BLA Filing Accepted for Novel PNH Therapy

The US Food and Drug Administration (FDA) has accepted the Biologics License Application (BLA) for crovalimab, an investigational anti-C5 recycling monoclonal antibody developed by Genentech for the treatment of patients with paroxysmal nocturnal hemoglobinuria (PNH). The BLA acceptance was based on results from the pivotal phase III COMMODORE 2 trial. In the trial, crovalimab controlled PNH symptoms and was well tolerated.

“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, the Chief Medical Officer and Head of Global Product Development at Roche Group, the parent organization of Genentech.

FDA Accepts Crovalimab License Filing from Genentech

In patients with PNH, the complement system targets and destroys red blood cells, leading to symptoms including anemia, fatigue, and blood clots, and potentially to eventual kidney disease. PNH affects approximately 20,000 people globally and is primarily treated with complement component C5 inhibitors.

In COMMODORE 2, crovalimab was administered subcutaneously every four weeks in patients with PNH who had not previously received complement inhibitor therapy. Crovalimab was noninferior to the current standard of care, eculizumab, which is administered intravenously every two weeks. Safety findings were comparable, with adverse events reported in 78% of participants treated with crovalimab compared with 80% of participants treated with eculizumab.

The BLA for crovalimab also included findings from COMMODORE 1, which identified favorable benefits in patients with PNH switching from conventional C5 inhibitors. Additionally, crovalimab is undergoing ongoing evaluation in five phase III trials and three earlier-phase trials on PNH and other similar disorders.

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