TFPI

FDA and EMA Accept Marstacimab Regulatory Submissions for the Treatment of Hemophilia A and B

Retrieved on: 
Monday, December 11, 2023
Biotechnology, FDA, Pharmaceutical, Health, Medicine, Safety, NYSE, Senior, PFE, Food, TFPI, Haemophilia, BLA, European Medicines Agency, European Commission, ASH, MAA, EMA, Civil service commission, Patient, Research and development, Research, Internal medicine, FIX, Rusyns, Exposition, Factor IX, Hemostasis, Development, Factor VIII, PDUFA, Society, Inc., Infection, Pharmaceutical industry, Pfizer

The European marketing authorization application (MAA) for marstacimab also passed validation and is currently under review by the European Medicines Agency (EMA).

Key Points: 
  • The European marketing authorization application (MAA) for marstacimab also passed validation and is currently under review by the European Medicines Agency (EMA).
  • If approved in the U.S. and EU, marstacimab is expected to become the first once-weekly subcutaneous treatment for people living with hemophilia B and the first treatment administered as a flat dose for people living with hemophilia A or B.
  • For appropriate patients living with hemophilia A and B, the goal of this treatment is to prevent potentially life-threatening bleeds with a once-weekly, subcutaneous flat-dose administration.
  • The submissions for marstacimab are based on efficacy and safety data from the Phase 3 BASIS trial ( NCT03938792 ).

Pfizer Announces Positive Marstacimab Results from Pivotal Phase 3 Hemophilia A and B Trial

Retrieved on: 
Tuesday, May 30, 2023
Biotechnology, FDA, Other Health, Health, General Health, Pharmaceutical, Oncology, Other Science, Research, Science, Clinical Trials, Inc., Factor IX, Bleeding, Factor VIII, PFE, ABR, Haemophilia, Pfizer, FIX, Assistant, McMaster University, Patient, Data, TFPI, Purpura, NYSE, Disease, Pharmaceutical industry

Pfizer Inc. (NYSE: PFE) today announced that the pivotal Phase 3 BASIS clinical trial ( NCT03938792 ) evaluating marstacimab has met its primary endpoints, having demonstrated statically significant and clinically meaningful effects.

Key Points: 
  • Pfizer Inc. (NYSE: PFE) today announced that the pivotal Phase 3 BASIS clinical trial ( NCT03938792 ) evaluating marstacimab has met its primary endpoints, having demonstrated statically significant and clinically meaningful effects.
  • “These results support the potential for marstacimab to become the first once-weekly non-factor treatment for people with hemophilia B and a treatment option that helps address the diverse needs of patients with hemophilia A or B without inhibitors.
  • The safety profile for marstacimab was consistent with Phase 1/2 results and treatment was generally well-tolerated.
  • Pfizer currently has three Phase 3 programs investigating novel treatment options for people living with hemophilia.