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FDA Approves Ravulizumab for Paroxysmal Nocturnal Hemoglobinuria

Gina Columbus @ginacolumbusonc
Published: Thursday, Dec 27, 2018

John Orloff, MD

John Orloff, MD

The FDA has approved ravulizumab-cwvz (Ultomiris) as an injection treatment of adult patients with paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder that leads to hemolysis.1,2

“We are proud to bring Ultomiris to patients suffering from this devastating disease less than a year after reporting our positive phase III data,” said John Orloff, MD, executive vice president and head of research and development at Alexion, in a press release. “Immediate and complete C5 inhibition with Ultomiris, sustained for 8 weeks, can provide meaningful benefits for patients and their families. Based on the totality of our compelling data from the largest phase III program ever conducted in PNH, we believe Ultomiris has the potential to become the new standard of care for patients with PNH.”

PNH is a rare acquired disorder that leads to hemolysis. Patients with PNH have episodes where red blood cells are prematurely destroyed, which may be triggered by stresses on the body. During these occurrences, severe anemia, profound fatigue, shortness of breath, intermittent episodes of dark colored urine, kidney disease or recurrent pain may occur. The disease is most often diagnosed in young adulthood, though it can occur at any age. Ravulizumab, a long-acting C5 complement inhibitor, is designed to prevent hemolysis from occurring in patients.

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