Tag Archive for: hemophilia A

A U.S. federal court upheld a prior ruling in favor of Roche’s Genentech, finding that its blockbuster hemophilia treatment Hemlibra did not infringe on patent protections held by Takeda’s Baxalta.

BioMarin Pharmaceutical Inc. announced that an individual in Germany with severe hemophilia A was treated with Roctavian (valoctocogene roxaparvovec-rvox), marking the first time that the gene therapy has been given commercially in Europe.

Amid the impending drug pricing pressures from the Inflation Reduction Act, Roche is discontinuing a mid-stage hemophilia A gene therapy candidate and four other early-stage hopefuls.

After an initial rejection, BioMarin has finally secured the FDA’s approval for Roctavian, the first gene therapy in the U.S. for the most common form of the bleeding disorder.

The FDA will close the month of June with four target action dates, including one for an investigational gene therapy for a rare disease and another for an already top-selling eye disease treatment.

The FDA said late on Monday it needed more time to review a three-year analysis from the company’s ongoing late-stage study, which BioMarin submitted earlier this year, and will make a decision by June 30.

The FDA approved Sanofi and Sweden-based Sobi’s efanesoctocog alfa, now marketed as Altuviiio, to treat bleeding in adults and children with hemophilia A.

The FDA last year accepted the marketing application for the therapy, which is being developed in collaboration with Swedish drugmaker Sobi, and is expected to decide on an approval status by Feb. 28.

End-to-end vector manufacturing optimizes batch success and delivers more doses at lower cost.

BioMarin Pharmaceutical announced the FDA no longer plans to hold an advisory committee meeting to review its Biologics License Application (BLA) for Roctavian (valoctocogene roxaparvovec), an AAV gene therapy for adults with severe hemophilia A.