The European Commission (EC) has granted marketing authorisation for Pfizer‘s anti-tissue factor pathway inhibitor (anti-TFPI), HYMPAVZI (marstacimab), to treat prophylaxis of bleeding incidents in individuals aged 12 years and above weighing a minimum of 35kg with severe haemophilia A without factor IX (FIX) inhibitors.
HYMPAVZI is the first anti-TFPI approved in the EU for this indication and the first therapy to be administered through a pre-filled auto-injector pen.
The authorisation is based on outcomes from the Phase III BASIS trial. This pivotal study demonstrated the safety and efficacy of marstacimab in decreasing the annualised bleeding rate by 35% compared to routine prophylaxis.
The authorisation extends to all 27 EU member states, along with Liechtenstein, Iceland, and Norway.
Pfizer chief international commercial officer and executive vice-president Alexandre de Germay stated: “HYMPAVZI offers a first-in-class treatment option for people living with haemophilia, a disease that often leads to recurring joint bleeds and can impact daily activities as simple as climbing stairs.
“This approval builds on Pfizer’s more-than-four-decade commitment to improve the standard of care in haemophilia, and we look forward to delivering this medicine that reduces bleeds as compared to factor prophylaxis and, importantly, requires limited preparation, meeting a key need for eligible patients.”
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By GlobalData
The company also received approvals in Europe and the US for its haemophilia B gene therapy BEQVEZ (fidanacogene elaparvovec).
HYMPAVZI is a once-weekly subcutaneous treatment option for individuals with severe haemophilia A or B without FIX inhibitors.
In October 2024, the US Food and Drug Administration (FDA) granted approval for HYMPAVZI to treat haemophilia.
The genetic blood disorder is caused by clotting factor deficiency and affects more than 800,000 individuals worldwide.
