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).[1][5][6]
It was approved for medical use in Canada in December 2023,[1] and in the United States in April 2024.[7][8]
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.[5][7]
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.[13] The applicant for this medicinal product is Pfizer Europe MA EEIG.[13][14]
^George, Lindsey A.; Sullivan, Spencer K.; Giermasz, Adam; Ducore, Jonathan M.; Teitel, Jerome M.; Cuker, Adam; et al. (2 December 2016). "Spk-9001: Adeno-Associated Virus Mediated Gene Transfer for Hemophilia B Achieves Sustained Mean Factor IX Activity Levels of >30% without Immunosuppression". Blood. 128 (22): 3.
doi:
10.1182/blood.V128.22.3.3.
ISSN0006-4971.
^
ab"Durveqtix EPAR". European Medicines Agency. 30 May 2024. Retrieved 31 May 2024. Text was copied from this source which is copyright European Medicines Agency. Reproduction is authorized provided the source is acknowledged.
Clinical trial number NCT03307980 for "Long-term Safety and Efficacy Study and Dose-Escalation Substudy of PF 06838435 in Individuals With Hemophilia B" at
ClinicalTrials.gov
Clinical trial number NCT03587116 for "A Study to Evaluate Prospective Efficacy and Safety Data of Current FIX Prophylaxis Replacement Therapy in Adult Hemophilia B Subjects (FIX:C≤2%) or Current FVIII Prophylaxis Replacement Therapy in Adult Hemophilia A Subjects (FVIII:C≤1%)" at
ClinicalTrials.gov
Clinical trial number NCT03861273 for "A Study to Evaluate the Efficacy and Safety of Factor IX Gene Therapy With PF-06838435 in Adult Males With Moderately Severe to Severe Hemophilia B (BENEGENE-2)" at
ClinicalTrials.gov
Clinical trial number NCT05568719 for "Safety and Effectiveness of Giroctocogene Fitelparvovec or Fidanacogene Elaparvovec in Patients With Hemophilia A or B Respectively" at
ClinicalTrials.gov