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Alexion Pharmaceuticals secured approval Friday from the Food and Drug Administration for a new drug called Ultomiris that will be used to treat an ultra-rare blood disease and ultimately replace the biotech’s current blockbuster, Soliris.

The initial Ultomiris approval granted by FDA is to treat patients with paroxysmal nocturnal hemoglobinuria, or PNH — a rare, inherited disease in which red blood cells are destroyed by over-activation of the complement system, a part of the body’s immune system.


Ultomiris is administered via an intravenous infusion every eight weeks, making it more convenient for patients than the every-two-week dosing schedule of Soliris, already one of the most expensive drugs in the world. Ultomiris will cost slightly less, Alexion said.

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