Benzinga | Oct 15, 2021 07:19AM EDT

Apellis Pharma Reports Positive CHMP Opinion For Aspaveli For Treatment Of Paroxysmal Nocturnal Hemoglobinuria

Apellis Pharmaceuticals, Inc. (NASDAQ:APLS) and Swedish Orphan Biovitrum AB (publ) (Sobi(tm)) (STO:SOBI) announced today that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency has adopted a positive opinion recommending the marketing authorization of Aspaveli(r) (pegcetacoplan) for the treatment of adults with paroxysmal nocturnal hemoglobinuria (PNH) who are anemic after treatment with a C5 inhibitor for at least three months. The positive opinion from the CHMP is now referred to the European Commission for an approval decision.

PNH is a rare, chronic, life-threatening blood disorder where uncontrolled complement activation leads to the destruction of oxygen-carrying red blood cells through intravascular and extravascular hemolysis. Characterized by persistently low hemoglobin, PNH can result in frequent transfusions and debilitating symptoms such as severe fatigue. Despite improvements in hemolytic activity with C5 inhibitor treatment, approximately 72% of C5 treated patients remain anemic, according to a retrospective and a cross-sectional study.1,2

"Building on our recent U.S. approval, the positive CHMP opinion moves us one step closer toward bringing this important treatment to patients across Europe," said Federico Grossi, M.D., Ph.D., chief medical officer, Apellis. "If approved, Aspaveli has the potential to redefine treatment for patients with PNH, so we look forward to the European Commission's final decision."

The positive opinion is based on the results from the head-to-head Phase 3 PEGASUS study, which evaluated the efficacy and safety of Aspaveli compared to eculizumab at 16 weeks in adults with PNH who had persistent anemia despite treatment with eculizumab. The results were published in the New England Journal of Medicine in March 2021.3

"Today's positive opinion by the CHMP is a significant milestone for people living with PNH across Europe," said Ravi Rao, head of research and development and chief medical officer, Sobi. "We hope to make a difference to the lives of people living with rare diseases, and if Aspaveli is approved by the European Commission, it will offer patients and treating physicians a new class of complement medicines for the treatment of PNH."

Aspaveli is the European trade name for pegcetacoplan, which is known as EMPAVELI(tm) in the United States where it is approved for the treatment of adults with PNH.