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

At Beacon Therapeutics, we are committed to developing products that bring new, innovative gene therapies, like AGTC-501 (laruparetigene zovaparvovec), to patients with serious diseases with no or limited treatment options.

Currently, Beacon Therapeutics does not offer an expanded access program and is not accepting expanded access requests for our investigational products, including AGTC-501.

We understand patient interest in accessing AGTC-501 outside of clinical trials and prior to regulatory approval. However, participation in one of our clinical trials is currently the most appropriate way to access our investigational products. Please see ClinicalTrials.gov and the Beacon Clinical Trials webpage for publicly available information related to Beacon Therapeutics’ ongoing clinical trials.

If you have any questions about Beacon Therapeutics’ EA policy, please contact expandedaccess@beacontx.com

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.

Available Therapies via Single-Patient EA

At BerGenBio, we are focused on enrolling and conducting clinical trials aimed at gaining regulatory approval and making bemcentinib available to patients as quickly as possible. As such, we believe participation in a company-approved 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 programs, except for patients who could benefit for continued treatment upon completion of a company-approved trial.

Single-Patient EA Policies/Criteria

Binhui Bio currently has only one clinical drug product that can be used for EA, which is OH2 for the treatment of patients with unresectable stage III or IV melanoma that are resistant to anti-PD1 therapy.

Available Therapies via Single-Patient EA

Binhui Bio, a leading biotechnology company specializing in the research, development, and pilot production of innovative drugs, places patient wellbeing at the forefront of our priorities. Among our breakthroughs, the OH2 injection, a unique product derived from oncolytic viruses, has garnered attention by securing five INDs and one orphan drug approval from China NMPA and US FDA since 2018. The OH2 injection, designed for monotherapy and combination therapy for advanced melanoma cancer.

Recognizing that there may be scenarios where patients require access to our yet-to-be-approved medicines beyond the scope of clinical trials, we have established guidelines to address these unique situations. Such exceptions might arise when patients battling serious or life-threatening diseases have no other means to join a clinical trial, and lack satisfactory alternative treatment options. Under these circumstances, a healthcare professional (HCP) may be granted the use of an unlicensed medicine for patient treatment. This kind of utilization of unapproved medicines outside the framework of clinical trials is commonly referred to as "compassionate use" or "expanded access".

This policy delineates Binhui's approach to the treatment use of our unlicensed pharmaceutical products and the criteria we employ to assess requests from HCPs.

• The patient's condition being addressed is either life-threatening or causes significant impairment to daily life.
• There are no other satisfactory treatment options available, as confirmed by the treating HCP.
• Sufficient evidence is available that indicates the potential benefits of the unlicensed medicine for the patient outweigh the potential risks involved.

Moreover, we consider several additional factors when making decisions about supplying unlicensed medicines:

• There should be enough detailed information available to guide the correct application of the investigational medicine, particularly concerning the appropriate dosage and duration of treatment for the patient.
• We assess the potential impact of the treatment use of the investigational medicine on any concurrent clinical trials or its regulatory pathway.
• We only allow the use of our medicines in countries with an adequate medical infrastructure to safely administer the treatment.
• We only extend use in broad access programs to countries where Binhui Bio intends to pursue regulatory approval and subsequently make the medicine available. This condition may not necessarily apply to individual-named patients.
• We consider the ethical implications of our decisions, with particular attention to the principle of fair treatment for all patients.
• We ensure that the proposed use of our treatment is in accordance with local legal and regulatory frameworks.

Our senior medical staff, strictly following medical and ethical criteria, makes decisions to supply unlicensed medicines for treatment use.

We at Binhui Bio strongly believe that participation in one of our clinical trials is the most effective way to access our OH2.

HCPs seeking to request unlicensed medicines for treatment use should file the treatment IND to FDA and submit relevant information to IRB.

For further information on submitting a request for treatment use of our investigational medicines, HCPs are directed to follow this link. https://www.binhui-bio.com/

Single-Patient EA Policies/Criteria

Expanded Access is a potential pathway for patients diagnosed with a serious and/or life-threatening disease or condition to gain access to an investigational product for treatment outside of a clinical trial when no comparable or satisfactory alternative therapy options are available. In these instances, a patient’s treating physician can request an investigational product prior to regulatory approval, where allowed by local laws.

Who is Eligible for Expanded Access Programs?
BioAtla considers granting expanded access to its investigational products when all of the following criteria are met:
• A patient is diagnosed with a serious and/or life-threatening disease or condition.
• There are no adequate standard or investigational therapies available.
• The patient is not eligible for or cannot participate in a BioAtla-sponsored study for the therapy being requested.
• There must be adequate data supporting use of the investigational medication in the treatment of the patient’s
disease.
• Adequate supply exists to support both the ongoing clinical trials and expanded access, until and if a product
becomes commercially available.
• Expanded 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 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.
• Expanded 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 BioAtla may preclude appropriate use of the
investigational medicine, will be considered.

Available Therapies via Single-Patient EA

A Phase 2 Open Label Study of BA3021 in Patients With Recurrent or Metastatic Squamous Cell Carcinoma of the Head and Neck

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

Bionomics Limited does not offer an expanded access program for its investigational products.

Available Therapies via Single-Patient EA

BioNova Pharmaceuticals (Shanghai) Limited (hereinafter referred to as BioNova) is currently not providing BN104 Tablets for Expanded Access use.

BN104 is a novel, highly selective and potent oral menin inhibitor developed by BioNova and currently being investigated as a potential treatment for acute leukemias patients who harbor a mixed-lineage leukemia rearranged (MLLr) or nucleophosmin (NPM1) mutation. On October 23, 2023, BN104 was designated as a Fast Track product by the FDA.

A phase 1 clinical study constitutes the Part 1 dose-escalation and Part 2 dose-optimization will be conducted in eligible patients with acute leukemias. The study is designed to establish a safe and optimized dose and appropriate dosing schedule for BN104. Once RP2D established, and with agreement from the FDA, the company will initiate the phase 2 program in patients with acute leukemias who harbor a MLLr or NPM1 mutation.

BioNova will review its EA policy annually to determine if a change in policy is warranted and feasible.

Disease/Category-Specific EA Policies/Criteria

BioNova will respond to all Expanded Access requests within 14 business days. All requests for Expanded Access or additional information may be sent by email to info@bionovapharma.com. For email requests, please indicate EXPANDED ACCESS REQUEST in the subject line.

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

CartiLife is an autologous chondrocyte cell-based therapy for the restoration of symptomatic and structural complications caused by articular cartilage defects.

Suitable for treatment of defects which are: ICRS grade III or IV, with defect sizes between 2 ~ 10 cm^2.

Available Therapies via Single-Patient EA

Biosolution Co.,Ltd does not provide access to investigational products outside of clinical trials. We encourage patients to participate in clinical trials of our investigational therapies whenever possible, because clinical trials are designed, conducted, and monitored to ensure that the safety and efficacy are appropriately evaluated before they are submitted to regulatory agencies for review with the intent to make them more broadly available to patients.

Single-Patient EA Policies/Criteria

CartiLife is an autologous chondrocyte cell-based therapy for the restoration of symptomatic and structural complications caused by articular cartilage defects.

Suitable for treatment of defects which are: ICRS grade III or IV, with defect sizes between 2 ~ 10 cm^2.

Available Therapies via Single-Patient EA

Biosolution Co.,Ltd does not provide access to investigational products outside of clinical trials. We encourage patients to participate in clinical trials of our investigational therapies whenever possible, because clinical trials are designed, conducted, and monitored to ensure that the safety and efficacy are appropriately evaluated before they are submitted to regulatory agencies for review with the intent to make them more broadly available to patients.

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.