FDA backs Ultomiris for pediatric patients with rare blood disease

8 June 2021

The US Food and Drug Administration has approved Ultomiris (ravulizumab-cwvz) injection to treat patients aged one month and older with paroxysmal nocturnal hemoglobinuria (PNH), a rare, life-threatening blood disease, that is marketed by Alexion Pharmaceuticals (Nasdaq: ALXN).

Alexion plans to make Ultomiris available to pediatric patients in the USA immediately. A regulatory filing for Ultomiris in pediatric patients with PNH is under review in the European Union (EU).

PNH is characterized by red blood cell destruction, anemia (red blood cells unable to carry enough oxygen to tissues), blood clots, and impaired bone marrow function (not making enough blood cells). The disease affects 1-1.5 people per million. Approximately 10% of cases are among pediatric patients. PNH can be serious, with median survival of 10 years after diagnosis. However, some patients live for decades with only minor symptoms.

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