Company Directory

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 clinicaltrials@c4therapeutics.com. 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 clinicaltrials.gov if such record becomes active.

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 www.clinicaltrials.gov.

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.

Available Therapies via Single-Patient EA

None

Disease/Category-Specific EA Policies/Criteria

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

Single-Patient EA Policies/Criteria

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

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

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

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

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

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

Available Therapies via Single-Patient EA

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

Single-Patient EA Policies/Criteria

Cellphire Policy: Expanded Access Use of Thrombosomes®

Overview

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

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

Policy Statements

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

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

Requesting Access

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

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

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

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

Available Therapies via Single-Patient EA

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

Single-Patient EA Policies/Criteria

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

Available Therapies via Single-Patient EA

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

Single-Patient EA Policies/Criteria

Checkpoint Therapeutics, Inc. (Checkpoint) is clinical-stage biotechnology company focused on accelerating the development and commercialization of potential life-changing oncology therapeutics. Checkpoint’s lead asset, cosibelimab (CK-301), is under clinical investigation for the potential treatment of solid tumor indications, and a biologics license application (BLA) for cosibelimab is currently under review with the US Food and Drug Administration for the potential approval of cosibelimab for advanced cutaneous squamous cell carcinoma. As clinical investigation of cosibelimab in solid tumors is ongoing, the preferred patient access pathway to cosibelimab treatment is via clinical trial participation. Information about cosibelimab clinical trials, including key eligibility criteria and clinical trial locations, is available at clinicaltrials.gov.
For access to cosibelimab treatment or other Checkpoint investigative therapies outside of ongoing clinical trials and prior to regulatory approval (eg, for patients who do not qualify for or are not able to participate in a current Checkpoint-sponsored clinical trial), Checkpoint will consider a physician’s request for access to a Checkpoint investigative therapy outside of the clinical trial setting. All such requests must be made in accordance with local laws and regulations and must be submitted by the treating physician to checkpointexpandedaccess@checkpointtx.com for Checkpoint’s review.

Available Therapies via Single-Patient EA

Cosibelimab (also referred to as CK-301)

Single-Patient EA Policies/Criteria

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

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

Available Therapies via Single-Patient EA

Pegunigalsidase alfa for Fabry disease

Single-Patient EA Policies/Criteria

Codagenix Inc. (www.codagenix.com) 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 www.clinicaltrials.gov and search using the company name.

Disease/Category-Specific EA Policies/Criteria

https://codagenix.com/expanded-access-policy/

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: https://resources.cookmyosite.com/terms-and-conditions-of-sale-and-delivery-0

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

Single-Patient EA Policies/Criteria

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

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

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

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

Single-Patient EA Policies/Criteria

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

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

Consistent with CStone’s mission, our development resources are focused on conducting clinical trials required by regulatory authorities to fully answer important scientific questions about the potential risks and benefits of sugemalimab, and to gain regulatory approvals to make sugemalimab available broadly to patients as quickly as possible. To reach this goal successfully, our top priorities must be to ensure that an adequate supply of sugemalimab is available for those patients in our clinical trials and that drug supply will not hinder us from completing the clinical studies and obtaining regulatory approvals that will lead to wider patient access. As such, CStone does not provide any Expanded Access programs for sugemalimab outside of clinical trials at this time. Please visit https://clinicaltrials.gov and search by company, disease or medicine for applicable clinical trials.

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

 

Available Therapies via Single-Patient EA

At Curadel Surgical Innovations, Inc. we aspire to improve human surgery by visually enhancing anatomy of every kind with FLARE® drugs. The technology we’ve developed uses low levels of invisible near-infrared (NIR) fluorescent light and NIR cameras available in most hospitals around the world to highlight any structure in the body that could help the surgeon perform better surgery. We are currently conducting clinical trials on our products, aiming to obtain regulatory approval to make this technology available to all patients as quickly as possible. As such, we believe that participation in our clinical trials is the most appropriate way to access FLARE® drugs at this time. Information about our clinical trials, including eligibility criteria and locations, is available at https://www.clinicaltrials.gov.

Curadel Surgical Innovations, Inc. understands the interest of patients and surgeons in accessing FLARE® drugs outside of clinical trials and prior to regulatory approval, however, we do not currently offer any Expanded Access Programs.