Early Access Program

We are committed to delivering innovative therapies to 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 not generally 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 opening an Early Access Program (EAP) and providing a requesting physician with access to pegcetacoplan when EAP core principles have been met and the patient meets the EAP eligibility criteria.

EAP Core Principles

  • The patient has a life-threatening, significantly progressive 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.
  • An EAP must 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.
  • An EAP must honor the commitment to patients participating in our clinical trials who are not receiving pegcetacoplan.
  • Apellis has plans to make pegcetacoplan available in the patient’s country.

Pegcetacoplan PNH Early Access Program

Apellis did have an Early Access Program for PNH in the United States.  However, as pegcetacoplan is approved by the FDA, this program has been closed to enrollment.

For any additional information please contact earlyaccess@apellis.com.