Company Directory

Single-Patient EA Policies/Criteria

If your doctor believes that an investigational medicine could help you but you cannot take part in a clinical trial, it might still be possible to access the treatment. Single patient access may not be allowed in some countries, so please check your local regulations to find out. This is only possible in exceptional circumstances when you, or someone you’re caring for, are suffering from a severely debilitating or life-threatening disease that cannot be treated with available approved drugs, or where all possible approved treatments have been unsuccessful. At this point, your doctor might consider an investigational treatment as the only way to help.

We will let your doctor know we have received the application within two working days. Once we have all the information we need, we will do our best to complete our assessment and get back to your doctor within five working days.
We can only agree to provide an investigational medicine to your doctor if we agree with their medical evaluation that the possible benefits of the drug outweigh the potential risks. We take care to ensure that we comply with the laws that apply in different countries for investigational medicines and treatments. We also work together with doctors to ensure that they comply with local laws regarding access to investigational medicines, including monitoring and recording patient safety information. We consider every request for access carefully, but we cannot guarantee that it will be granted.  

Single-Patient EA Policies/Criteria

Belite Bio is currently unable to support an Expanded Access to its investigational drug (Tinlarebant, aka, LBS-008) due to very limited supplies of drug product.

Available Therapies via Single-Patient EA

None of Belite Bio's investigational drug products are available through Expanded Access.

Disease/Category-Specific EA Policies/Criteria

https://belitebio.com/#product-008

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 clinical stage of development, being evaluated as a treatment option in COVID-19 and 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.

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 offer any of our developmental medicines through expanded access programmes, with the exception of patients who could benefit for continued treatment upon completion of a company sponsored trial. We encourage those who are interested in learning more about our clinical development programmes, including eligibility criteria and locations to visit clinicaltrials.gov.

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).

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.

Single-Patient EA Policies/Criteria

Biothera is evaluating the safety and efficacy of its proprietary innate immune modulator, Imprime PGG, in combination with various immune checkpoint inhibitors and monoclonal antibodies for the treatment of multiple cancers. At this time, Biothera does not have a compassionate use program; however, it will consider providing Imprime PGG for individual patients (Single Patient IND) on a case-by-case basis. This opportunity applies only to patients with serious or life-threatening conditions who are unable to participate in a clinical trial and for whom there are no comparable or satisfactory alternative therapy options.

In determining whether single patient access is appropriate outside of a clinical trial, Biothera will consider many factors, including: the strength of available efficacy and safety data; the patient’s medical condition and history; the benefit-risk profile of our drug in relation to the patient’s case; the potential impact on the clinical development program; and the ability and willingness of the patient’s physician to sponsor the treatment application.

If you are a potential patient and your physician believes you may be a candidate for single patient access to Imprime PGG, please ask your physician to contact Biothera at earlyaccess@biothera.com.

Available Therapies via Single-Patient EA

Imprime PGG

Single-Patient EA Policies/Criteria

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

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)

Single-Patient EA Policies/Criteria

BlueRock has initiated a Ph1 clinical trial to evaluate the safety, tolerability, and preliminary efficacy of embryonic stem cell-derived dopaminergic neurons in patients with advanced Parkinson’s disease. We understand that some patients may wish to access our investigational drugs that have not yet been approved. At this stage of drug development and trials, we do not have enough clinical product or evidence of clinical safety and efficacy of our investigational drugs to support expanded access. Therefore, BlueRock Therapeutics does not currently offer an expanded access program at this time. The most appropriate way for patients to access our investigational drug is by participating in our clinical trials, and we encourage you to speak with your doctor about whether you would qualify for this trial. Information regarding ongoing clinical trials can be accessed at www.clinicaltrials.gov. (ClinicalTrials.gov Identifier: NCT0480273)

If you have additional questions about BlueRock’s expanded access policy, please speak to your physician, or contact regulatory@bluerocktx.com.