In April, BioMarin forecast that its new gene therapy Roctavian would bring in $50 million to $100 million in sales by the end of 2023. Instead, it’s collected just $3.5 million in revenue for a mere three patients.
SAN RAFAEL, Calif., Feb. 6, 2024 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN), a global biotechnology company dedicated to transforming lives through genetic discovery, today announced that new data on ROCTAVIAN™ (valoctocogene roxaparvovec-rvox) will be presented at the 2024 European Association for Haemophilia and Allied Disorders (EAHAD) Congress, Feb. 6-9, 2024.
SAN RAFAEL, Calif., Aug. 30, 2023 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN), a global biotechnology company dedicated to transforming lives through genetic discovery, today 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.
TEMPE, Ariz., July 11, 2023 /PRNewswire/ -- BioCareSD is excited to announce the addition of ROCTAVIAN™, RYSTIGGO®, and VYVGART® HYTRULO to their rapidly growing portfolio of life-saving specialty therapies. Each product recently received approval from the U.S. Food & Drug Administration (FDA). BioCareSD is part of a limited distribution network for each therapy.
ELK GROVE VILLAGE, Ill., July 11, 2023 /PRNewswire/ -- Orsini Specialty Pharmacy, a leading independent specialty pharmacy focused on rare diseases and gene therapies, announced today that BioMarin Pharmaceutical has selected it to dispense ROCTAVIAN™ (valoctocogene roxaparvovec-rvox). ROCTAVIAN is the first gene therapy for the treatment of severe Hemophilia A. For more information about ROCTAVIAN, see the Full Prescribing Information. Orsini's dedicated Gene Therapy Care team is trained and ready to accept new patients.
June 29 (Reuters) - The U.S. Food and Drug Administration on Thursday approved BioMarin Pharmaceutical's (BMRN.O) gene therapy for severe hemophilia A, the company said, giving patients with the inherited bleeding disorder an alternative to regular injections of missing blood proteins.
SAN RAFAEL, Calif., June 29, 2023 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN), a global biotechnology company dedicated to transforming lives through genetic discovery, today announced that the United States Food and Drug Administration (FDA) approved ROCTAVIAN™ (valoctocogene roxaparvovec-rvox) gene therapy for the treatment of adults with severe hemophilia A (congenital factor VIII (FVIII) deficiency with FVIII activity < 1 IU/dL) without antibodies to adeno-associated virus serotype 5 (AAV5) detected by an FDA-approved test.
The Food and Drug Administration on Thursday approved a gene therapy to treat people with hemophilia A, an inherited and rare bleeding disorder.
SAN RAFAEL, Calif., June 22, 2023 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN), a global biotechnology company dedicated to transforming lives through genetic discovery, today announced it will present new data from the ROCTAVIAN™ (valoctocogene roxaparvovec) gene therapy clinical trial program. These data will be shared this week in four oral presentations and two posters at the 31st Congress of the International Society on Thrombosis and Haemostasis (ISTH) in Montreal, Canada.
The company’s chief executive Jean-Jacques Bienaimé told the JP Morgan healthcare conference this week that it expects to sign several more contracts with German insurers in the coming weeks, and has started reimbursement negotiations with the authorities in France and Italy.