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.

Go deeper with GlobalData

Data Insights

The gold standard of business intelligence.

Find out more

Related Company Profiles

View all

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.”

How well do you really know your competitors?

Access the most comprehensive Company Profiles on the market, powered by GlobalData. Save hours of research. Gain competitive edge.

Company Profile – free sample

Thank you!

Your download email will arrive shortly

Not ready to buy yet? Download a free sample

We are confident about the unique quality of our Company Profiles. However, we want you to make the most beneficial decision for your business, so we offer a free sample that you can download by submitting the below form

By GlobalData
Visit our Privacy Policy for more information about our services, how we may use, process and share your personal data, including information of your rights in respect of your personal data and how you can unsubscribe from future marketing communications. Our services are intended for corporate subscribers and you warrant that the email address submitted is your corporate email address.

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.