FDA Approves New Treatment for Adults With Rare Blood Disease PNH


Ravulizumab (Ultomiris, Alexion) is the first long-acting complement inhibitor approved for the treatment of paroxysmal nocturnal hemoglobinura.

Officials with the FDA have approved ravulizumab (Ultomiris, Alexion) injection for the treatment of adults with paroxysmal nocturnal hemoglobinura (PNH), a rare and life-threatening blood disease, according to a press release.

PNH, a rare acquired disorder, leads to the rupture or destruction of red blood cells called hemolysis. Patients with the disease experience sudden, recurring episodes where red blood cells are prematurely destroyed, which results in symptoms such as severe anemia, profound fatigue, shortness of breath, intermittent episodes of dark colored urine, kidney disease, or recurrent pain. Chronic hemolysis can be devastating, with the potential to damage vital organs and cause premature death, according to Alexion.

Ravulizumab, a long-acting complement inhibitor that prevents hemolysis, uses a novel formulation to allow treatment every 8 weeks versus every 2 weeks with previously approved therapies.

“Ultomiris is a compelling new therapy for patients with PNH,” Ilene Weitz, MD, associate professor at the Keck School of Medicine at the University of Southern California, said in a statement. “It has met the high bar for efficacy and safety established by Soliris and has a 4 times longer dosing interval. I am particularly pleased by the positive data in patients transitioning from Soliris to Ultomiris without interruption, which is critical when you treat a devastating disease like PNH. This gives me confidence in recommending that patients switch therapy.”

The approval is based on data from 2 phase 3 studies, which included a total of 441 patients who were complement inhibitor-naïve or who had been on stable eculizumab (Soliris).

The first trial included 246 patients who previously had not been treated for PNH. Patients received either ravulizumab or eculizumab, the current standard-of-care. The study showed that ravulizumab was non-inferior to treatment with eculizumab, based on measures including hemolysis and avoiding transfusion.

Additionally, ravulizumab was studied in a trial of 195 patients with PNH who were clinically stable after having been treated with eculizumab for at least 6 months prior. This trial also showed that ravulizumab demonstrated non-inferiority to eculizumab.

Commonly reported adverse effects include headache and upper respiratory infection. The FDA also advises health care providers to use caution when administering to patients with any other systemic infection. Ravulizumab also includes a boxed warning to advise health care professional and patients about the risk of life-threatening meningococcal infections and sepsis.

Ravulizumab was previously granted priority review and orphan drug designations by the FDA.


FDA approves new treatment for adult patients with rare, life-threatening blood disease [news release]. FDA’s website. https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm629022.htm. Accessed December 21, 2018.

Alexion Receives Early FDA Approval for Ultomiris (Ravulizumab-Cwvz) in Adults With Paroxysmal Nocturnal Hemoglobinuria (PNH) [news release]. Alexion’s website. https://bit.ly/2R9pzkY. Accessed December 21, 2018.

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