Apellis Announces FDA Acceptance and Priority Review of the New Drug Application for Pegcetacoplan for the Treatment of PNH
- PDUFA target action date is May 14, 2021
- FDA has stated that it is not currently planning to hold an advisory committee meeting to discuss the application
- Pegcetacoplan demonstrated superiority to eculizumab in improving hemoglobin levels in Phase 3 PEGASUS head-to-head study as well as substantial improvements in other clinical measures
Apellis plans to open an early access program in the United States for pegcetacoplan for people living with PNH
WALTHAM, Mass., Nov. 16, 2020 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals, Inc. (Nasdaq: APLS), a global biopharmaceutical company and leader in targeted C3 therapies, today announced that the U.S. Food and Drug Administration (FDA) has accepted and granted Priority Review designation for the New Drug Application (NDA) for pegcetacoplan for the treatment of paroxysmal nocturnal hemoglobinuria (PNH). The Prescription Drug User Fee Act (PDUFA) target action date is May 14, 2021. The FDA has stated that it is not currently planning to hold an advisory committee meeting to discuss the application.
“For more than a decade, the only treatment options available for PNH have been C5 inhibitors, and many patients still suffer from persistently low hemoglobin, often resulting in debilitating fatigue and frequent transfusions. The NDA priority review takes us one step closer to bringing pegcetacoplan, a targeted C3 therapy with the potential to redefine PNH treatment, to patients in need,” said Federico Grossi, M.D., Ph.D., chief medical officer of Apellis. “The data in the application validate the broad potential of targeting C3, and we continue to advance several registrational studies in serious diseases with few or no treatments.”
The NDA submission is based on results from the head-to-head 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. The data also demonstrated higher normalization rates across key markers of hemolysis and a clinically meaningful improvement in Functional Assessment of Chronic Illness Therapy (FACIT)-fatigue score. The safety profile of pegcetacoplan was comparable to eculizumab in the study.
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