Haemophilia treatment with new delivery method approved

The first anti-tissue factor pathway inhibitor (anti-TFPI) treatment for haemophilia A or B in the US has been approved by the US Food and Drug Administration (FDA). Hympavzi (marstacimab-hncq) is authorised for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in individuals 12 years of age and over with haemophilia A or haemophilia B without inhibitors.

Hympavzi “provides patients with haemophilia a new treatment option that is the first of its kind to work by targeting a protein in the blood clotting process,” noted Dr Ann Farrell, Director of the Division of Non-Malignant Hematology in the FDA’s Center for Drug Evaluation and Research.

FDA explained that Hympavzi reduces rather than replaces the clotting factor, therefore increasing the amount of thrombin, an enzyme that is essential for blood clotting.

What led to approval of the haemophilia treatment Hympavzi?

Findings from the Phase III BASIS trial supported authorisation of Hympavzi. The treatment was shown to reduce the annualised bleeding rate for treated bleeds by 35 percent and 92 percent after 12 months of active treatment. This was in comparison to routine prophylaxis and on-demand treatment, respectively, according to Pfizer.

“The approval of Hympavzi is a meaningful advancement for people living with haemophilia A or B without inhibitors for bleed prevention, with a generally manageable safety profile and a straightforward once-weekly subcutaneous administration,” commented Dr Suchitra Acharya, Director, Hemostasis and Thrombosis Center Northwell Health, Program Head, Bleeding Disorders and Thrombosis Program, Cohen Children’s Medical Center.

“Hympavzi is Pfizer’s second haemophilia treatment to receive FDA approval this year,” shared Aamir Malik, Chief US Commercial Officer and Executive Vice President, Pfizer. Malik added that Pfizer now has three classes of treatments for haemophilia: an anti-TFPI, gene therapy, and recombinant factor treatments.