Fidanacogene elaparvovec

Fidanacogene elaparvovec, sold under the brand name Beqvez, is a gene therapy delivered via adeno-associated virus used for the treatment of Hemophilia B (congenital Factor IX deficiency).

It was approved for medical use in Canada in December 2023, and in the United States in April 2024.

Medical uses
In the US, fidanacogene elaparvovec is indicated for the treatment of adults with moderate to severe hemophilia B (congenital factor IX deficiency) who currently use factor IX prophylaxis therapy; or have current or historical life-threatening hemorrhage; or have repeated, serious spontaneous bleeding episodes; and do not have neutralizing antibodies to adeno-associated virus serotype Rh74var (AAVRh74var) capsid as detected by an FDA-approved test.

Research
It partially restores factor IX production in preliminary studies.

Legal status
In May 2024, the Committee for Medicinal Products for Human Use of the European Medicines Agency adopted a positive opinion, recommending the granting of a conditional marketing authorization for the medicinal product Durveqtix, intended for the treatment of severe and moderately severe hemophilia B. The applicant for this medicinal product is Pfizer Europe MA EEIG.