The first gene therapies for sickle cell disease are now approved: Casgevy from Vertex Pharmaceuticals Casgevy and Lyfgenia from Bluebird Bio. Despite the same-day approval, key differences give one of these therapies commercialization advantages over the other.
Mirum Pharmaceuticals said its drug became the first US-approved therapy for treating itching in patients with rare liver disorder Alagille syndrome and would be available for distribution immediately.