Pfizer has received the US Food and Drug Administration (FDA) approval for Hympavzi (marstacimab-hncq) to treat haemophilia A and haemophilia B without inhibitors.
Hympavzi is a rebalancing agent that targets the Kunitz 2 domain of tissue factor pathway inhibitor (TFPI), a natural anticoagulation protein that prevents the formation of blood clots.
The drug is indicated to prevent or reduce bleeding in individuals aged 12 years and above with haemophilia A without factor VIII inhibitors, or haemophilia B without factor IX inhibitors.
Pfizer said that Hympavzi is the first haemophilia medicine approved in the US, for administration through a pre-filled, auto-injector pen.
The drug can provide a subcutaneous treatment option with a once-weekly dosing schedule and minimal preparation required for each individual administration, said the US drugmaker.
Pfizer US chief commercial officer and executive vice president Aamir Malik said: “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 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 FDA approval of Hympavzi is supported by the results from BASIS, a global, open-label, multicentre Phase 3 study of Hympavzi compared to routine prophylaxis (RP) and on-demand (OD) treatment regimen.
The Phase 3 study enrolled 116 adolescent and adult participants with severe haemophilia A or moderately severe to severe haemophilia B with or without inhibitors.
In the study, Hympavzi reduced 35% annualised bleeding rate (ABR) for treated bleeds and 92% after a 12-month active treatment period, compared to RP and OD, respectively.
Hympavzi showed a safety profile that was consistent with Phase 1/2 results of the drug.
The most commonly reported adverse reactions in the study include injection site reactions, headache, and pruritus.
National Bleeding Disorders Foundation president and CEO Phil Gattone said: “The haemophilia community continually seeks advancements in care that can improve the quality of life for our community members.
“We greatly appreciate Pfizer’s innovative efforts in developing this novel treatment option that addresses some of the ongoing challenges faced by people with haemophilia A and B.
“The availability of this therapy represents a powerful step forward in advancing care for more individuals and families in the bleeding disorders community.”
