Fidanacogene elaparvovec explained

Gt Vector:Adeno-associated virus
Tradename:Beqvez, others
Dailymedid:Fidanacogene elaparvovec
Routes Of Administration:Intravenous
Atc Prefix:None
Legal Ca:Rx-only
Legal Ca Comment:/Schedule D[1] [2] [3] [4]
Legal Us:Rx-only
Legal Us Comment:[5]
Legal Eu:Rx-only
Cas Number:1954659-47-2
Drugbank:DB16783
Unii:413EU9081Y
Kegg:D12908
Synonyms:SPK-9001, fidanacogene elaparvovec-dzkt

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).[6]

Fidanacogene elaparvovec was approved for medical use in Canada in December 2023, in the United States in April 2024,[7] [8] and in the European Union in July 2024.

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.

Society and culture

Legal status

Fidanacogene elaparvovec was approved for medical use in Canada in December 2023, in the United States in April 2024, and in the European Union in July 2024.

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.[9] The applicant for this medicinal product is Pfizer Europe MA EEIG.[10] [11] The conditional marketing authorization was granted in July 2024.[12]

Research

It partially restores factor IX production in preliminary studies.[13] [14] [15] [16]

External links

Notes and References

  1. Web site: Beqvez Product information . . 22 October 2009 . 3 March 2024 . 3 March 2024 . https://web.archive.org/web/20240303040332/https://health-products.canada.ca/dpd-bdpp/info?lang=eng&code=103268 . live .
  2. Web site: Notice: Multiple additions to the Prescription Drug List (PDL) [2024-02-28] ]. . 28 February 2024 . 2 March 2024 . 2 March 2024 . https://web.archive.org/web/20240302070630/https://www.canada.ca/en/health-canada/services/drugs-health-products/drug-products/prescription-drug-list/notices-changes/multiple-additions-2024-02-28.html . live .
  3. Web site: Details for: Beqvez . . 27 December 2023 . 3 March 2024 . 3 March 2024 . https://web.archive.org/web/20240303045105/https://dhpp.hpfb-dgpsa.ca/dhpp/resource/103268 . live .
  4. Web site: Regulatory Decision Summary for Beqvez . Drug and Health Products Portal . 27 December 2023 . 2 April 2024 . 2 April 2024 . https://web.archive.org/web/20240402041641/https://dhpp.hpfb-dgpsa.ca/review-documents/resource/RDS1707836185816 . live .
  5. Web site: Beqvez- fidanacogene elaparvovec-dzkt kit . DailyMed . 7 May 2024 . 18 May 2024.
  6. Web site: Fidanacogene elaparvovec . CADTH . 20 June 2023 . 2 March 2024 . 2 March 2024 . https://web.archive.org/web/20240302072000/https://www.cadth.ca/fidanacogene-elaparvovec . live .
  7. Web site: Beqvez . U.S. Food and Drug Administration (FDA) . 25 April 2024 . 29 April 2024 . STN: 125786 . 29 April 2024 . https://web.archive.org/web/20240429055135/https://www.fda.gov/vaccines-blood-biologics/cellular-gene-therapy-products/beqvez . live .
  8. U.S. FDA Approves Pfizer's Beqvez (fidanacogene elaparvovec-dzkt), a One-Time Gene Therapy for Adults with Hemophilia B . Pfizer . Business Wire . 26 April 2024 . 29 April 2024 . 29 April 2024 . https://web.archive.org/web/20240429055143/https://www.businesswire.com/news/home/20240425269649/en/U.S.-FDA-Approves-Pfizer%E2%80%99s-BEQVEZ%E2%84%A2-fidanacogene-elaparvovec-dzkt-a-One-Time-Gene-Therapy-for-Adults-with-Hemophilia-B . live .
  9. Meeting highlights from the Committee for Medicinal Products for Human Use (CHMP) 27-30 May 2024 . European Medicines Agency . 31 May 2024 . 13 June 2024.
  10. Web site: Durveqtix EPAR . European Medicines Agency . 30 May 2024 . 31 May 2024. Text was copied from this source which is copyright European Medicines Agency. Reproduction is authorized provided the source is acknowledged.
  11. New gene therapy treatment for haemophilia B . . 31 May 2024 . 1 June 2024.
  12. Web site: Durveqtix Product information . Union Register of medicinal products . 26 July 2024 . 5 August 2024.
  13. George . Lindsey A. . Sullivan . Spencer K. . Giermasz . Adam . Ducore . Jonathan M. . Teitel . Jerome M. . Cuker . Adam . Sullivan . Lisa M. . Majumdar . Suvankar . McGuinn . Catherine E. . Galvao . Aline M. . Luk . Alvin Y. . Wright . J. Fraser . Chen . Yifeng . Hui . Daniel J. . Wachtel . Katie . Urich . Teresa . Takefman . Daniel . Couto . Linda B. . Carr . Marcus E. . Anguela . Xavier M. . High . Katherine A. . Spk-9001: Adeno-Associated Virus Mediated Gene Transfer for Hemophilia B Achieves Sustained Mean Factor IX Activity Levels of >30% without Immunosuppression . Blood . 2 December 2016 . 128 . 22 . 3 . 10.1182/blood.V128.22.3.3 . en . 0006-4971.
  14. von Mackensen . Sylvia . Ducore . Jonathan M. . George . Lindsey A. . Giermasz . Adam . McGuinn . Catherine . Rasko . John E. J. . Samelson-Jones . Ben J . Sullivan . Spencer K. . Teitel . Jerome M. . Chhabra . Amit . Fang . Annie F. . O'Brien . Amanda . Plonski . Frank . Rupon . Jeremy . Smith . Lynne . Winburn . Ian . Health-Related Quality of Life in Adults with Hemophilia B after Receiving Gene Therapy with Fidanacogene Elaparvovec . Blood . 28 November 2023 . 142 . Supplement 1 . 3628 . 10.1182/blood-2023-179431. free .
  15. George . Lindsey A. . Sullivan . Spencer K. . Giermasz . Adam . Samelson-Jones . Ben J. . Ducore . Jonathan M. . Teitel . Jerome M. . Cuker . Adam . Von Mackensen . Sylvia . Majumdar . Suvankar . McGuinn . Catherine E. . Runoski . Alexa R. . Wright . Fraser . Dasen . Sue . Barber . Kimberly . Chen . Yifeng . Hui . Daniel J. . Patel . Ruchi . Liu . Yun . Wachtel . Katie . Takefman . Dan . Couto . Linda B. . Reape . Kathleen Z. . Carr . Marcus E. . Anguela . Xavier M. . High . Katherine A. . 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 . 8 December 2017 . 130 . 601 . 10.1182/blood.V130.Suppl_1.601.601 . 31 January 2024 . 0006-4971 . 8 December 2023 . 8 December 2023 . https://web.archive.org/web/20231208094907/https://www.sciencedirect.com/science/article/pii/S0006497119811175 . live .
  16. 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. . Runowski . Alexa R. . Wright . Fraser . Anguela . Xavier M. . High . Katherine A. . Rybin . Denis . Murphy . John E. . Rupon . Jeremy . 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 . 13 November 2019 . 134 . Supplement_1 . 3347 . 10.1182/blood-2019-124091. free .

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