Roctavian’s approval was a scientific milestone, the culmination of years of research developing a genetic medicine for the chronic blood disease hemophilia A.
Its launch, which began in Europe in 2022 and the U.S. last year, has been a focus for analysts and investors, many of whom viewed the product as a blockbuster-to-be. As a one-time, long-lasting treatment, Roctavian is billed as an alternative to the chronic therapies people with hemophilia A use to prevent bleeding.