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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 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.

4 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

C4 Therapeutics, Inc.

Expected Application Timeframe
5 business days
Single-Patient EA Policies/Criteria

On our website, both physicians and patients may explore information about our investigational agents. Our EAP policy is described below.

Several factors consistent with the FDA and other regulatory agencies’ guidelines, are essential when considering expanded access requests:

  • The illness must be serious or life-threatening with no other satisfactory treatment options (such as approved products or open clinical trials)
  • There is sufficient evidence that the potential benefit to the patient would likely outweigh the potential risks, based on available safety and efficacy information
  • The ability to provide a therapy in a fair and equitable manner, so that there is adequate manufacturing capacity to provide therapies across all ongoing clinical trials and expanded access 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

Given the early stage of our development program, we believe that participation in our clinical trial is the most appropriate way to access our investigational therapy. Therefore, we are not currently making our investigational product available through an expanded access program. If you have additional questions, please speak with your physician or have your physician contact We anticipate acknowledging receipt of requests sent to this email within five business days.

In line with the 21st Century Cures Act, C4 Therapeutics, Inc may revise this policy at any time. This website and policy will be updated with a hyperlink or other reference to the expanded access record on if such record becomes active.

Cara Therapeutics

Expected Application Timeframe
3 business days
Uremic Pruritus

This is an intermediate-size patient population expanded access protocol for the use of intravenous (IV) difelikefalin for the treatment of moderate-to-severe pruritus associated with chronic kidney disease in adult patients undergoing hemodialysis.

Single-Patient EA Policies/Criteria

Cara Therapeutics is committed to the development and commercialization of novel therapies for the treatment of chronic pruritic conditions. To do this, we conduct clinical trials to assess the safety and efficacy of our investigational products. For a list of clinical trials currently recruiting patients, please visit

Clinical trials generate data that may allow us to apply for approval of our therapies for commercialization from regulatory agencies such as the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA). Regulatory approval of our products may ultimately provide broader access to our medicines.


Phone Number & Email
Expected Application Timeframe
24 hours
Multiple Myeloma

This study is designed to evaluate the safety and efficacy of nonconforming idecabtagene vicleucel (ide-cel) in participants with multiple myeloma per the approved prescribing information. This is an expanded access protocol (EAP) to be conducted at Risk Evaluation and Mitigation Strategies (REMS) qualified sites approved for commercial administration of idecabtagene vicleucel and where the EAP is authorized to be conducted for use of nonconforming idecabtagene vicleucel. Non-conforming idecabtagene vicluecel is idecabtagene vicleucel that does not meet commercial release specifications but may be acceptable for use as an investigational product in the Expanded Access Protocol setting.

Acute Myelogenous Leukemia (AML)

This is an expanded access program (EAP) for eligible participants designed to provide access to CC-486.

Multiple Myeloma

To provide pomalidomide access to relapsed/refractory multiple myeloma subjects with a likelihood of benefit from the pomalidomide treatment while the medication is not commercially available

Myelodysplastic Syndrome

This is an expanded access program (EAP) for eligible participants designed to provide access to ACE-011.


This is an expanded access program (EAP) for eligible participants designed to provide access to fedratinib. Expanded access is only available in markets where fedratinib is not yet approved.

Celldex Therapeutics

Expected Application Timeframe
5 business days
Available Therapies via Single-Patient EA


Disease/Category-Specific EA Policies/Criteria

Cellectar Biosciences

Phone Number & Email
Expected Application Timeframe
5 business days
Single-Patient EA Policies/Criteria

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

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

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

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

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

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

Available Therapies via Single-Patient EA

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

Cellphire Therapeutics, Inc.

Expected Application Timeframe
24 hours
Single-Patient EA Policies/Criteria

Cellphire Policy: Expanded Access Use of Thrombosomes®


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

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

Policy Statements

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

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

Requesting Access

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

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

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

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

Available Therapies via Single-Patient EA

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

Center for Cancer Research, National Cancer Institute, National Institutes of Health

Expected Application Timeframe
5 business days
Single-Patient EA Policies/Criteria

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

Available Therapies via Single-Patient EA

Chiesi USA

Phone Number & Email
Expected Application Timeframe
5 calendar days
Fabry Disease

The objective of this treatment protocol is to provide guidance to Treating Physicians who seek access to pegunigalsidase alfa for Fabry patients whose clinical condition, in the opinion of the Treating Physician, requires treatment with enzyme replacement therapy (ERT) with pegunigalsidase alfa and a) cannot be adequately treated with currently approved FDA products and/or b) are not able or willing to participate in any of the on-going clinical trials in the United States.

Single-Patient EA Policies/Criteria

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

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

Available Therapies via Single-Patient EA

Pegunigalsidase alfa for Fabry disease

Codagenix Inc.

Expected Application Timeframe
10 business days
Single-Patient EA Policies/Criteria

Codagenix Inc. ( is developing investigational vaccines for the prevention of a variety of infectious diseases. Our vaccines are in early stages of development; therefore, their potential risks and benefits are in the process of being evaluated. Participation in clinical trials should be the primary route by which patients obtain access to investigational vaccines and contribute to the collection of safety and efficacy data needed to support regulatory approval worldwide. Because our vaccines by their nature are designed for prophylaxis, not treatment use, Codagenix does not intend to offer Expanded Access to these investigational vaccines. To learn more about available clinical trials, please visit and search using the company name.

Disease/Category-Specific EA Policies/Criteria

Cook MyoSite, Incorporated

Expected Application Timeframe
5 business days

This is an expanded access program (EAP) for eligible participants designed to provide access to autologous muscle derived cells (AMDCs). Treating physicians may submit requests for expanded access to Cook MyoSite's investigational product by email to

Single-Patient EA Policies/Criteria

Cook MyoSite is committed to conducting rigorous, controlled clinical trials with variable inclusion and exclusion criteria based upon the indicated use under investigation. These trials are designed to demonstrate to regulatory authorities that our investigational product is safe and effective, and as a result, allow our investigational cell therapy product to become a valuable treatment option for a wide range of patients. Participation in our clinical trials is the first and most preferable route to access our investigational product.

Cook MyoSite will consider providing an investigational product to a qualified requesting physician currently licensed within the United States via the United States Food and Drug Administration’s (FDA’s) expanded access pathway outside of an active clinical trial when the following general requirements are met:
• The patient has a serious or life-threatening disease or condition with no satisfactory alternative;
• Positive assessment that the anticipated benefits outweigh the risks to the patient;
• Positive assessment that Cook MyoSite has an adequate supply of resources for producing the investigational product;
• A determination that expanded access will not interfere with Cook MyoSite’s ability to complete clinical trials in a timely fashion or which might otherwise delay marketing approval and ultimately availability to all patients;
• The patient can undergo a muscle biopsy procedure(s), and;
• The patient can undergo Cook MyoSite’s required donor screening and testing.

For additional information, please refer to our website:

Available Therapies via Single-Patient EA

Autologous Muscle Derived cells (AMDC).
Single-patient EA studies have included AMDC for the treatment of esophageal aperistalsis and AMDC for the treatment of underactive bladder

Crestone, Inc.

Expected Application Timeframe
5 business days
Single-Patient EA Policies/Criteria

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

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

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

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

Curon Biopharma