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FDA Approves Hympavzi For Hemophilia A Or B Without Inhibitors

October 11, 2024

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The FDA approved marstacimab-hncq for treatment of certain individuals with hemophilia.

The indication applies to routine prophylaxis to prevent or reduce frequency of bleeding episodes among adults or children aged 12 years or older who have hemophilia A without factor FVIII inhibitors. The indication also applies to use of the agent for people with hemophilia B without factor IX inhibitors.

The FDA approved marstacimab-hncq for treatment of certain individuals with hemophilia A or B.

Marstacimab-hncq (Hympavzi, Pfizer) is the first anti-tissue factor pathway inhibitor approved in the United States for treatment of hemophilia A or B, according to a Pfizer press release. It is administered subcutaneously on a once-weekly dosing schedule.

Marstacimab-hncq targets the Kunitz 2 domain of tissue factor pathway inhibitor (TFPI). The natural anticoagulation protein prevents the formation of blood clots and restores hemostasis, according to the release.

The FDA based approval on results of the phase 3 BASIS trial, which included adults and adolescents with hemophilia A or B without inhibitors.

After a 12-month active treatment period, marstacimab-hncq reduced the annualized bleeding rate for treated bleeds by 35% compared with routine prophylaxis and 92% compared with on-demand treatment.

The most common reported adverse events included injection site reactions, headache and pruritus.

"The approval of Hympavzi is a meaningful advancement for people living with hemophilia A or B without inhibitors for bleed prevention, with a generally manageable safety profile and a straightforward once-weekly subcutaneous administration," Suchitra S. Acharya, MD, director of the hemostasis and thrombosis center at Northwell Health and program head of the bleeding disorders and thrombosis program at Cohen Children's Medical Center, said in the release. "Hympavzi aims to reduce the current treatment burden by meeting an important need for these patients, including many who have required frequent, time-consuming intravenous treatment infusion regimens."

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Alhemo Indication Expanded To Include Hemophilia A/B Without Inhibitors

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Pfizer's HYMPAVZI Approved In EU For Treatment Of Severe Hemophilia A Or B Without Inhibitors

(RTTNews) - Drug major Pfizer Inc. (PFE) announced Wednesday that the European Commission or EC has granted marketing authorization for HYMPAVZI (marstacimab) to treat adults and adolescents with severe Hemophilia A or B without inhibitors.

The approval is to treat the routine prophylaxis of bleeding episodes in patients 12 years of age and older weighing at least 35 kg with severe hemophilia A without FIX inhibitors.

Hemophilia is a family of rare genetic blood diseases caused by a clotting factor deficiency.

HYMPAVZI offers a subcutaneous treatment option with a once-weekly dosing schedule and minimal preparation required for each individual administration.

The marketing authorization is based on results from the pivotal Phase 3 BASIS study (NCT03938792), demonstrating non-inferiority and superiority compared to routine prophylaxis in eligible patients with hemophilia A or B without inhibitors.

The company noted that HYMPAVZI is the first and only anti-tissue factor pathway inhibitor or anti-TFPI approved in the European Union for the treatment of hemophilia A or B. It is also the first hemophilia medicine approved in the EU to be administered via a pre-filled, auto-injector pen.

Pfizer said the marketing authorization is valid in all 27 EU member states, as well as in Iceland, Liechtenstein, and Norway. The EC approval follows the regulatory approval of HYMPAVZI in the United States in October.

For More Such Health News, visit rttnews.Com

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