Company Directory

Single-Patient EA Policies/Criteria

ILiAD is committed to developing safe and effective vaccines, with a goal to providing our next generation vaccines as soon as possible through efficient vaccine development. At this time, ILiAD’s vaccines are in the investigational phase and not yet marketed. ILiAD does not have an expanded access program.

Disease/Category-Specific EA Policies/Criteria

https://iliadbio.com/clinical.html

Single-Patient EA Policies/Criteria

We are considering an intermediate group EAP.

Single-Patient EA Policies/Criteria

Imara is dedicated to developing novel therapeutics for patients with sickle cell disease and other hemoglobinopathies. Sickle cell disease represents a critical unmet medical need globally in which it is considered as a rare disease in many parts of the world, including in the United States, and as an endemic condition in several African countries. Imara’s lead product candidate, IMR-687, is under development for the treatment of sickle cell disease.

Expanded access may provide an avenue to use an investigational product, such as IMR-687, outside a clinical trial to diagnose, monitor, or treat a serious condition or disease in a patient. Imara has used guidelines from the US Food and Drug Administration (US FDA) and other regulatory agencies to develop the following criteria for when expanded access may be made available on a case-by-case basis for individual patients: 

•    Ongoing or planned clinical studies are not available to the patient, including lack of access due to geographical location of potential clinical trial sites, 
•    Significant evidence exists that supports both the safety and the efficacy of the investigational drug for the indication, 
•    The potential benefits to the particular patient seeking access to the investigational drug outweigh the potential risks to the patient,
•    An adequate supply of the investigational drug exists,
•    All necessary regulatory/institutional approvals have been obtained to allow drug administration, and 
•    The request for expanded access has been made by a qualified health care provider with expertise appropriate for the administration of the drug and for monitoring and managing the patient.

Imara supports expanded access programs and the need for a suitable policy, and it intends to provide patients with sickle cell disease access to IMR-687 at a suitable time and in the correct method when used outside a clinical trial. At this time, Imara believes that the most appropriate way to use our investigational lead product candidate, IMR-687, is by participation in one of our clinical trials (https://clinicaltrials.gov/).

Available Therapies via Single-Patient EA

IMR-687 for sickle cell disease

Single-Patient EA Policies/Criteria

http://immixbio.com/pipeline/compassionate-use-policy/

Available Therapies via Single-Patient EA

imx-110 - all advanced solid tumors

Single-Patient EA Policies/Criteria

Immuneonco will consider providing a requesting physician with pre-approval access to a specific 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.
• Adequate supply of the investigational drug is available.

Immuneonco is committed to evaluating all requests in a fair and equitable manner. All requests must be submitted by the patient’s treating physician. Immuneonco may require more detailed information in order to fully evaluate a request. Each request will be given careful consideration by Immuneonco whose decisions are final.

Physicians seeking pre-approval access for patients with no alternative treatment options should submit their requests to the mailbox (info@immuneonco.com). We regularly monitor this mailbox and will use our best efforts to acknowledge each submitted request within 7 working days after receipt.

To learn more about the EA policy by Immuneonco, please visit www.immuneonco.com.

Available Therapies via Single-Patient EA

IMM2902

IMM2902 is a recombinant bispecific monoclonal antibody. The indication is the use of IMM2902 in patients with HER2-expressing advanced solid tumors, breast cancer and gastric cancer.

Disease/Category-Specific EA Policies/Criteria

http://immuneonco.com/info.php?class_id=103102

Single-Patient EA Policies/Criteria

Immunic is dedicated to developing new therapies that have a positive impact on patient health, and to serving patients, patient families and patient
communities through education, empathy, and awareness. Consistent with Immunic’s mission to bring innovative medicines to patients with serious or life-threatening illnesses or conditions, we are focused on enrolling and conducting the clinical trials necessary to gain regulatory approvals to make our medicines 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, Immunic will consider providing a requesting physician with pre-approval access to a specific  Immunic 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 the clinical development having reached the stage of blinded Phase 3 data read-out
• 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. 

Immunic is committed to evaluating all requests in a fair and equitable manner. All requests must be submitted by the patient’s treating physician;  Immunic 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 Immunic whose decisions are final. 

Single-Patient EA Policies/Criteria

ImmVira is a biotechnology company focused on development of new generation novel drug vectors driven by clinical benefits in oncology and non-oncology fields. T3011 is a replication-competent, genetically modified virus derived from herpes simplex virus type 1 expressing IL-12 and PD-1 antibody.

ImmVira recognizes that some patients with serious or life-threatening disease may lack other treatment options and be unable to participate in a clinical trial. Under these circumstances, a healthcare professional (HCP) may request access to ImmVira's investigational medicines for patient treatment. This access is called "compassionate use" or "expanded access" and allows the use of an unapproved drug outside of a clinical trial.

This policy outlines ImmVira's process for reviewing requests from HCP for compassionate use of our investigational drugs. To be considered, the patient's condition must be:
• Severity of the patient's condition: The illness must be life-threatening or significantly impact daily life.
• Lack of alternatives: The doctor treating the patient must confirm there are no other suitable treatments available.
• Potential benefit outweighs risk: There must be evidence suggesting the potential benefits of the investigational drug are greater than the potential risks for this specific patient.

Beyond the initial criteria, several factors influence our decision on supplying investigational drugs:
• Treatment Guidance: Clear instructions for dosage and treatment duration are necessary for safe use.
• Clinical Trials: We evaluate if access might affect ongoing clinical trials or the drug's regulatory path.
• Medical Infrastructure: The treatment can only be provided in countries with facilities to safely administer it.
• Broad Access Programs: We prioritize countries where we plan to seek approval and make the drug commercially available. This might not apply to individual patients.
• Ethical Considerations: We ensure fair treatment principles are followed for all patients.
• Legal Compliance: The treatment must comply with local regulations.
• Drug Availability: We must have sufficient supply of the investigational drug to fulfill the request.

We strongly believe that participation in one of our clinical trials is the most effective way to access T3011.

ImmVira will also consider providing a requesting physician with pre-approval access to T3011, for the treatment of an individual patient outside of a clinical trial. ImmVira is committed to evaluating all requests in a fair and equitable manner. All requests must be submitted by the patient’s physician. Each request will be given careful consideration by ImmVira whose decisions are final.

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.immviragroup.com/.

Available Therapies via Single-Patient EA

T3011  Intratumoral Injection in Patients With Advanced Solid Tumors

Single-Patient EA Policies/Criteria

Indapta’s Expanded Access Policy (EAP) covers the use of its investigational products outside of an ongoing clinical trial. Indapta appreciates the need for additional treatments for patients who have serious or immediately life-threatening diseases and have limited available treatment options. The decision to establish an EAP is dependent on a number of key factors consistent with the US Food and Drug Administration (FDA) and other regulatory agencies’ requirements. These include, but are not limited, to the following:

• The illness for which the investigational cell therapy is being considered must be serious or life-threating, with no other satisfactory treatment options (such as approved products or enrolling clinical trials);

• There is sufficient evidence that the potential benefit of the investigational drug to the patient would outweigh the potential risks, based on all available safety and efficacy information; and

• Providing the investigational cellular immunotherapy as part of an EAP will not interfere with the enrollment of clinical trials to support the development and marketing approval. Based on these factors and the early stage of clinical development for our products, Indapta believes that participation in our clinical trials is the appropriate means to access our investigational cellular immunotherapies.

Therefore, Indapta does not currently have an expanded access program. If you are a patient seeking access to our investigational product(s), please contact your treating healthcare provider.

For more information about our ongoing clinical trials, please refer to www.clinicaltrials.gov, study number NCT06119685. Healthcare providers who are interested in learning more about clinical program(s) and investigational product(s) and/or participation in our clinical trial(s), please contact trials@indapta.com.

Available Therapies via Single-Patient EA

Universal, allogeneic Natural Killer cell therapy
Indications under investigation:
-Relapsed/Refractory Multiple Myeloma
-Relapsed/Refractory Non-Hodgkins Lymphoma

Single-Patient EA Policies/Criteria

Intensity Therapeutics’ (the Company) is the developer and drug manufacturer for a novel investigational drug product, INT230-6. This new product is in early clinical development and uses intratumoral injection for its delivery modality. Recently the FDA granted Fast Track Status to INT230-6 for development in patients with triple negative breast cancer who have failed 2 lines of therapy.

The drug is being used to treat solid tumor cancers. Intensity Therapeutics has chosen not to offer EA and will not accept EA requests for its products at this time.

Available Therapies via Single-Patient EA

There are no therapies available for single-patient EA at this time.

Single-Patient EA Policies/Criteria

Currently, Invyvid does not have an expanded access program that permits access to our investigational products prior to regulatory authority (such as FDA) authorization.

Single-Patient EA Policies/Criteria

At present we do not provide expanded access, also known as compassionate use or pre-approval access, to InxMed’s investigational medicinal products (IMP) outside the confines of clinical trials. And we do not have any expanded access programs at this time.
For information on InxMed’s available clinical trials, please visit: www.ClinicalTrials.gov or regional appropriate equivalent(s).
If you have additional questions regarding this policy, please speak with your health care provider or contact: expandedaccess@inxmed.com.

Single-Patient EA Policies/Criteria

IO Biotech ApS (IO Biotech) is the developer of a novel investigational drug product, IO102-IO103 Immunotherapeutic Cancer Vaccine. The investigational drug product is being evaluated for the treatment of previously untreated, unresectable or metastatic melanoma regardless of BRAF mutation status in combination with an anti-PD-1 antibody.

At the current stage of clinical development, IO Biotech has chosen not to offer Expanded Access (EA) and will not accept EA requests for the investigational drug product for the following reasons:

• IO Biotech’s ability to conduct clinical trials is limited. Offering EA under any setting at this time will impact IO Biotech’s ability to execute its trials due to a limited supply of the investigational drug product.
• IO Biotech does not have the personnel and other resources to offer EA on a fair and equitable basis.
• The investigational drug product requires special handling, transportation and training on the method of administration.

As IO Biotech is not currently accepting EA requests, the specified policy information on procedures and evaluation criteria is not provided at this time. The status of EA restriction will be re-evaluated at later development stages.

IO Biotech ApS Expanded Access Policy, Rev: 23 December 2020

Available Therapies via Single-Patient EA

Olezarsen for Familial Chylomicronemia Syndrome (FCS)

At present, Ionis Pharmaceuticals has an EAP available for olezarsen (ClinicalTrials.gov ID NCT06360237). Olezarsen is an investigational antisense oligonucleotide-GalNAc3 conjugate that causes degradation of apoC-III mRNA and a reduction of serum apoC-III protein. Studies suggest that apoC-III regulates both triglyceride metabolism and hepatic clearance of chylomicrons and other triglyceride-rich lipoproteins. The expanded access program for olezarsen is intended for those at risk of severely elevated triglyceride levels, particularly those living with FCS. The program is currently only available in the United States.

If you are a patient, family member or caregiver and you wish to know more about the olezarsen EAP for FCS, please discuss the EAP and all treatment options with your treating physician.

If you are a treating physician and are seeking information about the olezarsen EAP or would like to request access for a patient, please contact patient.access@bionicalemas.com.

Bionical Emas is a Clinical Research Organization managing the olezarsen EAP on behalf of Ionis.

Safety and efficacy have not been evaluated or established by any regulatory authorities for olezarsen.

Single-Patient EA Policies/Criteria

ISA Therapeutics (ISA) is developing ISA101b in HPV16 positive malignancies. The goal of our current clinical trial program is to obtain clinical data on ISA101b that is required by the US Food and Drug Administration (FDA) and other regulatory authorities for review and approval of marketing authorization applications for this investigational agent. ISA believes that focusing on clinical trials is the most appropriate way to achieve this goal; therefore, ISA101b is currently not offered outside of clinical trials and, at this time, ISA does not have an Expanded Access Program. In the event that ISA decides to consider expanded access, ISA will update its policy at that time and will then evaluate and respond to each request that it receives on a case-by-case basis. For more information on our investigational therapies and ongoing clinical trials, please visit clinicaltrials.gov.

Single-Patient EA Policies/Criteria

At this point of time, we do not have an expanded access program that allows patients to have access to our investigational products prior to FDA approval.

Single-Patient EA Policies/Criteria

Iterum Therapeutics was founded with one over-arching goal: to develop an antibiotic that addresses the growing crisis of multi-drug-resistant pathogens and meets the specific needs of physicians, their patients and other important stakeholders. Among the most important demands were new treatments for common, often serious conditions, including urinary tract infections (UTI), and intra-abdominal infections (IAI). Iterum Therapeutics is committed to conducting the clinical trials required to gain regulatory approvals to allow our medicines to be available to patients. Patients are encouraged to participate in one of our clinical trials whenever possible. For those patients with an unmet need who have exhausted all available therapy options and who are unable or ineligible to participate in a clinical trial, we may be able to provide access to our investigational medicine(s) in certain circumstances through our expanded access program.
Iterum Therapeutics may provide access for patients to our investigation medicine(s) outside of a clinical trial provided the patient meets Iterum Therapeutics’ evaluation criteria for such access. At a minimum, requests must meet the following criteria in order to be considered:
•    The patient has a serious or immediately life-threatening illness when there are no comparable or satisfactory alternative treatments available and the patient is ineligible or unable to participate in a clinical trial;
•    The benefit-risk analysis of the patient’s condition and medical history support the use of an investigational drug and justifies the potential risks of the treatment;
•    Making the investigational therapy available for expanded access will not interfere with the initiation, conduct, or completion of clinical trials being conducted by Iterum Therapeutics that could support marketing approval for a particular treatment indication;
•    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;
•    There is an adequate supply of the investigational medicines;
•    Provision of such access to investigational medicines is compliant with local regulations and laws.

All requests meeting the above criteria must be submitted by the patient’s treating physician to EAProgram@iterumtx.com. Iterum Therapeutics may request additional information from the patient’s treating physician to support the request. The treating physician must also agree to obtain appropriate regulatory and Institutional Review Board approvals and patient informed consent and to carry out patient monitoring and safety reporting. 

Iterum Therapeutics is committed to assessing every early access request with the highest degree of fairness and equality, however, Iterum Therapeutics cannot guarantee access to any investigational medicine. Iterum Therapeutics regularly monitors this email box and will attempt to acknowledge each submitted request within 3 business days after receipt. 

Available Therapies via Single-Patient EA

Sulopenem sodium (intravenous)
Sulopenem etzadroxil/probenecid (tablet)