Fidanacogene elaparvovec
| Gene therapy | |
|---|---|
| Vector | Adeno-associated virus |
| Clinical data | |
| Trade names | Beqvez |
| Other names | SPK-9001 |
| Biosimilars | fidanacogene elaparvovec-dzkt |
| License data | |
| Routes of administration | Intravenous |
| ATC code |
|
| 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][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[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][7]
Research[edit]
It partially restores factor IX production in preliminary studies.[9][10][11][12]
Society and culture[edit]
Legal status[edit]
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]
References[edit]
- ^ a b c "Beqvez Product information". Health Canada. 22 October 2009. Archived from the original on 3 March 2024. Retrieved 3 March 2024.
- ^ "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.
- ^ "Details for: Beqvez". Health Canada. 27 December 2023. Archived from the original on 3 March 2024. Retrieved 3 March 2024.
- ^ "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.
- ^ a b c "Beqvez- fidanacogene elaparvovec-dzkt kit". DailyMed. 7 May 2024. Retrieved 18 May 2024.
- ^ "Fidanacogene elaparvovec". CADTH. 20 June 2023. Archived from the original on 2 March 2024. Retrieved 2 March 2024.
- ^ a b "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.
This article incorporates text from this source, which is in the public domain.
- ^ "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.
- ^ 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.
- ^ 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.
- ^ 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) - ^ 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.
- ^ a b "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.
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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