Alexion's Ultomiris Gets Early FDA Nod For Rare Blood Disorder

 | Dec 23, 2018 09:26PM ET

Alexion Pharmaceuticals, Inc. (NASDAQ:ALXN) announced that the FDA has approved Ultomiris (ravulizumab-cwvz/ALXN1210) for the treatment of adult patients with paroxysmal nocturnal hemoglobinuria (PNH), well ahead of its action date set for Feb 18, 2019. PNH, a rarely acquired disorder, causes rupture or destruction of red blood cells called hemolysis and can induce a wide range of debilitating symptoms and complications including blood clotting.

Ultomiris is the first and the only long-acting C5 complement inhibitor to get an approval for the above indication. It is to be administered every eight weeks. The nod for Ultomiris is quite compelling for patients with PNH as they no longer have to go for twice-a-week infusions but can now settle for just six to seven infusions a year.

Share price of Alexion has decreased 20.9% year to date, narrower than the Zacks Investment Research

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