Fidanacogene elaparvovec

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Fidanacogene elaparvovec
Gene therapy
Vector Adeno-associated virus
Clinical data
Trade names Beqvez, others
Other namesSPK-9001, fidanacogene elaparvovec-dzkt
AHFS/Drugs.com Monograph
License data
Routes of
administration 		Intravenous
ATC code
  • None
Legal status
Legal status
Identifiers
CAS Number
DrugBank
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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]

Contents

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]

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] [11] The applicant for this medicinal product is Pfizer Europe MA EEIG. [6] [11] [12] 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. [13]

Research

Fidanacogene elaparvovec partially restored factor IX production in preliminary studies. [14] [15] [16] [17] 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. [18]

Related Research Articles

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Gene therapy is a medical technology that aims to produce a therapeutic effect through the manipulation of gene expression or through altering the biological properties of living cells.

<span class="mw-page-title-main">Haemophilia</span> Genetic disease involving blood clotting

Haemophilia, or hemophilia, is a mostly inherited genetic disorder that impairs the body's ability to make blood clots, a process needed to stop bleeding. This results in people bleeding for a longer time after an injury, easy bruising, and an increased risk of bleeding inside joints or the brain. Those with a mild case of the disease may have symptoms only after an accident or during surgery. Bleeding into a joint can result in permanent damage while bleeding in the brain can result in long term headaches, seizures, or an altered level of consciousness.

<span class="mw-page-title-main">Haemophilia A</span> Medical condition

Haemophilia A is a blood clotting disorder caused by a genetic deficiency in clotting factor VIII, thereby resulting in significant susceptibility to bleeding, both internally and externally. This condition occurs almost exclusively in males born to carrier mothers due to X-linked recessive inheritance. Nevertheless, rare isolated cases do emerge from de novo (spontaneous) mutations.

<span class="mw-page-title-main">Haemophilia B</span> Genetic X-linked recessive bleeding disorder

Haemophilia B, also spelled hemophilia B, is a blood clotting disorder causing easy bruising and bleeding due to an inherited mutation of the gene for factor IX, and resulting in a deficiency of factor IX. It is less common than factor VIII deficiency.

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Etranacogene dezaparvovec, sold under the brand name Hemgenix is a gene therapy used for the treatment of hemophilia B. Etranacogene dezaparvovec is an adeno-associated virus vector-based gene therapy which consists of a viral vector carrying a gene for clotting Factor IX. The gene is expressed in the liver to produce Factor IX protein, to increase blood levels of Factor IX and thereby limit bleeding episodes. Hemophilia B is a genetic bleeding disorder resulting from missing or insufficient levels of blood clotting Factor IX, a protein needed to produce blood clots to stop bleeding.

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Dirloctogene samoparvovec, also known as SPK-8011, is an experimental gene therapy developed for hemophilia A by Roche and Spark Therapeutics. It uses an engineered AAV vector to cause liver cells to produce the Factor VIII blood clotting protein.

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References

  1. 1 2 3 4 "Beqvez Product information". Health Canada . 22 October 2009. Archived from the original on 3 March 2024. Retrieved 3 March 2024.
  2. ↑ "Details for: Beqvez". Health Canada . 27 December 2023. Archived from the original on 3 March 2024. Retrieved 3 March 2024.
  3. ↑ "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.
  4. ↑ "Summary Basis of Decision for Beqvez". Drug and Health Products Portal. 1 September 2012. Retrieved 13 November 2024.
  5. 1 2 3 "Beqvez- fidanacogene elaparvovec-dzkt kit". DailyMed. 7 May 2024. Retrieved 18 May 2024.
  6. 1 2 3 4 5 6 "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.
  7. 1 2 "Durveqtix Product information". Union Register of medicinal products. 26 July 2024. Retrieved 5 August 2024.
  8. ↑ "Fidanacogene elaparvovec". CADTH. 20 June 2023. Archived from the original on 2 March 2024. Retrieved 2 March 2024.
  9. 1 2 3 "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.PD-icon.svg This article incorporates text from this source, which is in the public domain .
  10. 1 2 "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. 1 2 "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.
  12. ↑ "New gene therapy treatment for haemophilia B". European Medicines Agency (Press release). 31 May 2024. Retrieved 1 June 2024.
  13. ↑ Dunleavy, Kevin (26 April 2024). "Pfizer scores FDA nod for hemophilia B gene therapy, will charge $3.5M per dose". Fierce Pharma.
  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. ISSN   0006-4971.
  15. ↑ 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 .
  16. ↑ 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 1 November 2024). ISSN   0006-4971. Archived from the original on 8 December 2023. Retrieved 8 December 2023.{{cite journal}}: CS1 maint: DOI inactive as of November 2024 (link)
  17. ↑ 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 .
  18. ↑ Cuker, Adam; Kavakli, Kaan; Frenzel, Laurent; Wang, Jiaan-Der; Astermark, Jan; Cerqueira, Monica H.; et al. (26 September 2024). "Gene Therapy with Fidanacogene Elaparvovec in Adults with Hemophilia B". New England Journal of Medicine. 391 (12): 1108–1118. doi:10.1056/NEJMoa2302982. ISSN   0028-4793. PMID   39321362.
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