Indication Study Title NCT Number
Warm Autoimmune Hemolytic Anemia
A Phase 1b, Multicenter, Open-Label, Safety, Tolerability, and Activity Study of SYNT001 in Subjects with Chronic, Stable Warm Autoimmune Hemolytic Anemia (WAIHA) NCT03075878 Pemphigus (vulgaris or foliaceus) A Phase 1b, Multicenter, Open-Label, Safety, Tolerability, and Activity Study of SYNT001 in Subjects with Chronic Pemphigus (Vulgaris or Foliaceus) NCT03075904
COMPASSIONATE USE POLICY
At Syntimmune, our mission is to provide profound clinical benefit to patients with diseases mediated by FcRn, such as IgG-mediated autoimmune disease.
The biology we control with our medicines is well researched in order to bring treatment solutions to patients suffering from diseases with inadequate or nonexistent treatment options.
Investigational drugs are drugs that are not approved by regulatory agencies like the United States Food and Drug Administration (FDA). Clinical trials are used to test investigational drugs to establish their safety and efficacy before they are approved. Approval by regulatory authorities like the FDA is the only way to make treatments broadly available to the patient population at large. Thus, Syntimmune’s focus is on conducting a robust and efficient clinical development program to support regulatory approval as quickly as possible.
During clinical development, participation in clinical trials is the preferred way for patients to access an investigational drug because these trials generate the efficacy and safety data needed to determine whether the investigational drug should be approved. However, sometimes a patient may not be able to enroll in a clinical trial and may thus seek access to the investigational drug outside of a clinical trial through compassionate use, which may also be called expanded access or pre-approval access.
When evaluating requests for compassionate use of an investigational drug, Syntimmune must consider all of the following criteria:
- The disease or condition being studied is serious or life-threatening.
- There are no comparable or satisfactory alternative therapies or clinical trials available.
- 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. This information is not expected to be available before the end of randomized, placebo-controlled studies, and possibly even later depending on the clinical program.
- Sufficient clinical data is available to identify an appropriate dose of the investigational drug.
- There is adequate drug supply to support the ongoing and necessary clinical trials as well as to support approved compassionate use in a sustainable and equitable manner.
- The patient is not eligible to participate in any ongoing clinical trials of the investigational drug. An inability to participate in a trial due to geographic limitations does not typically mean a patient is not eligible.
- Compassionate use 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.
- The request is made by a qualified and licensed physician, unsolicited by Syntimmune or any other individual or organization. The requesting physician must have the expertise and facilities appropriate for the administration of the investigational drug and for monitoring, managing, and reporting any side effects and the patient’s overall experience.
- All required regulatory and institutional approvals have been obtained. The patient must provide written informed consent.
Based on the above criteria, Syntimmune has determined that none of our investigational drugs are suitable for compassionate use under any circumstance at this time. We will periodically reevaluate this policy as our clinical development programs advance.