/CATEGORY-SPECIFIC EA POLICIES/CRITERIA
Millendo Therapeutics (US), Inc.
15 daysAdditional Information
Available therapies via single-patient EA
Currently, none.
Minovia Therapeutics
5 business daysAdditional Information
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
Mirati Therapeutics
1 business dayAdditional Information
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%20Poli...
Momenta
5 Business DaysAdditional Information
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.
NeoMatrix Therapeutics, Inc.
Additional Information
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
Novartis Pharmaceuticals
ImmediatelyExpanded Access Listings
Conditions:
Multiple Myeloma
Conditions:
Acute Lymphoblastic Leukemia (ALL), Diffuse Large B-cell Lymphoma (DLBCL)
Conditions:
Neuroendocrine Tumors
Conditions:
Acute Lymphoblastic Leukemia
Conditions:
Non-small Cell Lung Cancer (NSCLC)
Conditions:
PIK3CA-Related Overgrowth Spectrum (PROS)
Conditions:
HR+ Advanced or Metastatic Breast Cancer
Conditions:
Sickle Cell Disease
Conditions:
Acute Myeloid Leukemia (AML) With, FLT3 Mutation, Internal Tandem Duplication (ITD) or Tyrosine Kinase Domain (TKD)
Conditions:
Heart Failure With Reduced Ejection Fraction (HF-rEF)
Additional Information
Available therapies via single-patient EA
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)
Novo Nordisk
5 business daysOncoceutics, Inc.
48 hoursExpanded Access Listings
Conditions:
Glioma
Additional Information
Available therapies via single-patient EA
ONC201 - H3 K27M mutant gliomas
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
Ondine Biomedical
7 business daysAdditional Information
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
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.
Orinove Inc.
2 weeksAdditional Information
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.
Otsuka America Pharmaceutical
1 weekPfizer
5 business daysExpanded Access Listings
Conditions:
Neoplasm
Conditions:
Metastatic Merkel Cell Carcinoma
Sildenafil Therapy For Subjects Who Derive Clinical Benefit From Continued Treatment With Sildenafil
Conditions:
Pulmonary Arterial Hypertension
Additional Information
SINGLE-PATIENT EA POLICIES/CRITERIA
Pharnext SAS
NAAdditional Information
Available therapies via single-patient EA
NA
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’.
DISEASE/CATEGORY-SPECIFIC EA POLICIES/CRITERIA
NA - see above
Retrophin
7 business daysSanofi
1 business dayExpanded Access Listings
Conditions:
Cutaneous Squamous Cell Carcinoma
Savara Inc.
5 business daysAdditional Information
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.
SCYNEXIS, Inc.
24 hoursAdditional Information
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.
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.
Shire
3 business daysSilk Road Therapies, Inc.
N/AAdditional Information
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.
St. Jude Children's Research Hospital
Additional Information
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
Takeda Pharmaceuticals
3 business daysExpanded Access Listings
Conditions:
Disease, Hodgkin, Lymphoma, Large-Cell, Anaplastic, Lymphoma, Non-Hodgkin, Lymphoma, T-Cell, Cutaneous
Conditions:
Chronic Myeloid Leukemia (CML), Philadelphia Chromosome Positive Acute Lymphoblastic Leukemia (Ph+ ALL)
Additional Information
DISEASE/CATEGORY-SPECIFIC EA POLICIES/CRITERIA
https://www.takedaoncology.com/medicines/expanded-access-to-investigatio...
Triumvira Immunologics, Inc.
3 business daysAdditional Information
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
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.
UCB
N/AExpanded Access Listings
Conditions:
Epilepsy
Conditions:
Epilepsies, Partial
Conditions:
Crohn's Disease
Conditions:
Rheumatoid Arthritis
Additional Information
SINGLE-PATIENT EA POLICIES/CRITERIA
http://www.ucb.com/_up/ucb_com_patients/documents/UCB_Policy%20Access%20...
Ultragenyx
N/AExpanded Access Listings
Conditions:
Very Long-chain acylCoA Dehydrogenase (VLCAD) Deficiency, Carnitine Palmitoyltransferase Deficiencies (CPT1, CPT2), Mitochondrial Trifunctional Protein Deficiency, Long-chain Hydroxyacyl-CoA Dehydrogenase Deficiency, Glycogen Storage Disorders, Pyruvate Carboxylase Deficiency Disease, ACYL-CoA DEHYDROGENASE FAMILY, MEMBER 9, DEFICIENCY of, Barth Syndrome
Conditions:
X-linked Hypophosphatemia, Tumor-Induced Osteomalacia
Conditions:
MPS VII, Mucopolysaccharidosis VII, Sly Syndrome
Conditions:
Long-chain Fatty Acid Oxidation Disorders (LC-FAOD)
Additional Information
SINGLE-PATIENT EA POLICIES/CRITERIA
Requests for early access to investigational therapies must be made by a qualified physician.
Usona Institute
5 business daysAdditional Information
Available therapies via single-patient EA
Psilocybin capsules (25 mg)
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.
DISEASE/CATEGORY-SPECIFIC EA POLICIES/CRITERIA
Viela Bio
24 hoursAdditional Information
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.
Xynomic Pharmaceuticals, Inc.
5 business daysAdditional Information
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.
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.