Begin Your Search

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.

A B C D E F G H I J K L M N O P Q R S T U V W X Y Z
Company Name
Phone Number & Email
Company Acknowledgement

Bayer

Expected Application Timeframe
Two working days
Tumors Harboring NTRK Fusion

Larotrectinib expanded access is for patients with cancer with a NTRK1, NTRK2, or NTRK3 gene fusion, who are ineligible for an ongoing larotrectinib clinical trial or have other considerations that prevent access to larotrectinib through an existing clinical trial. Gene fusion occurs when a gene is made by joining parts of two different genes. NTRK gene fusion can lead to the development of solid tumors in a variety of tissue types. The study drug larotrectinib blocks the action of the NTRK gene fusion. Expanded access is intended to treat individual patients with different types of cancers with a NTRK gene fusion who are unresponsive to current standard treatment for their condition and also are unable to participate in ongoing clinical trials.

Solid Tumors Harboring NTRK Fusion

Selitrectinib expanded access is for minor and adult patients with cancer having a change in a particular gene (NTRK1, NTRK2, or NTRK3 gene fusion). The patients are ineligible for an ongoing selitrectinib clinical trial or have other considerations that prevent access to selitrectinib through an existing clinical trial. Expanded access is intended to treat individual patients with different types of cancers with a NTRK gene fusion, including blood cancers, who have previously received tropomyosin receptor kinase (TRK) inhibitor therapy.

BerGenBio ASA

Phone Number & Email
Expected Application Timeframe
N/A
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

Phone Number & Email
Expected Application Timeframe
1 business day
Infantile-onset Spinal Muscular Atrophy

To provide access to nusinersen to eligible patients with Infantile-onset Spinal Muscular Atrophy (SMA) (consistent with Type 1) to address a high-unmet medical need.

Biohaven Pharmaceuticals

Expected Application Timeframe
2 business days
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).

Biosight

Single-Patient EA Policies/Criteria

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

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

Biothera Pharmaceuticals, Inc.

Expected Application Timeframe
7 business days
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

BioXcel Therapeutics

Expected Application Timeframe
5 Business Days

Blaze Bioscience, Inc.

Expected Application Timeframe
10 business days

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

Single-Patient EA Policies/Criteria

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

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

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

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

Available Therapies via Single-Patient EA

tozuleristide

Blueprint Medicines Corporation

Phone Number & Email
Expected Application Timeframe
5 business days
GIST

This is a US, multicenter, open-label expanded access program to provide access to avapritinib until such time that avapritinib becomes available through other mechanisms or the Sponsor chooses to discontinue the program.

Non-Small Cell Lung Cancer
Medullary Thyroid Cancer

This is a global, multicenter, open-label pre-approval access program to provide access to pralsetinib (BLU-667) until such time that pralsetinib becomes available through other mechanisms or the Sponsor chooses to discontinue the program.

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)

Boehringer Ingelheim Pharmaceuticals

Phone Number & Email
Expected Application Timeframe
5 business days
HIV Infections

To provide early access to tipranavir and evaluate the safety and tolerance of tipranavir combined with low dose of ritonavir in patients with progressive, HIV-1 disease who have failed or are intolerant to currently approved treatments for HIV infection, who are unable to participate in another tipranavir controlled clinical trial and have an urgent need for anti-HIV treatment.

HIV Infections

This study was designed to provide early access to and evaluate the safety of TPV/r in PI-experienced patients with HIV-1 infection.

HIV Infections

The purpose of this Open Label Safety Study is to provide access to and evaluate the safety and tolerability of TPV/r in treatment-experienced patients with advanced HIV-1 infection who have failed at least two PI-containing regimens, and have limited treatment options.

Carcinoma, Non-Small-Cell Lung

The program will provide early access to the investigational drug BIBW 2992 to treat patients with advanced NSCLC who have failed at least 12 weeks on erlotinib or gefitinib. The Compassionate Use Programme will also provide additional safety and efficacy information on BIBW 2992 use. Named Patient Use (NPU)

Carcinoma, Non-Small-Cell Lung

This is an open-label, multi-center, single-arm trial, designed to provide early access to afatinib and to provide additional information on the safety and efficacy of afatinib in advanced NSCLC patients who harbor an EGFR mutation.

Carcinoma, Non-Small-Cell Lung

To provide expanded access and to evaluate the safety, tolerability and efficacy of afatinib in patients with locally advanced or metastatic non-small cell lung cancer (NSCLC) harboring Epidermal Growth Factor Receptor (EGFR) mutation(s) and have never been treated with an EGFR tyrosine kinase inhibitor (TKI)

Idiopathic Pulmonary Fibrosis

To provide early access and to evaluate the safety and tolerability of nintedanib in patients with idiopathic pulmonary fibrosis (IPF).

Lung Diseases, Interstitial

This Expanded Access Program is intended to facilitate the availability of OFEV to patients suffering from non Idiopathic Pulmonary Fibrosis-Interstitial Lung Disease (non IPF-ILD) with a progressive clinical course despite Standard of Care treatment and for whom no satisfactory authorised alternative therapy exists or who cannot enter a clinical trial.

Bristol-Myers Squibb

Phone Number & Email
Expected Application Timeframe
48 hours
Leukemia

This protocol allows CML and Ph+ ALL subjects who are resistant to or intolerant of imatinib mesylate, to potentially benefit from dasatinib. It is intended to provide patients with access to dasatinib while awaiting reimbursement decision in Korea and it will also provide additional data on the safety of dasatinib in Korean population

Renal Transplantation

To make belatacept available for recipients of a renal allograft who are currently intolerant to or have contraindications to CNIs and/or m-TOR inhibitors and are either: - unable to construct an adequate immunosuppression regimen due to non-renal toxicity / contraindication (and withdrawing the causative agent would lead to renal graft loss) OR - at imminent risk of losing the allograft kidney due to nephrotoxicity and have no other options for renal replacement therapy

Multiple Myeloma

The objective of this expanded access program is to provide treatment with elotuzumab in combination with lenalidomide and dexamethasone for patients with relapsed or refractory multiple myeloma at U.S. sites where licensed physicians determine clinical need.

Multiple Myeloma

The objective of this expanded access program is to provide treatment with elotuzumab in combination with lenalidomide and dexamethasone for patients with relapsed or refractory multiple myeloma at Japanese sites where licensed physicians determine clinical need.

Multiple Myeloma

At BMS, we work with physicians/investigators to make investigational products available to patients with life-threatening diseases that have exhausted other treatment options and where there is a reasonable expectation of benefit over risk. When contacted by a treating physician, BMS will consider requests for providing early patient access to Elotuzumab in patients with multiple myeloma who are residents of Belgium, Thailand, Turkey, Argentina, and Colombia.

Pediatric Cancer

At BMS, we work with physicians/investigators to make investigational products available to patients with life-threatening diseases that have exhausted other treatment options and where there is a reasonable expectation of benefit over risk. When contacted by a treating physician, BMS will consider requests for providing early patient access to Nivolumab in pediatric patients exhibiting a high mutational load.