e-Pharmatimes

With AstraZeneca’s dominance in the rare blood disease paroxysmal nocturnal hemoglobinuria (PNH) threatened by the recent approval of the first pill on the market for the condition, Novartis’ Fabhalta, AstraZeneca has answered in the United States for its add-on oral treatment in the indication. The FDA has signed off on Voydeya (danicopan), a factor D inhibitor, to treat PNH patients with extravascular hemolysis (EVH), a condition that causes red blood cell destruction outside of the blood vessels. The nod comes three months after the Japanese Ministry of Health, Labour and Welfare (JHLW) became the first regulator worldwide to endorse the therapy. Voydeya works as an add-on for AZ’s pair of PNH standard-of-care blockbusters—Soliris, which has been on the market for 17 years and generated $3.2 billion in sales in 2023, and its follow-on Ultomiris, which was approved in 2018 and accounted for $3 billion in sales last year. AZ said that 10% to 20% of those who use C5 inhibitors such as