Fidanacogene elaparvovec

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Fidanacogene elaparvovec
Gene therapy
VectorAdeno-associated virus
Clinical data
Trade namesBeqvez
Other namesSPK-9001
Biosimilarsfidanacogene elaparvovec-dzkt
License data
Routes of
administration		Intravenous
ATC code
  • None
Legal status
Legal status
Identifiers
CAS Number
DrugBank
UNII

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][7]

It was approved for medical use in Canada in December 2023,[1] and in the United States in April 2024.[6][8]

Medical uses[edit]

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][6]

Research[edit]

It partially restores factor IX production in preliminary studies.[9][10][11][12]

References[edit]

  1. ^ a b c "Beqvez Product information". Health Canada. 22 October 2009. Archived from the original on 3 March 2024. Retrieved 3 March 2024.
  2. ^ "Notice: Multiple additions to the Prescription Drug List (PDL) [2024-02-28]". Health Canada. 28 February 2024. Archived from the original on 2 March 2024. Retrieved 2 March 2024.
  3. ^ "Details for: Beqvez". Health Canada. 27 December 2023. Archived from the original on 3 March 2024. Retrieved 3 March 2024.
  4. ^ "Regulatory Decision Summary for Beqvez". Drug and Health Products Portal. 27 December 2023. Archived from the original on 2 April 2024. Retrieved 2 April 2024.
  5. ^ a b c "Archived copy". Archived from the original on 29 April 2024. Retrieved 29 April 2024.{{cite web}}: CS1 maint: archived copy as title (link)
  6. ^ a b c "Beqvez". U.S. Food and Drug Administration (FDA). 25 April 2024. STN: 125786. Archived from the original on 29 April 2024. Retrieved 29 April 2024. Public Domain This article incorporates text from this source, which is in the public domain.
  7. ^ "fidanacogene elaparvovec". CADTH. 20 June 2023. Archived from the original on 2 March 2024. Retrieved 2 March 2024.
  8. ^ "U.S. FDA Approves Pfizer's Beqvez (fidanacogene elaparvovec-dzkt), a One-Time Gene Therapy for Adults with Hemophilia B" (Press release). Pfizer. 26 April 2024. Archived from the original on 29 April 2024. Retrieved 29 April 2024 – via Business Wire.
  9. ^ 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. ISSN 0006-4971.
  10. ^ von Mackensen, Sylvia; Ducore, Jonathan M.; George, Lindsey A.; Giermasz, Adam; McGuinn, Catherine; Rasko, John E. J.; et al. (28 November 2023). "Health-Related Quality of Life in Adults with Hemophilia B after Receiving Gene Therapy with Fidanacogene Elaparvovec". Blood. 142 (Supplement 1): 3628. doi:10.1182/blood-2023-179431.
  11. ^ George, Lindsey A.; Sullivan, Spencer K.; Giermasz, Adam; Samelson-Jones, Ben J.; Ducore, Jonathan M.; Teitel, Jerome M.; et al. (8 December 2017). "Spk-9001: Adeno-Associated Virus Mediated Gene Transfer for Hemophilia B - 1 Year Follow up and Impact of Baseline Characteristics on Transgene-Derived Factor IX Activity and Persistence". Blood. 130: 601. doi:10.1182/blood.V130.Suppl_1.601.601 (inactive 31 January 2024). ISSN 0006-4971. Archived from the original on 8 December 2023. Retrieved 8 December 2023.{{cite journal}}: CS1 maint: DOI inactive as of January 2024 (link)
  12. ^ George, Lindsey A.; Sullivan, Spencer K.; Rasko, John E.J.; Giermasz, Adam; Samelson-Jones, Benjamin J.; Ducore, Jonathan M.; et al. (13 November 2019). "Efficacy and Safety in 15 Hemophilia B Patients Treated with the AAV Gene Therapy Vector Fidanacogene Elaparvovec and Followed for at Least 1 Year". Blood. 134 (Supplement_1): 3347. doi:10.1182/blood-2019-124091.

External links[edit]

  • "Fidanacogene Elaparvovec (Code C171800)". NCI Thesaurus.
  • Clinical trial number NCT02484092 for "A Gene Therapy Study for Hemophilia B" at ClinicalTrials.gov
  • 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


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