FDA Accepts Application for Novel Treatment of Paroxysmal Nocturnal Hemoglobinuria

The US Food and Drug Administration (FDA) has accepted a Biologics License Application (BLA) for crovalimab, a novel, investigational, anti-C5 recycling antibody developed to treat paroxysmal nocturnal hemoglobinuria (PNH).

“This filing acceptance reinforces the value of crovalimab, which was engineered to be recycled in the bloodstream, with the goal of offering a sustained response while reducing treatment burden,” said Levi Garraway, MD, PhD, chief medical officer and head of global product development at Genentech, the manufacturer of the drug. “Crovalimab could provide an option to self-administer as infrequently as every 4 weeks, thereby reducing clinic visits for people with this lifelong condition.”

PNH is a rare and life-threatening blood disorder in which red blood cells are destroyed by the body’s complement system. The condition affects approximately 20,000 people worldwide. It is characterized by symptoms such as anemia, fatigue, and blood clots and can lead to kidney disease. C5 inhibitors like  crovalimab have shown efficacy in treating PNH because they block part of the complement system cascade.

The FDA BLA acceptance was based on the findings of the randomized, phase 3 COMMODORE 2 study in patients with PNH who had not been previously treated with complement inhibitors. According to the results, crovalimab administered as subcutaneous injections every 4 weeks achieved disease control and was noninferior in terms of safety to eculizumab, a current standard-of-care treatment, given intravenously every 2 weeks. Crovalimab is being investigated in a broad clinical development program, which includes 5 ongoing phase 3 studies and 3 earlier-phase studies in PNH and similar disorders.