Fidanacogene elaparvovec, sold under the brand name Beqvez among others, is a gene therapy delivered via adeno-associated virus used for the treatment of hemophilia B (congenital Factor IX deficiency).
[1][5][8] Fidanacogene elaparvovec was approved for medical use in Canada in December 2023,[1] in the United States in April 2024,[9][10] and in the European Union in July 2024.
[6] 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.
[10] Fidanacogene elaparvovec was approved for medical use in Canada in December 2023,[1] in the United States in April 2024,[9] and in the European Union in July 2024.
[14] Fidanacogene elaparvovec partially restored factor IX production in preliminary studies.