FDA grants priority review to APL-2 for paroxysmal nocturnal hemoglobinuria
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The FDA granted priority review to pegcetacoplan for treatment of paroxysmal nocturnal hemoglobinuria, according to the agent’s manufacturer.
Pegcetacoplan (APL-2, Apellis Pharmaceuticals) is a targeted C3 therapy designed to regulate excessive activation of the complement cascade, which can contribute to the onset and progression of several serious diseases. It consists of a synthetic cyclic peptide conjugated to a polyethylene glycol polymer that binds to C3 and C3b.
The new drug application is based on results of the randomized phase 3 PEGASUS study. The study included 80 adults with paroxysmal nocturnal hemoglobinuria, a chronic life-threatening blood disorder characterized by destruction of oxygen-carrying red blood cells through extravascular and intravascular hemolysis.
The study met its primary endpoint, showing a significant improvement in hemoglobin levels at 16 weeks with pegcetacoplan compared with eculizumab (Soliris, Alexion). Researchers reported improvement in other measures — including normalization rates across key markers of hemolysis, as well as Functional Assessment of Chronic Illness Therapy fatigue score — in the pegcetacoplan group.
The two agents exhibited comparable safety profiles.
“For more than a decade, the only treatment options available for [paroxysmal nocturnal hemoglobinuria] have been C5 inhibitors,” Federico Grossi, MD, PhD, chief medical officer of Apellis, said in a company-issued press release. “Many patients still suffer from persistently low hemoglobin, often resulting in debilitating fatigue and frequent transfusions.
“The [priority review designation] takes us one step closer to bringing pegcetacoplan ... to patients in need,” Grossi added. “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 FDA previously granted fast track designation to pegcetacoplan for treatment of PNH. The agency is expected to make a decision on approval of pegcetacoplan for this indication by May 14.
Several clinical studies are underway to evaluate the agent for indications in hematology, neurology, nephrology and ophthalmology.
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