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FDA approves Ultomiris for paroxysmal nocturnal hemoglobinuria
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The FDA today approved ravulizumab injection for the treatment of adults with paroxysmal nocturnal hemoglobinuria.
Paroxysmal nocturnal hemoglobinuria (PNH) — a rare and life-threatening blood disease — leads to the rupture or destruction of red blood cells, or hemolysis. Ravulizumab (Ultomiris, Alexion Pharmaceuticals) is a long-acting complement inhibitor that prevents hemolysis.
“The approval of Ultomiris will change the way that patients with PNH are treated,” 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, said in a press release. “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.”
Patients with PNH have frequent episodes where red blood cells are prematurely destroyed — sometimes triggered by stress, such as infections or physical exertion — during which patients may also experience severe anemia, profound fatigue, shortness of breath, intermittent episodes of dark-colored urine, kidney disease or recurrent pain.
This approval is based, in part, on a clinical trial of 246 treatment-naive patients randomly assigned ravulizumab or eculizumab (Soliris, Alexion Pharmaceuticals), the current standard of care for PNH.
Results showed ravulizumab and eculizumab appeared comparable in terms of the proportion of patients who did not receive a transfusion and the incidence of hemolysis as measured by the normalization of lactate dehydrogenase levels in patients’ blood.
Another study randomly assigned 195 patients with PNH who were clinically stable after having been treated with eculizumab for at least the past 6 months to be treated with ravulizumab or to continue eculizumab. Ravulizumab again appeared noninferior to eculizumab in terms of hemolysis and avoiding transfusion.
Common adverse events associated with ravulizumab included headache and upper respiratory infection.
Ravulizumab contains a boxed warning regarding risk for life-threatening meningococcal infections and sepsis. Health care providers should comply with recommendations for meningococcal vaccination in patients with complement deficiencies, and patients should be immunized with meningococcal vaccines at least 2 weeks prior to the first dose of ravulizumab.
Ravulizumab received priority review and orphan drug designation.