Hemophilia Treatment Companies

Latest Hemophilia Treatment Companies Update

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  May 2023: Pfizer Inc. declared that the primary endpoints of the critical Phase 3 BASIS clinical trial (NCT03938792) assessing marstacimab have been met; the trial's observed effects are both statistically significant and clinically meaningful. Marstacimab, an innovative anti-tissue factor pathway inhibitor (anti-TFPI) under investigation for the management of hemophilia A or B in individuals lacking Factor VIII (FVIII) or Factor IX (FIX) inhibitors, was administered once weekly at a subcutaneous loading dose of 300 mg without regard to weight in the trial. The findings of this study provide evidence that marstacimab has the capacity to serve as the initial once-weekly non-factor treatment option for individuals with hemophilia B. Additionally, it aids in meeting the varied requirements of patients with hemophilia A or B who do not require inhibitors. These requirements encompass the prevention of excessive or potentially fatal hemorrhages, as well as the reduction of treatment burden through subcutaneous administration once per week.

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  June 2023: The FDA approved BioMarin's Roctavian, a gene therapy for hemophilia A. Even getting the medication into the hands of patients does not anticipate to be a quick process. Roctavian (previously BMN270), the initial one-time treatment to receive approval from the FDA in this therapeutic category, was approved for patients aged 18 and older with the severe form of the inherited bleeding disorder. Biologic estimates that approximately 2,500 of the 6,500 adult patients in the United States would qualify for the medication. By delivering functional factor VIII DNA to liver cells in order to replace a deficient blood-clotting protein, Roctavian has the potential to eradicate the requirement for ongoing treatment for a minimum of five years. The infusion drug reduced the mean annualized factor VIII infusion rate by 99% and the mean annualized hemorrhage rate by 84% in Phase 3 clinical trials.

• Hoffmann-La Roche Ltd


• Octapharma


• CSL Behring


• Kedrion


• Sanofi


• Bayer AG


• Baxter


• Swedish Orphan Biovitrum AB


• Grifols International S.A


• Shire,