Annexon’s Expanded Access Program
Expanded access, also called “compassionate use” or “early access,” is the use of an investigational medicine prior to regulatory approval and outside of a clinical trial.
We are a clinical-stage biopharmaceutical company developing a pipeline of novel therapies for patients with complement-mediated disorders of the body, eye and brain.
We do this by conducting clinical trials in which we collect safety and efficacy data about our investigational drugs with the goal of submitting those data to regulatory authorities, like the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA), so that these investigational drugs can potentially be approved for use by patients.
At this time, participation in one of our clinical trials is generally the most appropriate way to access our experimental drugs. However, in very rare circumstances, Annexon may be able to make an experimental drug that we are developing available for patients outside of a traditional clinical trial under an expanded or early access protocol, sometimes referred to as a treatment protocol. This is a program where patients may receive the experimental drug for as long as they meet certain criteria and are informed about the risks of participation.
A number of factors, consistent with regulatory agencies’ regulations and guidelines, are taken into account by Annexon when considering requests for expanded access. They include:
- The patient's illness must be serious or life-threatening;
- there are no other satisfactory treatment options available to the patient;
- patient enrollment in a clinical trial is not possible;
- there is sufficient evidence (based on available safety and efficacy data) that the potential benefit to the patient justifies the potential risks of treatment;
- providing the investigational drug will not interfere with the conduct of clinical trials or other aspects of development that could interfere with or delay regulatory filings designed to make the investigational drug more broadly accessible;
- there is adequate supply of the investigational drug; and
- the investigational drug can be administered, and it is logistically feasible to make it available, outside of the clinical trial setting.
The existence of an expanded access program does not guarantee any individual patient access to any of our investigational drugs. Participation in one of our clinical trials is the most appropriate way to access our investigational drugs. We encourage patients to speak first with their physician about their eligibility to enroll in a clinical trial. If a treating physician believes expanded access may be the only option for a patient, the physician should contact Annexon to make a formal request on behalf of the patient. This will enable the physician to work with Annexon to determine the best course of action. The request for access to an investigational drug can only be considered if the patient's treating physician is committed to, and supportive of, the requested treatment.
Treating physicians, patients, and/or caregivers interested in learning more about our investigational drugs currently undergoing clinical trials can find more information on clinicaltrials.gov. If you have additional questions, please speak with your physician or contact firstname.lastname@example.org. We anticipate acknowledging receipt of requests sent to this email within 10 business days.