Alexion, the rare disease unit of AstraZeneca (AZ; NASDAQ: AZN) based in the UK, has secured regulatory approval from the US Food and Drug Administration (FDA) for its Factor D inhibitor Voydeya (danicopan). The drug is approved as an add-on therapy to ravulizumab or eculizumab, which are marketed as Ultomiris and Soliris by AstraZeneca respectively, for the treatment of extravascular hemolysis (EVH) associated with paroxysmal nocturnal hemoglobinuria (PNH) in adults. It is estimated that 10% to 20% of PNH patients experience clinically significant EVH.
The FDA’s decision is supported by the successful outcomes of a Phase III study, which achieved its primary endpoint, showing a significant change in hemoglobin levels from baseline to week 12. The study also met secondary endpoints, including transfusion avoidance and improvements in fatigue scores. Voydeya demonstrated good tolerability among patients.
In addition to its US approval, Voydeya has received approval in Japan and is currently under review in other global markets, with a priority review status in Europe.- Flcube.com
