A new record

The FDA has approved the new most expensive drug in the world

Nikolay Sokolov, Farmvestnik

Food and Drug Administration (FDA) reported that it approved the first therapy against hemophilia B. The Hemgenix drug was developed by the Dutch biotech company UniQure. It will be sold by the Australian CSL. The estimated price in the United States will be $ 3.5 million. This will make the drug the most expensive in the world, more expensive than the recently approved therapy for beta-thalassemia Zynteglo, which costs $ 2.8 million.

Hemophilia B is caused by a deficiency or absence in the body of a critical protein called factor IX. It is necessary for the body to effectively coagulate blood.

In patients with hemophilia B, a mutation of the gene regulating the synthesis of factor IX occurs. As a result, the function of the gene is disrupted, and the factor is produced in insufficient quantities. Hemgenix is administered by infusion and is intended to replace the gene that encodes factor IX. The drug uses a viral vector to transfer the gene to the liver, where it is absorbed by cells and stimulates the production of factor IX.

The safety and efficacy of Hemgenix were evaluated in two studies involving 57 adult men aged 18 to 75 years with severe or moderate hemophilia B. The efficacy was calculated based on a decrease in the annual frequency of bleeding (CCM) in men. In the study, which involved 54 people, the subjects had an increase in the level of activity of factor IX, a decrease in the need for conventional preventive replacement therapy with factor IX and a decrease in CCM by 54% compared to the baseline level.

CSL has stated that it plans to bring Hemgenix to market as quickly as possible. In Europe, the drug is still being studied. European regulators recently approved a gene therapy from BioMarin Pharmaceutical for the more common hemophilia A.

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