Company Directory

Single-Patient EA Policies/Criteria

Sagimet Biosciences Inc. (“Sagimet”) is dedicated to developing new therapies that have a positive impact on public health. Our ongoing clinical trials can be found by visiting the clinicaltrials.gov website. Consistent with Sagimet’s mission to bring innovative medicines to patients with serious or life-threatening illnesses or conditions, we are currently focused on enrolling and conducting the clinical trials necessary to apply for, and potentially gain, regulatory approvals to make our medicines available to patients. 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 is best suited to help us develop products that, if approved, could help patients.

We recognize that there may be situations where a patient with a serious or life-threatening condition may not be eligible for a Sagimet-sponsored clinical trial. However, at this time, Sagimet is not making any of its investigational drug candidates, including denifanstat, available via expanded access (also referred to as compassionate use). We have considered many factors in arriving at this determination, including making reasonable assessments of potential risk versus benefit for patients outside the clinical trial setting, and our ability to maintain supply for clinical trials, from enrollment to completion. Nevertheless, we will evaluate and respond to expanded access requests on a case-by-case basis, consistent with the steps below.

1. How to request: With questions about Sagimet’s expanded access policy or to make an expanded access request, please contact access@Sagimet.com. All requests must be submitted by the patient’s treating physician and provide sufficient supporting detail to enable us to evaluate your request. The physician’s contact information must be included to enable us to respond.

2. Criteria for consideration of requests: Although Sagimet retains sole discretion in deciding whether to grant an expanded access request, the following criteria will guide our evaluation of requests:

The patient has a serious disease or condition, or their life is immediately threatened by their disease or condition, and there are no available alternative treatments
The patient is not eligible for an ongoing or planned clinical trial;
The potential benefit to the patient clearly outweighs the potential risks of treatment with the investigational product
The patient meets any other relevant medical criteria for expanded access to the investigational product, as determined by Sagimet
In addition to the patient eligibility requirements, the investigational product must meet the following criteria:
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 sufficient evidence to expect that the investigational product will have an acceptable safety profile for the intended patient population
The provision of the investigational product will not interfere with or compromise the clinical development of the product
3. Timing: We will endeavor to acknowledge each submitted request from a treating physician within 15 business days of its receipt.

Sagimet is committed to evaluating all requests in a fair and equitable manner. Each request will be carefully considered by Sagimet whose decisions are final. Any early access we provide will be in line with the laws and requirements of the country involved. As authorized by the 21st Century Cures Act, Sagimet may revise this expanded access policy at any time. The posting of this policy by Sagimet does not serve as a guarantee of access to any specific investigational drug by any individual patient or physician.

Available Therapies via Single-Patient EA

Denifanstat for metabolic dysfunction-associated fatty liver disease

Available Therapies via Single-Patient EA

Procedure for Submitting Requests to Savara:
Savara will consider expanded access requests from treating physicians subject to US laws and regulations. All requests should be submitted via e-mail to EAP.US@savarapharma.com. 

Process for Review of Requests:
Savara is committed to a fair and impartial evaluation of each request for access to its investigational products. Therefore, all decisions are based solely on clinical circumstances and are guided by the criteria outlined below. Patients will be referred to ongoing clinical trials as the primary way to access investigational products. 

When evaluating requests for expanded access, Savara considers all of the following criteria: 
1. The patient for whom expanded access is requested suffers from a disease or condition that is serious or life-threatening. 
2. There are no comparable or satisfactory alternative therapies or clinical trials available. 
3. Sufficient preliminary efficacy and safety data exist to support an assessment that the benefit for the patient outweighs the potential risks and that the potential risks are not unreasonable in the context of the disease or condition being treated. 
4. Sufficient clinical data are available to identify an appropriate dose (amount and frequency) of the investigational drug. 
5. There is adequate drug supply to support the ongoing and necessary clinical trials as well as to support approved expanded access in a sustainable and equitable manner, until and if product becomes commercially available.
6. The patient is not eligible to participate in any ongoing clinical trials of the investigational drug. 
7. Expanded access will not adversely affect the clinical development program, in particular, the initiation, conduct, or completion of the clinical trials that are required for regulatory approval. 
8. The unsolicited request is made by a U.S. qualified and licensed physician who will take primary responsibility for supervising use of the investigational product from Savara and will comply with all applicable FDA regulatory requirements associated with treatment and use of an investigational product. 
9. All required regulatory and institutional approvals have been obtained. The patient must provide written informed consent. 

Requests for expanded access will be individually reviewed in accordance with these criteria. Savara is committed to evaluating all requests for expanded access in a fair and equitable manner. All requests will be evaluated by medical professionals and decisions will be based on available scientific evidence at the time of the request. 

Questions regarding Savara's ongoing U.S. expanded access program(s) can be forwarded to: EAP.US@savarapharma.com 

This policy is not applicable to countries outside the U.S. If you have questions on expanded access in a non-US country, please submit your request to info@savarapharma.com. 

Single-Patient EA Policies/Criteria

Scilex Holding Company (Scilex) is dedicated to the development and commercialization of non-opioid pain management products using innovative delivery technologies and is actively evaluating the safety and effectiveness of these therapies.

Expanded Access Policy
The US Food and Drug Administration (FDA) has established criteria for the provision of investigational medicines* to patients outside of clinical trials (compassionate use/expanded access). That guidance provides that expanded access is a potential pathway for a patient with a serious or immediately life-threatening disease or condition to gain access to an investigational medicine for treatment outside of clinical trials when no comparable or satisfactory alternative therapy options are available⁽¹⁾.
Expanded access may be appropriate when the following factors have been considered⁽²⁾:

• Patient has a serious or life-threatening illness or condition and is not responsive to or 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
• Patient enrollment in a clinical trial is not possible
• Potential patient benefit justifies the potential risks of treatment
• Providing the investigational medicine will not negatively impact or delay the conduct of clinical trials or regulatory review or approval of the investigational medicine for broader patient access
• Adequate supply of the investigational medicine is available

Currently, Scilex is not accepting applications for expanded access of our investigational medicines. Our development resources are focused on conducting clinical trials that evaluate the safety and effectiveness of our investigational medicines for treating acute and chronic pain. Our clinical trial programs are the primary way to gain access to a Scilex investigational medicine and Scilex encourages patients to speak with their treating physicians about participating in a clinical trial whenever possible.
Scilex may reconsider making its investigational medicines available through an Expanded Access Program in the future as we continually evaluate each of our investigational medicines for appropriateness of expanded access based on FDA’s established criteria.
Healthcare providers, patients and caregivers interested in learning more about Scilex’s investigational therapies should contact Scilex at eap@scilexpharma.com.
Additional information regarding our clinical trials may be accessed on www.clinicaltrials.gov.

*This policy is subject to change. Scilex may revise this policy at any time and may establish different expanded access policies for each investigational medicine under development. The posting of this policy by Scilex shall not serve as a guarantee of access to any specific investigational medicine by any individual patient.

**Investigational medicines have not yet been approved or cleared by FDA and FDA has not found these products to be safe and effective for their specific use. Furthermore, the investigational medicine may, or may not, be effective in the treatment of the condition, and use of the product may cause unexpected serious side effects.

References:
(1) https://www.fda.gov/news-events/public-health-focus/expanded-access (accessed September 1, 2022); see also 21 C.F.R. 312.305(a)
(2) https://navigator.reaganudall.org/companies-and-sponsors#introduction (accessed September 1, 2022)

Single-Patient EA Policies/Criteria

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@scorpiontx.com.

In line with the 21st Century Cures Act, Scorpion 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

SCYNEXIS is committed to making Investigational Products available to seriously ill patients who have exhausted other treatment options.  A treating physician, who is able to comply with the SCYNEXIS requirements, may request information about how to apply for access to SCYNEXIS’ Investigational Products by contacting the company.

Available Therapies via Single-Patient EA

SCY-078 is a triterpenoid, glucan synthase inhibitor, antifungal agent undergoing investigation for:
•    The treatment of invasive candidiasis, including candidemia (IC). 
•    The treatment of invasive aspergillosis (IA), including IA that is refractory to or intolerant of other antifungal therapies.
•    The treatment of invasive candidiasis in patients who are refractory to or intolerant of other antifungal therapies.

Single-Patient EA Policies/Criteria

Scytek Laboratories Inc- #1 diagnostic reagents: Special Stains, Antibodies, Microbiology ScyTek Biotech Life Sciences manufactures diagnostic reagents, Special Stains, Antibodies, Hematology, Immunohistochemistry, buffers, Microbiology, Hematoxlyn, Gram Stain kit, Methylene Blue Solution, Giemsa Stain kit etc
https://www.scytek.com

Single-Patient EA Policies/Criteria

Serac Healthcare will consider providing a requesting physician with pre-approval access to the Serac Healthcare investigational drug, for the treatment of an individual patient outside of a clinical trial, when certain conditions are met.

• The patient has a disease for which there is sufficient evidence of a projected benefit from the use of the investigational product and the benefit outweighs the known or anticipated risks
• 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
• 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
• No viable alternative option is available

Available Therapies via Single-Patient EA

Maraciclatide is an in vivo imaging agent (non-therapeutic) for use in endometriosis.

Single-Patient EA Policies/Criteria

Single-Patient Expanded Access Policies and Criteria -. Physicians seeking general information may contact Servier by emailing expandedaccess@servier.com .

Available Therapies via Single-Patient EA

Vorasidenib - The vorasidenib EAP is designed to provide access to vorasidenib for patients with IDH1- or IDH2-mutated glioma who are not eligible for other vorasidenib clinical trials, and who in the opinion of the treating oncologist would potentially benefit from treatment from vorasidenib.

Disease/Category-Specific EA Policies/Criteria

https://www.earlyaccesscare.com/companies/servier-pharmaceuticals-llcVo

Single-Patient EA Policies/Criteria

Henlius is a global biopharmaceutical company with the vision to offer high-quality, affordable, and innovative biologic medicines for patients worldwide with a focus on oncology, autoimmune diseases, and ophthalmic diseases.

 

HLX42, a novel antibody-drug conjugate (ADC) that targeting epidermal growth factor receptor (EGFR)，is one of the first ADC candidates of Henlius to enter into clinical development. HLX42 is designed to overcome the resistance of current EGFR-targeted therapies and to provide more effective treatment options to fulfill the unmet clinical needs. HLX42 is to be investigated as a single agent in a Phase I trial for the treatment of solid tumors, including EGFR-mutated non-small cell lung cancer that progressed on a third-generation EGFR tyrosine kinase inhibitor.

 

Expanded Access is a potential pathway for a patient with a serious or immediately life-threatening 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.  

 

Henlius understands the interest of patients in accessing HLX42 outside of clinical trials for potentially life-threatening diseases, however we do not provide any Expanded Access programs for HLX42 at this time.

 

Henlius recognizes the need for Expanded Access programs and will revise this policy based on data from future clinical trials.

Available Therapies via Single-Patient EA

Shanghai Miracogen Inc. (Miracogen) is a biopharmaceutical company focusing on development of innovative antibody-drug conjugates (ADC) and bringing new therapies for patients with various cancers. Miracogen conducts human clinical trials for its investigational products to generate safety and efficacy data as required to support the regulatory approval by the U.S. Food and Drug Administration (FDA) and regulatory authorities in other countries and subsequent wider accessibility to patients.

Considering the stage of development of the investigational products, Miracogen is seeking to retain its capacity of manufacturing and drug supply to assure adequate supply to ongoing clinical trials and development programs to generate data on the safety and efficacy of the investigational products in order to support regulatory approval and make the products accessible to the broader patient population. At this time, Miracogen is unable to provide any of its investigational drug products on an expanded access or right-to-try basis. We encourage patients to speak with their physicians and to participate in the available clinical trials.

If you have any questions about our expanded access policy, please contact Miracogen via email at: expanded_access@miracogen.com.cn.

For more information on the available clinical trials, please visit https://clinicaltrials.gov and search company, condition, or treatment. For additional information on Expanded Access, please visit the U.S. Food and Drug Administration at: https://www.fda.gov/news-events/public-health-focus/expanded-access.

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

Single-Patient EA Policies/Criteria

At SIMR we aspire to discover and develop differentiated therapeutics for the treatment of peripheral neuropathic pain. SIMR’s lead product, SR419, is a candidate drug with novel mechanism of action for the treatment of peripheral neuropathic pain, which is discovered and developed by SIMR. Peripheral neuropathic pain is the pain caused by damage or pathological changes of peripheral nerves, including post-herpetic neuralgia, diabetic peripheral neuropathy, chemotherapy-induced peripheral neuropathy, and postoperative peripheral neuropathy. The results of preclinical studies show that SR419 has excellent safety and pharmacokinetic properties. In addition, SR419 demonstrates the potential of better analgesic efficacy and fewer side effects than pregabalin, a commonly used first-line treatment for neuropathic pain. SR419 has completed phase I clinical trials in the Australia and China with positive safety results, and is planning to carry out international multi-center phase II clinical trials. At SIMR, we are focused on enrolling and conducting clinical trials aimed at gaining regulatory approval, making SR419 available to patients as quickly as possible. As such, we believe participation in our clinical trials is the most appropriate way to access SR419 at this stage of development. Information about our clinical trials, including eligibility criteria and locations, is available at www.clinicaltrials.gov. SIMR understands the interest of patients in accessing SR419 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 SR419. SIMR 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.

Disease/Category-Specific EA Policies/Criteria

http://www.simrbio.com/en/ExpandedAccessPolicy/

Single-Patient EA Policies/Criteria

At Silence, our vision is to transform peoples’ lives around the world by silencing diseases through our precision engineered medicines and driving positive change for the communities around us. As pioneers in the design and development of siRNAs (short interfering RNAs), we are advancing a new generation of medicines to potentially address the needs of patients who have limited or inadequate treatment options. Our investigational product candidates are designed to harness the body's natural mechanism of RNA interference (RNAi) to precisely target and silence disease-associated genes.

Silence conducts clinical trials to ensure that our investigational product candidates are safe and effective, which supports approval of the treatment by regulatory authorities and ultimately enables patients to access approved, safe and effective treatments.

At this time, Silence does not offer an expanded access program for its investigational products and these can only be accessed by participation in a clinical trial. If in the future Silence decides to offer an expanded access program, Silence will update its policy accordingly. If you are a patient who is interested in accessing one of our investigational products by participating in a clinical trial, please consult with your physician. Anyone who wishes to learn more about Silence’s investigational products or this policy can contact Silence by emailing info@silence-therapeutics.com. More information on Silence’s clinical trials can be found at www.clinicaltrials.gov.

Available Therapies via Single-Patient EA

At this time Silk Road Therapies, Inc./Ipekyolu Ilac Ltd. is testing a topical pentoxifylline formulation under US IND, but exclusively conducted in a trial in Istanbul, Turkey. At this time, we are unable to import our topical formulation from Turkey and do not have sufficient supply to offer expanded access to this drug. Therefore, until such time as the proof of concept trial is completed, Silk Road Therapies, Inc. is not offering expanded access beyond participation in the Istanbul trial.

However, we anticipate completing the proof of concept trial 3Q2019 and having reformulated material available in the US 1Q2020. At that time, we will revise this expanded access policy and may permit access to our product for patients who are ineligible for entrance into our later phase III pivotal trial.

Single-Patient EA Policies/Criteria

In very specific cases, SonALAsense may be able to provide access to our investigational drug outside of our clinical trials if certain conditions are met. Single Patient Expanded Access may not always be available as each patient is unique and this will be at the discretion of a physician based on many factors and conditions. To be eligible for access to the investigational medicine, a physician must apply and certify that the patient for whom the application is being submitted meets the following criteria:

Diagnosed with a serious, life-threatening or severely debilitating disease;
1. Unable to participate in SonALAsense clinical trials;
2. Have no comparable or satisfactory alternative treatment option available;
3. The risk/benefit profile of the investigational drug is favorable in light of the sufficient safety and efficacy information and the treating physician’s assessment of the patient’s medical condition.

 

Available Therapies via Single-Patient EA

Diffuse intrinsic pontine glioma
Recurrent or progressive glioblastoma

Single-Patient EA Policies/Criteria

Expanded access is used to describe treatment with an investigational product for a patient with a serious or life-threatening illness or condition when there are no comparable or satisfactory alternative treatments available and the patient is not eligible for a clinical trial. These investigational products are not approved by the U.S. Food and Drug Administration (FDA). The expanded access pathway is often used by commercial pharmaceutical companies to provide their products before they are approved.

St. Jude Children’s Research Hospital (St. Jude) is dedicated to its mission of advancing cures for pediatric catastrophic diseases through research and treatment. St. Jude provides the best possible care for our patients. We also make discoveries that will lead to better treatment for children and young adults around the world. We accomplish this by offering patients participation in clinical trials such as the LVXSCID-ND study (NCT01512888). 

St. Jude Children’s Research Hospital is not a commercial pharmaceutical company and does not have the capability to manufacture products at an industrial level. Supplies of the investigational product for the LVXSCID-ND study are limited and earmarked for the completion of a prospective research guided clinical trial. At this time, the safety and efficacy of the product is under investigation. St. Jude is not able to provide the investigational product in the LVXSCID-ND study for expanded access. If your child has been diagnosed with X-Linked Severe Combined Immunodeficiency (SCID-X1), please contact the physician below to discuss possible treatment options.

Disease/Category-Specific EA Policies/Criteria

https://www.stjude.org/lvxscid-nd

Single-Patient EA Policies/Criteria

We currently do not accept requests for expanded access use of our investigational products. Supporting single patient expanded access would negatively impact our resources and ability to complete the active clinical investigations.

We encourage anyone interested in receiving investigational products manufactured for Stanford Center for Cell Therapy investigator-initiated trials to contact the Principal Investigator through ClinicalTrials.gov listing to see if they are eligible to enroll on an open study.

Available Therapies via Single-Patient EA

None are available via single-patient EA.

Disease/Category-Specific EA Policies/Criteria

https://www.stebabiotech.com/pipeline

Single-Patient EA Policies/Criteria

Describe your company's single-patient EA policies and criteria.
Patient Eligibility Criteria:
To be eligible for accessing to the investigational product, patients must meet following criteria:
• Has a serious or life-threatening condition with no satisfactory alternative.
• Ineligible for participation in any ongoing clinical study of the investigational product, which includes lack of access due to geographic limitations.
• Did not receive any cell-based therapy within 1 year.
• Assessment that benefi ts outweigh the risks to the patient.
• Must agree to comply with all study requirements and be willing to complete all study visits.
Investigational Product Criteria:
• The product is under clinical investigation in one or more clinical trials, with high safety profi les.
• Assessment that the company has an adequate supply of the investigational product.
• A determination that expanded access will not interfere with the company's ability to complete clinical trials in a timely fashion or which might otherwise delay marketing approval and ultimately availability to all patients.
Treating Physician Criteria and Responsibility:
The physician must agree in writing to comply with:
• Physician(s) is (are) properly licensed.
• Physician(s) meet the applicable country-specifi c legal and regulatory requirements related to providing the investigational products under the Expanded Access.
• Physician(s) must be experienced and familiar in cord blood or stem cell infusion.
• Any StemCyte’s requirements in terms of safety reporting and protection of intellectual properties.
• A treating physician may submit questions and requests regarding to the Expanded Access to email: expandedaccess@stemcyte.com. We will do our best efforts to acknowledge each submitted request within 10 business days.

Available Therapies via Single-Patient EA

Post-COVID syndrome

Single-Patient EA Policies/Criteria

Summit is committed to improving patients’ quality of life, extending their potential lifespan, and addressing serious unmet medical needs. Our goal is to provide access to our medicines at the appropriate time and in the manner that is most beneficial to the relevant patient populations. We believe that participation in our ongoing clinical trials is the safest and most effective way to achieve this goal, and we encourage patients to discuss these options with their physicians.

Currently, our investigational medicine, ivonescimab, is being studied to demonstrate its efficacy and establish its safety profile in patients across North America, Europe as well as Japan. Until ivonescimab’s efficacy and safety are demonstrated, Summit will not be making it available through expanded access. We strongly encourage participation in a clinical trial as the best way to access our investigational therapies. More information about our ongoing clinical trials can be found on the ClinicalTrials.gov website.
In accordance with the 21st Century Cures Act, Summit reserves the right to revise this policy at any time.

 

Single-Patient EA Policies/Criteria

Sobi may make an investigational medicine available if the following eligibility criteria and additional requirements are met:
§ The patient has a serious or life-threatening disease without a satisfactory alternative therapy or treatment and an unsolicited Managed Access request has been submitted to Sobi by the treating physician
§ The patient is not eligible or able to enter an ongoing clinical trial
§ There is sufficient data to indicate that the benefit of the investigational medicine outweighs the potential risks in the context of the disease or condition being treated
§ There is a sufficient supply of the investigational medicine available and providing the medicine will not interfere with ongoing clinical studies or other development activities
§ The patient meets any eligibility criteria set by Sobi’s Medical Committee evaluating Managed Access requests
§ Laws and regulations in the country of request allow Managed Access

Single-Patient EA Policies/Criteria

Syncromune is an oncology-focused biopharmaceutical company committed to developing therapies that may improve the lives of cancer patients. Our focus centers around conducting clinical research that evaluates the safety and effectiveness of new therapies for patients with solid tumor cancers. Our clinical trial programs are the primary way to get access to a Syncromune investigational therapy. These clinical trials provide the most effective way to assess how our investigational therapies may treat cancer, and are used to support regulatory approval.

At this time, Syncromune does not provide access to investigational therapies outside of clinical trials or prior to regulatory approval. You and your health care provider may learn more about our clinical trials by going to the clinical trials section of our website, or visiting ClinicalTrials.gov and searching for Syncromune.

If you are a health care provider who is interested in learning more about one of our investigational therapies, or a physician with questions about participation in one of our clinical trials, please submit a request to trials@syncromune.com. Syncromune will acknowledge your request as soon as possible, typically within 3 days of receipt.

If applicable, this website will be updated with hyperlinks to the relevant expanded access information on ClinicalTrials.gov. Syncromune reserves the right to revise this expanded access policy at any time.

Pursuant to the 21st Century Cures Act, the posting of policies by manufacturers and distributors shall not serve as a guarantee of access to any specific investigational therapy by any individual.

Available Therapies via Single-Patient EA

Metastatic castrate-resistant prostate cancer