Chemistry:Fidanacogene elaparvovec

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Short description: Gene therapy

Fidanacogene elaparvovec
Gene therapy
VectorAdeno-associated virus
Clinical data
Trade namesBeqvez, others
Other namesSPK-9001, fidanacogene elaparvovec-dzkt
AHFS/Drugs.comMonograph
MedlinePlusa624028
License data
Routes of
administration		Intravenous
ATC code
Legal status
Legal status
Identifiers
CAS Number
DrugBank
UNII
KEGG

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]

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][9] It is given as a one-time infusion.[10]

Society and culture

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.[6] The FDA granted the application breakthrough therapy designation.[11]

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.[6][12] The applicant for this medicinal product is Pfizer Europe MA EEIG.[6][12][13] The conditional marketing authorization was granted in July 2024.[6][7]

Economics

Pfizer announced a cost of 3.5 million per treatment, the same cost as the CSL Behring's competing hemophilia gene therapy etranacogene dezaparvovec.[14]

Research

Fidanacogene elaparvovec partially restored factor IX production in preliminary studies.[15][16][17][18] The results of a phase 3 trial were published in September 2024. It showed that even 15 months after treatment factor IX was still being expressed and the number of bleedings had decreased significantly compared to the time before the treatment, when study participants had been given prophylactic infusions of factor IX.[19]

References

  1. ↑ 1.0 1.1 1.2 1.3 "Beqvez Product information". 22 October 2009. https://health-products.canada.ca/dpd-bdpp/info?lang=eng&code=103268. 
  2. ↑ "Details for: Beqvez". 27 December 2023. https://dhpp.hpfb-dgpsa.ca/dhpp/resource/103268. 
  3. ↑ "Regulatory Decision Summary for Beqvez". 27 December 2023. https://dhpp.hpfb-dgpsa.ca/review-documents/resource/RDS1707836185816. 
  4. ↑ "Summary Basis of Decision for Beqvez". 1 September 2012. https://dhpp.hpfb-dgpsa.ca/review-documents/resource/SBD1728318721334. 
  5. ↑ 5.0 5.1 5.2 "Beqvez- fidanacogene elaparvovec-dzkt kit". 7 May 2024. https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=5f477a47-bdc2-41fb-a720-30f9809dc6e8. 
  6. ↑ 6.0 6.1 6.2 6.3 6.4 6.5 "Durveqtix EPAR". 30 May 2024. https://www.ema.europa.eu/en/medicines/human/EPAR/durveqtix.  Text was copied from this source which is copyright European Medicines Agency. Reproduction is authorized provided the source is acknowledged.
  7. ↑ 7.0 7.1 "Durveqtix Product information". 26 July 2024. https://ec.europa.eu/health/documents/community-register/html/h1838.htm. 
  8. ↑ "Fidanacogene elaparvovec". 20 June 2023. https://www.cadth.ca/fidanacogene-elaparvovec. 
  9. ↑ 9.0 9.1 9.2 "Beqvez". 25 April 2024. https://www.fda.gov/vaccines-blood-biologics/cellular-gene-therapy-products/beqvez.  Public Domain This article incorporates text from this source, which is in the public domain.
  10. ↑ 10.0 10.1 "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.
  11. ↑ "CY 2024 CDER Breakthrough Therapy Calendar Year Approvals" (PDF). U.S. Food and Drug Administration (FDA). September 30, 2024. https://www.fda.gov/media/97001/download. 
  12. ↑ 12.0 12.1 "Meeting highlights from the Committee for Medicinal Products for Human Use (CHMP) 27-30 May 2024". European Medicines Agency (Press release). 31 May 2024. Retrieved 13 June 2024.
  13. ↑ "New gene therapy treatment for haemophilia B". European Medicines Agency (Press release). 31 May 2024. Retrieved 1 June 2024.
  14. ↑ Dunleavy, Kevin (26 April 2024). "Pfizer scores FDA nod for hemophilia B gene therapy, will charge $3.5M per dose". https://www.fiercepharma.com/pharma/pfizer-scores-fda-nod-hemophilia-b-gene-therapy-will-charge-35m-dose. 
  15. ↑ George, Lindsey A.; Sullivan, Spencer K.; Giermasz, Adam; Ducore, Jonathan M.; Teitel, Jerome M.; Cuker, Adam; Sullivan, Lisa M.; Majumdar, Suvankar 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" (in en). Blood 128 (22): 3. doi:10.1182/blood.V128.22.3.3. ISSN 0006-4971. 
  16. ↑ von Mackensen, Sylvia; Ducore, Jonathan M.; George, Lindsey A.; Giermasz, Adam; McGuinn, Catherine; Rasko, John E. J.; Samelson-Jones, Ben J; Sullivan, Spencer K. 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. 
  17. ↑ George, Lindsey A.; Sullivan, Spencer K.; Giermasz, Adam; Samelson-Jones, Ben J.; Ducore, Jonathan M.; Teitel, Jerome M.; Cuker, Adam; Von Mackensen, Sylvia 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. ISSN 0006-4971. https://www.sciencedirect.com/science/article/pii/S0006497119811175. Retrieved 8 December 2023. 
  18. ↑ George, Lindsey A.; Sullivan, Spencer K.; Rasko, John E.J.; Giermasz, Adam; Samelson-Jones, Benjamin J.; Ducore, Jonathan M.; Teitel, Jerome M.; McGuinn, Catherine E. 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. 
  19. ↑ Cuker, Adam; Kavakli, Kaan; Frenzel, Laurent; Wang, Jiaan-Der; Astermark, Jan; Cerqueira, Monica H.; Iorio, Alfonso; Katsarou-Fasouli, Olga et al. (26 September 2024). "Gene Therapy with Fidanacogene Elaparvovec in Adults with Hemophilia B" (in en). New England Journal of Medicine 391 (12): 1108–1118. doi:10.1056/NEJMoa2302982. ISSN 0028-4793. PMID 39321362. http://www.nejm.org/doi/10.1056/NEJMoa2302982. 




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