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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Are you sure it’s an inherited condition? First time I’ve heard that?
PNH is an acquired disease, NOT an inheriting disease, please check your sources.
PNH isn’t an inherited disease. It’s a rare disease with a genetic component but not inherited.