Apellis Pharmaceuticals receives FDA approval of EMPAVELI® (pegcetacoplan)

We're thrilled to celebrate our client, Apellis Pharmaceuticals, on their recent FDA approval of EMPAVELI® (pegcetacoplan) for the treatment of C3G and IC-MPGN — a major milestone for patients facing these rare and challenging kidney diseases. This approval reflects not only the strength of Apellis’s science, but their unwavering commitment to advancing targeted complement therapies. At GSS, we're proud to partner with companies like Apellis who are pushing the boundaries of innovation and making real impact in the lives of patients. #FDAApproval #Biotech #RareDisease #GSSPartners #LifeSciencesTalent

2021 · U.S. Food and Drug Administration

FDA Approves New Treatment for Adults with Serious Rare Blood Disease

Empaveli (pegcetacoplan) is approved for adults with paroxysmal nocturnal hemoglobinuria (PNH) and is the first PNH treatment that binds to complement protein C3.

PNH is a rare, life-threatening blood disease marked by red blood cell destruction, anemia, and blood clots. In a study of 80 patients with PNH and anemia who had been taking eculizumab, patients switched to Empaveli saw average hemoglobin rise by 2.4 g/dL over 16 weeks, while those who stayed on eculizumab saw an average decrease of 1.5 g/dL.

Empaveli is available only through a restricted program (REMS) because of the risk of serious infections, including life-threatening meningococcal infections. The approval was granted to Apellis Pharmaceuticals.

Read the FDA announcement

Source: U.S. Food and Drug Administration. Reflects the approval as announced in 2021; see the current prescribing information for up-to-date indications and safety information.