Vanzacaftor/tezacaftor/deutivacaftor

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Vanzacaftor/tezacaftor/deutivacaftor
Vanzacaftor, tezacaftor and deutivacaftor.svg
Combination of
Vanzacaftor Medication
Tezacaftor Medication
Deutivacaftor CFTR potentiator
Clinical data
Trade names Alyftrek
AHFS/Drugs.com Multum Consumer Information
License data
Routes of
administration 		By mouth
ATC code
Legal status
Legal status
Identifiers
KEGG

Vanzacaftor/tezacaftor/deutivacaftor, sold under the brand name Alyftrek, is a fixed-dose combination medication used for the treatment of cystic fibrosis. [2] It is a combination of deutivacaftor, a CFTR potentiator; tezacaftor; and vanzacaftor, as the calcium salt, vanzacaftor calcium dihydrate. [2] It is taken by mouth. [2]

Contents

The combination was approved for medical use in the United States in December 2024, [5] [6] in the European Union in June 2025, [4] and in Canada in July 2025. [1]

Medical uses

The combination is indicated for the treatment of cystic fibrosis in people aged six years of age and older who have at least one F508del mutation or another responsive mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. [2] [7]

Adverse affects

The US Food and Drug Administration (FDA) prescription label for the combination contains a boxed warning about drug-induced liver injury and liver failure. [2]

History

The US Food and Drug Administration (FDA) granted the application for vanzacaftor, tezacaftor, and deutivacaftor combination therapy orphan drug designation. [8]

Society and culture

The combination was approved for medical use in the United States in December 2024. [5] [9]

In April 2025, the Committee for Medicinal Products for Human Use of the European Medicines Agency adopted a positive opinion, recommending the granting of a marketing authorization for the medicinal product Alyftrek, intended for the treatment of cystic fibrosis in people aged six years of age and older who have at least one non‑class I mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. [3] The applicant for this medicinal product is Vertex Pharmaceuticals (Ireland) Limited. [3] The combination was authorized for medical use in the European Union in June 2025. [3] [4]

Names

The combination is sold under the brand name Alyftrek. [2]

References

  1. 1 2 "Product information". Health Canada . 21 July 2025. Retrieved 20 August 2025.
  2. 1 2 3 4 5 6 7 "Alyftrek- vanzacaftor, tezacaftor, and deutivacaftor tablet, film coated". DailyMed. 9 January 2025. Retrieved 25 January 2025.
  3. 1 2 3 4 "Alyftrek EPAR". European Medicines Agency (EMA). 25 April 2025. Retrieved 2 May 2025. Text was copied from this source which is copyright European Medicines Agency. Reproduction is authorized provided the source is acknowledged.
  4. 1 2 3 "Alyftrek Product information". Union Register of medicinal products. 1 July 2025. Retrieved 10 July 2025.
  5. 1 2 "Novel Drug Approvals for 2024". U.S. Food and Drug Administration (FDA). 23 December 2024. Archived from the original on 19 April 2024. Retrieved 24 December 2024.
  6. ↑ New Drug Therapy Approvals 2024 (PDF). U.S. Food and Drug Administration (FDA) (Report). January 2025. Archived from the original on 21 January 2025. Retrieved 21 January 2025.
  7. ↑ Bacalhau M, Camargo M, Magalhães-Ghiotto GA, Drumond S, Castelletti CH, Lopes-Pacheco M (March 2023). "Elexacaftor-Tezacaftor-Ivacaftor: A Life-Changing Triple Combination of CFTR Modulator Drugs for Cystic Fibrosis". Pharmaceuticals (Basel, Switzerland). 16 (3): 410. doi: 10.3390/ph16030410 . PMC   10053019 . PMID   36986509.
  8. ↑ "Alyftrek Orphan Drug Designations and Approvals". U.S. Food and Drug Administration (FDA). Archived from the original on 21 January 2022. Retrieved 24 December 2024.
  9. ↑ "Vertex Announces US FDA Approval of Alyftrek, a Once-Daily Next-in-Class CFTR Modulator for the Treatment of Cystic Fibrosis" (Press release). Vertex. 20 December 2024. Retrieved 24 December 2024– via Business Wire.
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