Apellis Pharmaceuticals
Rare disease drugmaker · part of Biogen · based in Waltham, United States · founded 2009 · apellis.com
Apellis Pharmaceuticals works on 12 rare diseases we track, including Amyotrophic Lateral Sclerosis, C3 Glomerulopathy and 10 more. It has 6 active clinical trials, and 4 are looking for volunteers now. It also has 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).
Apellis Pharmaceuticals pipeline
Each row lists the diseases Apellis Pharmaceuticals is studying at that stage, from the ones closest to approval down to the earliest work.
Focal Segmental Glomerulosclerosis recruiting · C3 Glomerulopathy
Paroxysmal Nocturnal Hemoglobinuria recruiting · Geographic Atrophy recruiting
Geographic Atrophy 2 trials recruiting
Apellis Pharmaceuticals clinical trials
These are Apellis Pharmaceuticals trials for the rare diseases we track, from ClinicalTrials.gov. Statuses there can lag behind company news, so confirm with the study team before you plan around one.
4 trials looking for volunteers now
A Sequential Phase 2/3 Study of APL2 in Patients With Focal Segmental Glomerulosclerosis
Tests APL2, Placebo
For FSGS
A Study of Pegcetacoplan in Pediatric Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH)
Tests Pegcetacoplan
For Paroxysmal Nocturnal Hemoglobinuria (PNH) and Paroxysmal Hemoglobinuria
A Phase 2, Randomized, Placebo Controlled, Multicenter, Masked Study to Evaluate the Efficacy, Safety, Tolerability, and Pharmacodynamics of Multidose APL 3007 in Combination With Syfovre/Pegcetacoplan (APL-2) in Patients Diagnosed With Geographic Atrophy Secondary to Age Related Macular Degeneratio
Tests APL-3007, pegcetacoplan (APL-2), APL-3007, pegcetacoplan (APL-2), Placebo, Syfovre
For Geographic Atrophy Secondary to Age-related Macular Degeneration
Lesion-tailored, Fundus-controlled Perimetry in Geographic Atrophy (GA).
For Geographic Atrophy From Age-related Macular Degeneration
2 trials underway, not taking new volunteers
A Prospective, Multicenter, Open-Label, Observational Phase 4 Study to Evaluate Real-World Safety, Tolerability, and Treatment Patterns of Pegcetacoplan (Syfovre) in Patients With Geographic Atrophy Secondary to Age-Related Macular Degeneration
Tests Pegcetacoplan
An Open-Label, Nonrandomized, Multicenter Extension Study to Evaluate the Long-term Safety and Efficacy of Pegcetacoplan in Participants With C3 Glomerulopathy or Immune-Complex Membranoproliferative Glomerulonephritis
Tests Pegcetacoplan
For C3G, IC-MPGN, C3 Glomerulopathy, C3 Glomerulonephritis and Complement 3 Glomerulopathy
Pick a condition. One email when a new trial opens, a trial changes or the FDA decides, from any company, not only Apellis Pharmaceuticals. Unsubscribe anytime.
- Trial Alerts, when a new trial opens or a trial changes
- Disease Updates, FDA decisions and condition news, usually 1 or 2 emails a month
FDA-approved drugs from Apellis Pharmaceuticals
From the openFDA database. Tap a drug for side effects, dosing and help paying.
Empaveli pegcetacoplan
For Paroxysmal Nocturnal Hemoglobinuria and C3 Glomerulopathy. Approved May 14, 2021. Taken by subcutaneous route.
Syfovre pegcetacoplan
For Geographic Atrophy. Approved Feb 17, 2023. Taken by intravitreal route.
More about Apellis Pharmaceuticals
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.
Where Apellis Pharmaceuticals trials take place
The trials above run at 174 study sites in 18 countries. Tap a country to see its states, cities and hospitals.
Rare diseases Apellis Pharmaceuticals works on
Each disease page lists every open trial, not only Apellis Pharmaceuticals's, plus approved treatments.
Amyotrophic lateral sclerosis is a progressive neurodegenerative disease destroying motor neurons in the brain and spinal cord.
How common: About 5,000 new cases per year in the U.S.; CDC's National ALS Registry estimates about 33,000 Americans were living with ALS in 2022, a number projected to pass 36,000 by 2030
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.
How common: 1 to 2 new cases per million per year; approximately 5,000 to 10,000 affected individuals in the U.S
Cold agglutinin disease is a rare autoimmune hemolytic anemia caused by IgM antibodies that stick to red blood cells in the cold.
How common: About 16 per million people for primary CAD in a Norwegian population study, roughly 1 in 60,000, with about 1 new case per million people each year
Focal Segmental Glomerulosclerosis
Focal Segmental Glomerulosclerosis (FSGS) is a kidney disease characterized by scarring of some glomeruli, the tiny filtering units that remove waste from the blood.
How common: Approximately 7% of all kidney biopsies in the United States show FSGS; incidence is 0.2-1.7 cases per 100,000 person-years
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...
How common: About 1 million people in the U.S. are estimated to have geographic atrophy, the advanced dry form of age-related macular degeneration
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...
How common: About 2 new cases per 100,000 people each year in the U.S., and an estimated 199,000 to 208,000 Americans were living with IgA nephropathy in 2021 (Otsuka-funded study, Clinical Nephrology, 2025)
Lupus nephritis is kidney inflammation caused by systemic lupus erythematosus (SLE), in which the immune system attacks the kidneys' filters and, without treatment, can lead to kidney failure.
How common: About 21 per 100,000 people in a Minnesota population study in 2015 (Lupus Midwest Network, 2022)
Membranous nephropathy is a rare autoimmune kidney disease where the body's own antibodies attack proteins in the kidney's filtering units (glomeruli).
How common: About 12 new cases per million people each year in the U.S.; roughly 40,000-50,000 Americans living with the disease
Where to get support
Patient groups can explain a diagnosis, point you to specialists and tell you about trials before they open. Apellis Pharmaceuticals's own site is apellis.com, and our help paying for treatment page lists drugmaker programs and charity funds.
- National Kidney Foundation - C3GC3 Glomerulopathy and IgA Nephropathy and 1 more
- NephCure Kidney International - C3GC3 Glomerulopathy and Membranous Nephropathy
- The ALS AssociationAmyotrophic Lateral Sclerosis
- ALS Therapy Development InstituteAmyotrophic Lateral Sclerosis
- NIH GARD - Cold Agglutinin DiseaseCold Agglutinin Disease
- NORD - Cold Agglutinin DiseaseCold Agglutinin Disease
- Travere Therapeutics — Filspari FSGS ApprovalFocal Segmental Glomerulosclerosis
- National Kidney FoundationFocal Segmental Glomerulosclerosis