The U.S. Food and Drug Administration (FDA) approved ravulizumab, a long-acting complement inhibitor that prevents hemolysis, for the treatment of adults with paroxysmal nocturnal hemoglobinuria (PNH).
The agency’s decision was based on data from two clinical trials: In the first, ravulizumab was noninferior to eculizumab (the current standard of care for PNH) in 246 patients with treatment-naïve PNH, with similar proportions of patients achieving transfusion-independence and experiencing hemolysis. In the second, ravulizumab was evaluated in a clinical trial of 195 patients with PNH who had been previously treated with eculizumab for at least the past six months; patients were randomized to either continue receiving eculizumab or start treatment with ravulizumab. Ravulizumab again was found to be noninferior to eculizumab.
“The approval of [ravulizumab] will change the way that patients with PNH are treated,” said Richard Pazdur, MD, director of the FDA’s Oncology Center of Excellence and acting director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research. “Prior to this approval, the only approved therapy for PNH required treatment every two weeks, which can be burdensome for patients and their families. [Ravulizumab] uses a novel formulation so patients only need treatment every eight weeks, without compromising efficacy.”
The most common adverse events associated with ravulizumab included headache and upper respiratory infection.
The prescribing information for ravulizumab includes a boxed warning about the risk of life-threatening meningococcal infections and sepsis and also advises health-care providers on meningococcal vaccination and monitoring for early signs of infection.
Ravulizumab was granted priority review and orphan drug designations.
Source: FDA news release, December 21, 2018.