The Food and Drug Administration has granted Priority Review to pegcetacoplan (Apellis Pharmaceuticals) for the treatment of paroxysmal nocturnal hemoglobinuria (PNH).

Pegcetacoplan is a synthetic cyclic peptide conjugated to a polyethylene glycol polymer that inhibits the complement cascade centrally at C3 and C3b. The NDA is supported by data from the phase 3 PEGASUS study that assessed the efficacy and safety of pegcetacoplan in 80 adults with PNH. Patients were randomized to receive either pegcetacoplan (n=41) or eculizumab (n=39) for 16 weeks. The primary end point was the change from baseline in hemoglobin level to week 16. Key secondary end points included transfusion avoidance, absolute reticulocyte count and Functional Assessment of Chronic Illness Therapy (FACIT)-fatigue score.

Results showed that pegcetacoplan met the primary end point demonstrating superiority to eculizumab with a statistically significant improvement in hemoglobin levels at week 16 (P <.0001). On key secondary end points, pegcetacoplan was found to be noninferior to eculizimab. Additionally, the safety profile of pegcetacoplan was observed to be comparable to eculizumab.

Continue Reading

A Prescription Drug User Fee Act (PDUFA) target action date of May 14, 2021 has been set for this 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,” said Federico Grossi, MD, PhD, 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 Company is also investigating pegcetacoplan for the treatment of geographic atrophy, autoimmune hemolytic anemia, and glomerulopathies.

For more information visit


Apellis announces FDA acceptance and Priority Review of the New Drug Application for pegcetacoplan for the treatment of PNH. [press release]. Waltham, MA: Apellis Pharmaceuticals, Inc; November 16, 2020. 

This article originally appeared on MPR