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FDA approves Empaveli for paroxysmal nocturnal hemoglobinuria

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The U.S. Food and Drug Administration (FDA) has approved pegcetacoplan (Empaveli; Apellis Pharmaceuticals), for the treatment of adults with paroxysmal nocturnal hemoglobinuria (PNH).

PNH is a rare acquired hematopoietic stem cell disorder in which red blood cells break apart prematurely.

Pegcetacoplan is approved for adult treatment-naïve patients with PNH and adult patients switching from the C5 inhibitors eculizumab (Soliris; Apellis Pharmaceuticals) and ravulizumab (Ultomiris; Alexion). This is the first approved targeted C3 therapy for use in PNH.

Approval was based on a multi-center, randomized, head-to-head Phase 3 study in 80 patients with PNH who had been on eculizumab and stable for ≥3 month, with a hemoglobin level of <10.5 g/dL at the screening visit. A total of 41 patients were treated dosed with 1080 mg of pegcetacoplan twice weekly in addition to their current dose of eculizumab during the 4-week run-in period. During the 16-week randomized, controlled period, patients were randomized to receive either 1080 mg of pegcetacoplan twice per week or their current dose of eculizumab (n = 39).

Pegcetacoplan demonstrated superiority to eculizumab for the change from baseline in hemoglobin level at week 16 with an adjusted mean increase of 3.84 g/dL of hemoglobin (P < 0.0001).

The most common serious adverse reaction in patients treated with Pegcetacoplan was infections (5%). The most common adverse reactions (≥10%) were injection site reactions (39%), infections (29%), diarrhea (22%), abdominal pain (20%), respiratory tract infection (15%), viral infection (12%), and fatigue (12%).

“As the first, FDA-approved targeted C3 therapy, Empaveli has the potential to redefine treatment for adults with PNH, including patients switching from any C5 inhibitor and treatment-naïve patients. Thank you to the clinical trial participants, PNH community, investigators, healthcare professionals, SFJ Pharmaceuticals, and more who helped contribute to this significant milestone,” said Cedric Francois, MD, PhD, co-founder and chief executive officer, Apellis, in a press release. “This approval represents a major scientific advancement as Empaveli ushers in the first new class of complement medicine in almost 15 years. We look forward to exploring the full potential of targeting C3 and continue to advance registrational programs of this therapy across multiple complement-driven diseases with high unmet need.”

Read the full press release here.