Early Access Program
We are committed to developing innovative therapies for patients with serious diseases.
Clinical trials are a critical component of this effort, and they help to determine whether a new therapy is safe and effective. Until regulatory authorities make the decision whether to approve a new therapy for a specific disease, it remains experimental and is generally not available to patients with that disease or condition outside of the clinical trials. If you would like to learn more about our clinical programs, visit clinical trials or www.clinicaltrials.gov.
We understand that there are critically ill patients who will not be eligible for available clinical trials and don’t have options for alternative therapies. In these circumstances, we will consider allowing access to the investigational therapy through Compassionate Use (CU) or an Early Access Program (EAP). CU is for an individual patient when no comparable or satisfactory alternative therapy options are available. An EAP is for a group of patients who have the same disease that was evaluated in clinical trials that we plan to submit to regulatory authorities in support of approval of the therapy. Both types of access programs require that the request for access to the drug is submitted by the treating physician and that the Early Access Core Principles are met:
EAP Core Principles
- Providing access will not jeopardize either the clinical trials which determine whether the therapy is safe and effective, or regulatory pathways which make the decision whether to approve the therapy.
- The patient has a serious and/or life-threatening disease and the physician believes there are no satisfactory alternative therapies on the market.
- The patient can potentially benefit from treatment and there is scientific evidence that the potential benefit outweighs risk to the patient’s safety.
- Providing access will not jeopardize the ability for Apellis to conduct essential tasks that are necessary to bring effective therapies to people suffering from the diseases we seek to treat.
Pegcetacoplan PNH Early Access Program
Apellis did have an Early Access Program for Paroxysmal Nocturnal Hemoglobinuria (PNH) in the United States. However, as pegcetacoplan is approved by the FDA, this program has been closed to enrollment. We do not have any further Early Access Programs available currently.
For any additional information please email email@example.com or submit a Compassionate Use request, below.