London: European Medicines Agency (EMA) recommends the use of Pfizer’s gene therapy. Rare bleeding disorder called Hemophilia BThis usually requires regular transfusions of blood-clotting proteins, the regulator said on Friday.

The regulator has recommended granting ‘conditional marketing authorisation’, to approve a medicine that addresses unmet medical needs of patients based on less extensive data than normally required.

The decision comes a few months after a one-time medical examination. regulatory approvals In the United States and Canada, where it is branded under the name Bakeway’s.

Any recommendation by the committee of EMA Medicinal products for human use (CHMP) has to be formally approved. The European CommissionWhich usually happens after a decision by the regulator.

The therapy costs $3.5 million in the US – the same as rival gene therapy Hemigenics from Australian drugmaker CSL Ltd.

In the European Union it will be sold under the brand name Dervectics.

People with hemophilia have a defect in a gene that controls the production of proteins called clotting factors, which can cause sudden and severe bleeding after an injury or surgery. mainly affects Male.

This therapy is designed to stimulate the production of a protein called Factor IX (FIX) by the patient’s own body, rather than intravenous infusions of FIX several times a week or month.

The recommendation is based on a late-stage trial that showed that after one year, a single dose of the therapy was as effective as a standard-of-care protein infusion, with bleeding completely eliminated in 60 percent of patients, compared with 29 percent of patients receiving infusions.

Pfizer said it would continue to monitor the long-term durability and safety of the treatment for 15 years.

Pfizer, citing the World Federation of Haemophilia, said more than 38,000 people worldwide suffer from haemophilia B. (Reporting by Sriparna Roy and Puyan Singh in Bengaluru; Editing by Shailesh Kuber)