Pfizer's hemophilia therapy reduces bleeding in late-stage study
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[May 31, 2023]
(Reuters) - U.S. drugmaker Pfizer Inc said on Tuesday late-stage
study data for its experimental hemophilia therapy showed superiority to
the current standard of care treatment in reducing bleeding rates in
patients.
The therapy, marstacimab, met its main trial goal, demonstrating
superiority to factor replacement therapies, with a 92% reduction in
bleeding in patients with severe hemophilia A and moderately severe to
severe hemophilia B, Pfizer said.
Hemophilia hampers the body's ability to make blood-clotting proteins,
leading to prolonged bleeding following injuries or surgery, and it
primarily affects males, according to government data.
The estimated prevalence in the United States is 12 cases per 100,000
males for hemophilia A and 3.7 cases per 100,000 males for hemophilia B.
The current standard of care requires regular infusion of the missing
proteins.
Pfizer's once weekly marstacimab is an anti-tissue factor pathway
inhibitor that could help initiate blood clotting.
Australian drugmaker CSL Ltd's gene therapy Hemgenix offers a long-term
solution for hemophilia B patients, but is among the world's most
expensive treatments.
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The Pfizer logo is pictured on their
headquarters building in the Manhattan borough of New York City, New
York, U.S., November 9, 2020. REUTERS/Carlo Allegri/File Photo/File
Photo/File Photo
Pfizer is developing at least two
other therapies for treating hemophilia. Last year in December, the
drugmaker met its main goal in a late-stage study for a hemophilia B
gene therapy.
The current trial of marstacimab only tested patients who did not
have any antibodies that stop the production of the blood-clotting
proteins called factor VIII or factor IX.
Pfizer is also testing marstacimab in patients with those
inhibitors, with data expected in late 2024.
(Reporting by Vaibhav Sadhamta and Aditya Samal in Bengaluru;
Editing by Shinjini Ganguli)
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