The Food and Drug Administration has approved ravulizumab (Ultomiris) injection for the treatment of adult patients with paroxysmal nocturnal hemoglobinuria (PNH).
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“The approval of Ultomiris will change the way that patients with PNH are treated. Prior to this approval, the only approved therapy for PNH required treatment every 2 weeks, which can be burdensome for patients and their families. Ultomiris uses a novel formulation so patients only need treatment every 8 weeks, without compromising efficacy,” Richard Pazdur, MD, director of the FDA’s Oncology Center of Excellence, said in a press release from the agency.
Patients with PNH, a rare disorder, lack a protein which protects red blood cells from being destroyed in the immune system. Episodes can be triggered by stresses on the body such as infection or physical exertion, and symptoms include severe anemia, profound fatigue, shortness of breath, intermittent episodes of dark-colored urine, kidney disease, or recurrent pain.
FDA approval for ravulizumab is based on results from a pair of clinical trials. In the first, 246 treatment-naive PNH patients received either ravulizumab or eculizumab, the current standard of care; ravulizumab was noninferior, with no patients undergoing a transfusion and all patients having similar incidence of hemolysis. In the second trial, 195 patients who had clinically stable PNH after receiving eculizumab for 6 months were randomized to receive ravulizumab or continue eculizumab; again, ravulizumab was noninferior.
The most common adverse events associated with ravulizumab were headache and respiratory tract infection. Caution is recommended when prescribing ravulizumab to patients with any type of infection.
Find the full press release on the FDA website.