- 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.
Priority Review designation is granted to marketing applications for medicines that treat a serious condition and if approved, would provide a significant improvement in the safety or effectiveness of the treatment, prevention, or diagnosis of a serious condition. Pegcetacoplan was previously granted Fast Track designation by the FDA for the treatment of PNH.
Apellis plans to open an early access program (EAP) in the United States for pegcetacoplan for patients with PNH who are experiencing ongoing disease activity despite treatment with C5 inhibition. The EAP will be available for a limited time while the FDA is reviewing the pegcetacoplan NDA. EAPs are potential pathways for patients with life-threatening or serious diseases to access investigational therapies outside of clinical trials when no comparable or satisfactory alternative therapy options are available. More information on the EAP is available at https://apellis.com/for-patients/early-access-program/.
About the PEGASUS StudyThe PEGASUS study (APL2-302; NCT03500549) is a multi-center, randomized, active-comparator controlled Phase 3 study in 80 adults with paroxysmal nocturnal hemoglobinuria (PNH). The primary objective of this study was to establish the efficacy and safety of pegcetacoplan compared to eculizumab. Participants must have been on eculizumab (stable for at least three months) with a hemoglobin level of