MHRA nod for Hympavzi for hemophilia A or B

22 April 2025

The UK’s Medicines and Healthcare products Regulatory Agency (MHRA) has approved Hympavzi (marstacimab) to prevent or reduce bleeding in patients 12 years of age and older weighing at least 35kg with hemophilia A and B.

Developed by US pharma giant Pfizer (NYSE: PFE), this medicine is the first of its kind to work by targeting a protein in the blood clotting process, noted the MHRA. The drug was approved by the European Commission and the US Food and Drug Administration (FDA) late last year.
Hemophilia A and B are inherited bleeding disorders caused by a lack of factor VIII (haemophilia A) or factor IX (hemophilia B) which are proteins required for blood to clot and to stop bleeding.

This approval is supported by evidence from a main study that evaluated marstacimab in 116 adults and adolescents 12 years and older with severe hemophilia A or B without inhibitors. In the study, marstacimab significantly reduced the annualized bleeding rate (ABR) for treated bleeds during the 12-month active treatment period, demonstrating non-inferiority and statistical superiority compared to routine factor-based prophylaxis.  
The most common side effects of the medicine (which may affect more than 1 in 10 people) are headache, high blood pressure and itching (pruritus).

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