For Patients

We care deeply about the needs and experiences of people with serious diseases and aim to champion the patient and caregiver voice in everything we do.

To make the greatest impact, we are committed to listening and learning from patients, caregivers, and patient advocacy groups, and integrating their perspectives and insights into our decision-making.

Our goal is to be representative of the communities we serve, and to develop clinical trials, therapies, and resources that consider the unique needs of patients. We value our collaborations with patient organizations and the patients and caregivers they serve, and are committed to an authentic, continuous, and transparent dialogue.

Living with GA

Living with ALS

Learn more about our ongoing clinical trials and early access program below.

Contact our Patient Advocacy team to learn more about how we work with patient organizations and the communities they support: patientadvocacy@apellis.com.

Our Areas of Focus

Ophthalmology

Geographic Atrophy (GA)

GA is an advanced form of age-related macular degeneration (AMD) that leads to progressive and irreversible vision loss. There are no approved therapies for GA, which impacts ~5 million people worldwide.

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Rare Disease

Paroxysmal Nocturnal Hemoglobinuria (PNH)

PNH is an acquired, rare, chronic, and potentially life-threatening blood disease that can appear at any age and in any race or gender, and is most often diagnosed in people in their early 30s.

Learn more » Learn more about our ongoing clinical trials »

Amyotrophic Lateral Sclerosis (ALS)

ALS is a neurodegenerative disease that results in progressive muscle weakness and paralysis. There are currently no treatments that have been shown to stop or reverse the progression of ALS.

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Immune Complex Membranoproliferative Glomerulonephritis (IC-MPGN) and C3 Glomerulopathy (C3G)

IC-MPGN and C3G are rare kidney diseases that cause inflammation and damage to the organ. There are no medicines currently approved to treat IC-MPGN or C3G.

Learn more » Learn more about our ongoing clinical trials »

Cold Agglutinin Disease (CAD)

CAD is a chronic, rare blood disorder where the body’s immune system attacks and destroys its own red blood cells by mistake. There are limited treatment options for CAD, which can result in anemia, chest pain, and the need for frequent transfusions.

Learn more » Learn more about our ongoing clinical trials »

Hematopoietic Stem Cell Transplantation-Associated Thrombotic Microangiopathy (HSCT-TMA)A

HSCT-TMA is a rare blood disease that can result from a bone marrow transplant complication and lead to organ damage. There are currently no approved treatments for HSCT-TMA.

Learn more » Learn more about our ongoing clinical trials »

Clinical Trials & Early Access Program

Clinical trials are a critical component of our efforts to deliver innovative therapies to people living with serious disease, and they help to determine whether a new therapy is safe and effective. Until regulatory authorities decide 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:

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 pegcetacoplan is submitted by the treating physician and that the Early Access Core Principles are met:

Early Access 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.

For any additional information please contact earlyaccess@apellis.com or submit a Compassionate Use request below.