Apellis Pharmaceuticals
Apellis Pharmaceuticals works on 10 rare diseases tracked on Trial Friend, including Amyotrophic Lateral Sclerosis, C3 Glomerulopathy, Cold Agglutinin Disease and 7 more, with 6 active clinical trials (4 recruiting) and 2 FDA-approved rare disease drugs.
Apellis Pharmaceuticals develops targeted C3 complement inhibitors for hematologic and renal rare diseases. Empaveli (pegcetacoplan) is FDA-approved for adults with paroxysmal nocturnal hemoglobinuria (2021) and for people 12 and older with C3 glomerulopathy or primary immune-complex membranoproliferative glomerulonephritis (July 2025). Syfovre, an eye injection of pegcetacoplan, is approved for geographic atrophy caused by age-related macular degeneration (February 2023). On May 14, 2026, Biogen completed its acquisition of Apellis for $41 per share in cash plus a contingent value right worth up to $4 per share, tied to Syfovre sales; Apellis is now a wholly owned subsidiary of Biogen.
Apellis Pharmaceuticals Drug Pipeline
Apellis Pharmaceuticals has 6 active clinical trials across 3 development stages, with 4 currently recruiting participants. Clinical trials advance through phases: Phase 1 tests safety in a small group, Phase 2 evaluates effectiveness and side effects, Phase 3 confirms benefit in a larger population, and Phase 4 monitors long-term safety after FDA approval.
Note: This pipeline counts the active and recruiting trials listed on this page, from ClinicalTrials.gov. For companies that work outside rare disease, we list only trials for rare diseases we track, so the company's full pipeline may be larger.
Apellis Pharmaceuticals Clinical Trials (6)
Active and recruiting clinical trials sponsored by Apellis Pharmaceuticals, sourced live from ClinicalTrials.gov. Each trial card shows the study phase, current recruitment status, conditions under investigation, study locations, eligibility criteria, and a direct link to the full ClinicalTrials.gov record. You can also download a one-page PDF summary to share with your doctor.
Note: Recruitment statuses on ClinicalTrials.gov may not immediately reflect recent FDA decisions, sponsor announcements, or enrollment changes. Always confirm a trial's current status directly with the study coordinator.
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Apellis Pharmaceuticals FDA-Approved Drugs (2)
Medications developed or marketed by Apellis Pharmaceuticals that have received U.S. Food and Drug Administration approval. Drug data is sourced from the openFDA database and includes brand names, generic names, approval dates, and matched rare disease indications. Where a drug treats a condition covered by Trial Friend, the disease name links directly to that disease page.
| Drug Name | Brand Name | Rare Diseases | Approval Date |
|---|---|---|---|
| PEGCETACOPLAN Complement Inhibitor [EPC] | Empaveli subcutaneous | May 14, 2021 | |
| PEGCETACOPLAN Complement Inhibitor [EPC] | Syfovre intravitreal | Feb 17, 2023 |
Apellis Pharmaceuticals Trial Locations
Apellis Pharmaceuticals clinical trials are running at 174 sites in 18 countries. Click any country to drill down by state, city, and individual research facility. Proximity to a trial site is one of the most important factors in deciding whether to participate.
Rare Disease Focus Areas (10)
Diseases targeted by Apellis Pharmaceuticals's clinical trial and drug development programs
Amyotrophic lateral sclerosis is a progressive neurodegenerative disease destroying motor neurons in the brain and spinal cord. This causes progressive weakness and paralysis of voluntary muscles whil...
C3 glomerulopathy is a group of rare kidney diseases caused by dysregulation of the alternative complement pathway, leading to uncontrolled C3 deposition in the glomeruli. The two subtypes, C3 glomeru...
Cold agglutinin disease is a rare autoimmune hemolytic anemia caused by IgM antibodies that stick to red blood cells in the cold. Most of the red cell destruction happens outside the blood vessels, ma...
Focal Segmental Glomerulosclerosis (FSGS) is a kidney disease characterized by scarring of some glomeruli, the tiny filtering units that remove waste from the blood. The disease can develop on its own...
Geographic atrophy is the advanced form of dry age-related macular degeneration, characterized by progressive loss of retinal pigment epithelium, photoreceptors, and choriocapillaris in well-defined a...
IgA nephropathy is the most common primary glomerulonephritis worldwide, caused by abnormal IgA1 antibodies depositing in the kidney's mesangium, activating complement pathways and triggering inflamma...
Patient Resources
Organizations and resources related to Apellis Pharmaceuticals's rare disease focus areas
Frequently Asked Questions About Apellis Pharmaceuticals
Common questions about Apellis Pharmaceuticals's rare disease programs, clinical trials, and treatments.