Pfizer’s gene therapy cuts hemophilia A bleeding rate in late-stage trial

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Pfizer hemophilia gene therapy

(Reuters) -Pfizer’s hemophilia gene therapy significantly reduced annual bleeding episodes in patients with this rare disorder. The Pfizer hemophilia gene therapy outperformed the current standard treatment. The company reported these results from a late-stage study on Wednesday.

The data takes Pfizer one step closer to securing U.S. regulatory approval for a second gene therapy to treat hemophilia. #PfizerHemophiliaGeneTherapy

If approved, Pfizer’s hemophilia A gene therapy will compete with BioMarin Pharmaceutical’s one-time treatment Roctavian, which was approved in the U.S. last year and is priced at $2.9 million.

Currently, the standard-of-care treatment for hemophilia A patients is to undergo routine replacement of the clotting protein called Factor VIII.

Data from the 75-patient pool showed that 84% of patients who received Pfizer’s gene therapy had Factor VIII levels above 5% at 15 months post-infusion. The company reported these results.

People with hemophilia have a fault in a gene that regulates the production of clotting factors. This fault leads to spontaneous bleeding and severe bleeding from injuries or surgery. #PfizerHemophiliaGeneTherapy

Pfizer’s gene therapy marks a major step forward in treating hemophilia A. As the Pfizer hemophilia gene therapy continues to show promising results, it could revolutionize patient care. This innovation provides a new standard for managing this challenging condition.

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