Washington: pharmaceutical giant Pfizer Has received We approval for a gene therapy against a form of hemophiliaIt is a rare and inherited blood clotting disorder, the company said on Friday.

Bekways, which is given as a single intravenous infusion, was shown in a clinical trial of 45 people to be better at stopping bleeding in adults with moderate to severe hemophilia B than a regular infusion of a protein called proteinase K. Better, which promotes clotting. factor ix (solve).

The current standard of care is cumbersome, requiring injections 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 problems,” said Adam Cukor, MD, director of Comprehensive Hemophilia at the University of Pennsylvania. Can.” In a statement from Pfizer, the thrombosis program.

“One-time treatment with BEQVEZ has the potential to be transformative for appropriate patients by reducing medical and treatment burden over the long term.”

Therapy was generally well tolerated, but a common side effect was elevation of liver enzymes, indicating liver inflammation that was not accompanied by external symptoms. Nevertheless, patients are advised to avoid alcohol for a year after treatment to prevent liver damage.

Patients will be followed for 15 years to gather more data.

Pfizer’s genetic therapy targets hemophilia B, the second most common form of the condition, which primarily affects men. According to the World Federation of Hemophilia, more than 38,000 people worldwide suffer from hemophilia B.

It works by infecting the body with a virus that has been modified to be harmless, which delivers a functional copy of the Factor IX gene to liver cells, instructing them to produce a protein that promotes clotting. Gives which is not otherwise present in the patient.

It received approval by Canada in January and is awaiting review by the European Medicines Agency.

It comes with a hefty list price of $3.5 million. But a Pfizer spokesperson said costs are likely to be lower for people with insurance. By comparison, the annual cost of current treatments “can range from more than $600,000 and up to $1.1 million,” while Bacvese is a one-time dose.