NDASUBCUTANEOUSSOLUTIONPriority Review
Approved
May 2021
Lifecycle
Peak
Competitive Pressure
0/100
Clinical Trials
20
Data completeness
7/8 — Comprehensive
Priority Review
EMPAVELI (pegcetacoplan) by Apellis Pharmaceuticals is complement inhibitors [moa]. First approved in 2021.
Drug data last refreshed 7h ago · AI intelligence enriched 1w ago
AI summarySynthesized from FDA + DailyMed + ClinicalTrials.gov data
EMPAVELI (pegcetacoplan) is a complement C3 inhibitor administered subcutaneously for the treatment of paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder characterized by hemolysis. It works by blocking C3 activation in the complement cascade, preventing red blood cell destruction.
$25MPart D
Peak12.2 years to LOEProduct is at peak commercial maturity with modest Part D uptake (657 claims in 2023), indicating a niche market with stable but limited growth potential and focused commercial teams.
Mechanism of Action
Complement Inhibitors
Pharmacologic Class:
Complement Inhibitor
Indications
Indication data is being enriched from DailyMed and FDA labeling. Check back soon for approved therapeutic uses.
Clinical Trials (20)
Real-World Effectiveness and Safety of Pegcetacoplan in Patients With C3G or IC-MPG: A Multi-Country Study
Started Aug 2026
150 enrolled
C3 Glomerulopathy (C3G)Immune Complex Membranoproliferative Glomerulonephritis (IC-MPGN)
Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Adults at High Risk of Delayed Graft Function (DGF) Following Kidney Allograft Transplantation
Started May 2026
0Delayed Graft FunctionEnd Stage Renal DiseaseDeceased Donor Kidney Transplant
Study to Evaluate a Pegcetacoplan (Syfovre®) Prefilled Syringe
Started Oct 2025
44 enrolled
Geographic Atrophy Secondary to Age-related Macular Degeneration
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
Started Jun 2025
240 enrolled
Geographic Atrophy Secondary to Age-related Macular Degeneration
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
Started Sep 2023
300 enrolled
Geographic Atrophy
Worked on EMPAVELI at Apellis Pharmaceuticals? Share your interview experience or compensation data (+7 days Pro)
Data Sources
- Drug label: DailyMed / FDA
- Clinical trials: ClinicalTrials.gov
- Patent data: FDA Orange Book
- Spending data: CMS Medicare
The information on this page is for informational purposes only and should not be used as a substitute for professional medical advice. Drug information is sourced from FDA, DailyMed, and other government databases. Adverse event data from FAERS does not establish causation. Always consult a healthcare professional for medical decisions.