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.
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.
“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.”
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.
