Apellis Announces Submission of Pegcetacoplan Marketing Applications to FDA and EMA for Patients with PNH
WALTHAM, Mass., Sept. 15, 2020 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (Nasdaq: APLS), a global biopharmaceutical company and leader in targeted C3 therapies, today announced that the company has submitted a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) and a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for pegcetacoplan for the treatment of paroxysmal nocturnal hemoglobinuria (PNH) in the United States and European Union, respectively.
“Pegcetacoplan has demonstrated its potential to elevate the standard of care in PNH and the submissions of the U.S. and EU marketing applications represent an important step in our efforts to bring the first targeted C3 therapy to people with PNH as quickly as possible,” said Federico Grossi, M.D., Ph.D., Chief Medical Officer of Apellis. “Building on our progress with the FDA and EMA, we are also proud that the TGA has granted pegcetacoplan orphan drug designation for the treatment of patients with PNH in Australia.”
The NDA and MAA submissions are based on results from the Phase 3 PEGASUS study, which met its primary endpoint, demonstrating the superiority of pegcetacoplan to eculizumab with a statistically significant improvement in hemoglobin levels at 16 weeks, as well as higher normalization rates across key markers of hemolysis and clinically meaningful improvement in FACIT-fatigue score. The safety profile of pegcetacoplan was comparable to eculizumab in the study. The FDA and EMA decisions on acceptance of the NDA and MAA submissions are expected in Q4 2020.
In another regulatory milestone, the Australian Therapeutic Goods Administration (TGA) granted orphan drug designation to pegcetacoplan in PNH. This designation in Australia is granted to therapies for serious rare diseases that have the potential to provide a significant benefit in comparison to approved treatments. Apellis plans to submit a marketing application in Australia in the fourth quarter of 2020.
About Pegcetacoplan (APL-2)
Pegcetacoplan is an investigational, targeted C3 inhibitor designed to regulate excessive complement activation, which can lead to the onset and progression of many serious diseases. Pegcetacoplan is a synthetic cyclic peptide conjugated to a polyethylene glycol polymer that binds specifically to C3 and C3b. Apellis is evaluating pegcetacoplan in several clinical studies including paroxysmal nocturnal hemoglobinuria (PNH), geographic atrophy (GA), cold agglutinin disease (CAD), and C3 glomerulopathy (C3G). Pegcetacoplan was granted Fast Track designation by the U.S. Food and Drug Administration (FDA) for the treatment of PNH and the treatment of GA. For additional information regarding our clinical trials, visit https://apellis.com/our-science/clinical-trials.