BioMarin Pharmaceutical Inc. announced that the U.S. Food and Drug Administration accepted for Priority Review the Biologics License Application to the FDA for the company’s investigational AAV5 gene therapy, valoctocogene roxaparvovec, for adults with hemophilia A.
BioMarin Pharmaceutical Inc. announced the submission of a Biologics License Application (BLA) to the U.S. Food and Drug Administration for the company’s investigational AAV gene therapy valoctocogene roxaparvovec for adults with hemophilia A.
Swiss pharma giant Roche’s 10-month journey to complete the acquisition of the gene therapy company Spark Therapeutics was finalized.
Bayer announced a three-year collaboration agreement with Children’s Hospital of Philadelphia (CHOP) for the discovery and development of small molecules to develop a first-in-class oral non-replacement therapy for the treatment of hemophilia A and B.
Cambridge, Mass.-based bluebird bio and Bagsvaerd, Denmark-based Novo Nordisk agreed to develop next-generation genome editing therapies for genetic diseases, including hemophilia.
The U.S. Food and Drug Administration approved Octapharma USA’s Wilate for the treatment of adults and adolescents with hemophilia A for routine prophylaxis to reduce the frequency of bleeding episodes and on demand treatment and control of bleeding episodes.
BioMarin’s Hemophilia A Gene Therapy Shows Promise in Clinical Trial
Analysts, Breakthrough Therapy Status, Clinical Data, Clinical Trials, European Medicines Agency, European Medicines Agency (EMA), Factor VIII Inhibitors, FDA, Gene Therapy, Hemophilia A, Orphan Drug Designation, Priority Medicines (PRIME) access, R&DSan Rafael, Calif.-based BioMarin Pharmaceutical announced positive Phase III clinical trial results for the company’s gene therapy, valoctocogene roxaparvovec, for adults with hemophilia A.
Bayer plans to build a Cell Culture Technology Center in Berkeley, Calif., investing $150 million in the project. The center will focus on developing biologics, an area where Bayer lags behind the company’s competitors.
The EU’s CHMP adopted a positive opinion for Hemlibra (emicizumab) for routine prophylaxis of bleeding episodes in people with hemophilia A with factor VIII inhibitors.
The European Commission granted Marketing Authorization for Shire’s ADYNOVI [Antihemophilic Factor (Recombinant), PEGylated], for on-demand and prophylactic use in patients 12 years and older living with hemophilia A.
