Empaveli FDA Approval History
Last updated by Judith Stewart, BPharm on July 30, 2025.
FDA Approved: Yes (First approved May 14, 2021)
Brand name: Empaveli
Generic name: pegcetacoplan
Dosage form: Injection
Company: Apellis Pharmaceuticals, Inc.
Treatment for: Paroxysmal Nocturnal Hemoglobinuria, C3 Glomerulopathy, Glomerulonephritis
Empaveli (pegcetacoplan) is a targeted C3 inhibitor for the treatment of paroxysmal nocturnal hemoglobinuria, and C3 glomerulopathy or primary immune-complex membranoproliferative glomerulonephritis.
- Empaveli is indicated:
- for the treatment of adult patients with paroxysmal nocturnal hemoglobinuria (PNH).
- for the treatment of adult and pediatric patients aged 12 years and older with C3 glomerulopathy (C3G) or primary immune-complex membranoproliferative glomerulonephritis (IC-MPGN), to reduce proteinuria. - PNH is a rare, chronic, life-threatening blood disorder characterized by the destruction of oxygen-carrying red blood cells through extravascular and intravascular hemolysis. Low hemoglobin levels can result in frequent transfusions and debilitating symptoms such as severe fatigue, hemoglobinuria, and dyspnea. C3G and IC-MPGN are rare and debilitating kidney diseases that can lead to kidney failure. Excessive C3 deposits are a key marker of disease activity, which can lead to kidney inflammation, damage, and failure.
- Empaveli is a targeted C3 therapy that works in the treatment of PNH, C3G and IC-MPGN by binding to complement protein C3 and its activation fragment C3b to regulate excessive activation of the complement cascade.
- Empaveli is administered subcutaneously twice weekly by subcutaneous infusion via a commercially available infusion pump or with the Empaveli Injector.
- The Empaveli product label carries a boxed warning for the increased risk of meningococcal and other serious infections caused by encapsulated bacteria. Because of this risk, Empaveli is available only through a restricted program under a Risk Evaluation and Mitigation Strategy (REMS).
- Common adverse reactions (incidence ≥10%)
- in patients with PNH include injection site reactions, infections, diarrhea, abdominal pain, respiratory tract infection, pain in extremity, hypokalemia, fatigue, viral infection, cough, arthralgia, dizziness, headache, and rash.
- in patients with C3G or primary IC-MPGN include infusion site reactions, pyrexia, nasopharyngitis, influenza, cough, and nausea.
Development timeline for Empaveli
Further information
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