US approves gene therapy treatment for hemophilia

Washington: Pharmaceutical giant Pfizer has received US approval for a gene therapy against a form of hemophilia, a rare and inherited blood clotting...

Washington: Pharmaceutical giant Pfizer has received US approval for a gene therapy against a form of hemophilia, a rare and inherited blood clotting disorder, the company said Friday.

Beqvez, which is given as a single intravenous infusion, was shown in a clinical trial of 45 people to be better at preventing bleeding among adults with moderate to severe hemophilia B compared to regular infusions of a protein that promotes clotting, called protein factor IX (FIX).

The current standard of care is cumbersome, requiring infusions up to several times per week.

"Many people with hemophilia B struggle with the commitment and lifestyle disruption of regular FIX infusions, as well as spontaneous bleeding episodes, which can lead to painful joint damage and mobility issues," said Adam Cuker, director of the University of Pennsylvania's Comprehensive Hemophilia and Thrombosis Program, in a Pfizer statement.

"A one-time treatment with BEQVEZ has the potential to be transformative for appropriate patients by reducing both the medical and treatment burden over the long term."