Pfizer has announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) adopted a positive opinion for HYMPAVZI (marstacimab) to expand the approved indication to include adolescents and adults with haemophilia A or B who have developed inhibitors to factor replacement therapies.
Currently, HYMPAVZI is approved in the EU for routine prophylaxis of bleeding episodes in similar patients with severe haemophilia A or B without inhibitors.
The European Commission will review the CHMP recommendation for HYMPAVZI and is expected to make a final decision in the coming months.
If approved, this new indication will be valid in all 27 EU member states, as well as Iceland, Liechtenstein and Norway.
HYMPAVZI could provide improved bleeding protection compared to on-demand treatments.
It offers a well-tolerated, once-weekly subcutaneous injection without the need for routine lab monitoring, benefiting patients aged 12 and older with difficult-to-treat inhibitors.
Why this matters
Inhibitors, or antibodies, can develop in people with haemophilia, rendering factor replacement therapies ineffective.
These inhibitors are diagnosed through a blood test.
Among the more than 800,000 individuals worldwide with haemophilia A or B, about 20% of those with haemophilia A and three per cent with haemophilia B are unable to continue these therapies due to the development of inhibitors to FVIII and FIX, respectively.
Consequently, these therapies fail to prevent or stop bleeding episodes, especially in those resistant to immune tolerance induction therapy.
"People living with [haemophilia] with inhibitors continue to face significant unmet needs, with a treatment journey that can be complex and burdensome with limited options available today," said Dr Michael Vincent, Chief Inflammation & Immunology Officer, Pfizer.
This positive CHMP opinion for HYMPAVZI in adults and adolescents living with inhibitors brings us closer to offering a once-weekly subcutaneous treatment without routine lab monitoring—an important potential advance for a patient community in need across the European Union.
The company added in its statement that the US Food and Drug Administration (FDA) has granted Priority Review for a supplemental Biologics License Application for HYMPAVZI.
This application seeks to expand the approval for routine prophylaxis to reduce bleeding episodes in patients aged 6 years and older with haemophilia A or B, with or without inhibitors.
Currently, HYMPAVZI is approved for patients aged 12 and older with haemophilia A without FVIII inhibitors or haemophilia B without FIX inhibitors.
The FDA has set the action date for the second quarter of 2026.