The FDA approval of HYMPAVZI was supported by the Phase III BASIS clinical trial. Credit: Andrey_Popov/Shutterstock.

Pfizer has announced the approval of HYMPAVZI (marstacimab-hncq) by the US Food and Drug Administration (FDA) for routine prophylaxis in patients with haemophilia A and B.

HYMPAVZI, a rebalancing agent that targets the tissue factor pathway inhibitor’s (TFPI) Kunitz 2 domain, offers a once-weekly subcutaneous treatment option. TFPI is a natural anticoagulant protein that helps to prevent blood clot formation and restore haemostasis.

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The approval of HYMPAVZI provides a new treatment that can decrease the intensity of bleeding episodes in patients aged 12 years and older. It marks a significant advancement in treatment options available for individuals with these rare genetic blood disorders without using inhibitors.

The new therapy is the first and only anti-TFPI approved in the US and can be administered using a pre-filled auto-injector pen.

Pfizer executive vice-president and US chief commercial officer Aamir Malik stated: “HYMPAVZI is Pfizer’s second haemophilia treatment to receive FDA approval this year and is the latest meaningful scientific advancement in our more than 40-year commitment to improve care for people living with haemophilia.

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 “We look forward to launching this latest medical breakthrough and to now offer[ing] three distinct classes of haemophilia medicines – an anti-TFPI, gene therapy and recombinant factor treatments – that can meet the unique treatment needs of a wide range of patients.”

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The Phase III BASIS trial, which supported the approval, demonstrated a significant reduction in the annualised bleeding rate for treated bleeds.

The study showed a 35% and 92% decrease in bleeding compared to routine prophylaxis and on-demand treatment, respectively, after a 12-month treatment period.

The European Medicines Agency’s  (EMA) Committee for Medicinal Products for Human Use (CHMP) has also adopted a positive opinion for HYMPAVZI for the given indication.

In early 2024, Pfizer received regulatory approvals for its one-time haemophilia B gene therapy, BEQVEZ (fidanacogene elaparvovec-dzkt) in the US.