FDA approves new Alexion’s therapy targeting paroxysmal nocturnal hemoglobinuria

Biotech Alexion has issued a press release announcing that FDA has approved, two months earlier than scheduled, its ALXN1210 therapy for the treatment of paroxysmal nocturnal hemoglobinuria (PNH), a rare condition. The drug will be marketed as Ultomiris, the company has added.

The new therapy is expected to replace Alexion’s Soliris, an expensive drug launched over 11 years ago and still the only solution to PNH.

PNH is a rare disorder characterized by destruction of blood cells,  corpuscular hemolytic anemia, bone marrow disorders and frequent thrombosis. It can appear at any age, but more frequently in young adults, and is estimated to affect 1/500,000 individuals.  Symptoms vary and include hemolytic anemia, large vessel thrombosis (affecting liver, abdominal, brain and skin veins), and moderate-serious hematopoiesis disorders, which can cause pancytopenia.

(Source: Alexion)