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Pfizer's bleeding disorder therapy succeeds in trial with patients with certain antibodies
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Pfizer's bleeding disorder therapy succeeds in trial with patients with certain antibodies
Jun 26, 2025 5:01 AM

June 26 (Reuters) - Pfizer's ( PFE ) hemophilia therapy,

Hympavzi, met the main goal of a late-stage study in patients

with certain types of antibodies, the U.S. drugmaker said on

Thursday.

The data comes months after the company decided to stop

global development and sale of its hemophilia gene therapy,

Beqvez, citing soft demand from patients and their doctors.

Hympavzi is a weekly injection and is approved for patients

with hemophilia A without factor VIII antibodies or inhibitors,

or hemophilia B without factor IX inhibitors.

In the trial, 48 patients treated with Hympavzi saw a 93%

reduction in annualized bleeding rate over a year. It also met

all secondary goals, including showing a benefit in spontaneous

bleeds and joint bleeds.

The company's statement did not disclose the total number of

patients involved in the trial.

People with hemophilia have a defect in a gene that

regulates the production of proteins called clotting factors,

causing spontaneous and severe bleeding following injuries or

surgery.

Pfizer ( PFE ) said patients with inhibitors require treatments that

are less burdensome.

The hemophilia market has been dominated by factor

replacement therapies for decades.

Around 20% of people with hemophilia A and 3% of people with

hemophilia B are unable to continue factor replacement therapies

as they develop antibodies and the therapies fail to prevent or

stop bleeding episodes, Pfizer ( PFE ) said.

Other treatments include Sanofi's bimonthly gene

therapy Qfitlia for people with or without inhibitors, Novo

Nordisk's daily Alhemo, and gene therapies such as

CSL's Hemgenix and BioMarin Pharmaceutical's ( BMRN )

Roctavia.

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