Elexacaftor/tezacaftor/ivacaftor

Elexacaftor/tezacaftor/ivacaftor, sold under the brand name Trikafta, is a fixed-dose combination medication used in those that have cystic fibrosis with a f508del mutation.[1] It is made up of a combination of elexacaftor, tezacaftor, and ivacaftor.[1]

Elexacaftor/tezacaftor/ivacaftor
Combination of
ElexacaftorCystic fibrosis transmembrane conductance regulator (CFTR) corrector
TezacaftorCFTR corrector
IvacaftorChloride channel opener
Clinical data
Trade namesTrikafta
AHFS/Drugs.comMonograph
MedlinePlusa619061
License data
Routes of
administration		By mouth
ATC code
  • None
Legal status
Legal status
Identifiers
KEGG

It was approved for medical use in the United States in 2019.[1][2][3]

In June 2020, the Committee for Medicinal Products for Human Use of the European Medicines Agency (EMA) recommended the approval of elexacaftor/tezacaftor/ivacaftor (Kaftrio) for the treatment of cystic fibrosis.[4][5] It was approved for medical use in the European Union in August 2020.[6]

Pharmacology

A phase III trial showed people treated with elexacaftor/tezacaftor/ivacaftor improved in FEV1 at four weeks with sustained improvement at 24 weeks. Rate of pulmonary exacerbation was 63% lower and a sweat chloride concentration that was 41.8 mmol/L lower.[7][8][9]

Society and culture

Formulations

Elexacaftor/tezacaftor/ivacaftor is a combination tablet containing elexacaftor 100 mg, tezacaftor, 50 mg and ivacaftor 75 mg.[1] It is sold together with ivacaftor 150 mg tablets with which it is used.[1]

The combination is approved for use in the United States and is indicated for people 12 years and older with cystic fibrosis who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, which is estimated to represent 90% of the cystic fibrosis population.[2]

The U.S. Food and Drug Administration (FDA) granted the application priority review, in addition to fast track and breakthrough therapy designations. Trikafta also received orphan drug designation, which provides incentives to assist and encourage the development of drugs for rare diseases. The drug's manufacturer Vertex Pharmaceuticals will receive a rare pediatric disease priority review voucher for having developed this therapy.[2]

Cost

The list price of a year's treatment in the US is $311,000.[10] However, a 2020 report by Institute for Clinical and Economic Review found that the price has made the treatment not cost effective and that "an appropriate health-benefit price would range from $67,900–$85,500 per year".[11][12]

References
  1. "Trikafta- elexacaftor, tezacaftor, and ivacaftor kit". DailyMed. January 29, 2020. Retrieved August 22, 2020.
  2. "FDA approves new breakthrough therapy for cystic fibrosis". U.S. Food and Drug Administration (FDA) (Press release). October 21, 2019. Archived from the original on November 13, 2019. Retrieved November 13, 2019. This article incorporates text from this source, which is in the public domain.
  3. "Drug Trials Snapshots: Trikafta". U.S. Food and Drug Administration (FDA). October 31, 2019. Archived from the original on November 20, 2019. Retrieved November 20, 2019. This article incorporates text from this source, which is in the public domain.
  4. "New medicine for cystic fibrosis patients". European Medicines Agency (EMA) (Press release). June 26, 2020. Retrieved June 26, 2020.
  5. "Elexacaftor + ivacaftor + tezacaftor". SPS - Specialist Pharmacy Service. July 28, 2020. Retrieved August 21, 2020.
  6. "Kaftrio EPAR". European Medicines Agency (EMA). June 23, 2020. Retrieved August 21, 2020.
  7. Middleton PG, Mall MA, Dřevínek P, et al. (VX17-445-102 Study Group) (October 2019). "Elexacaftor–Tezacaftor–Ivacaftor for Cystic Fibrosis with a Single Phe508del Allele". N. Engl. J. Med. 381 (19): 1809–19. doi:10.1056/NEJMoa1908639. ISSN 0028-4793. PMC 7282384. PMID 31697873.
  8. Taylor-Cousar JL, Mall MA, Ramsey BW, et al. (April 2019). "Clinical development of triple-combination CFTR modulators for cystic fibrosis patients with one or two F508del alleles". ERJ Open Res. 5 (2): 00082–2019. doi:10.1183/23120541.00082-2019. PMC 6571452. PMID 31218221.
  9. Clinical trial number NCT03525444 for "A Phase 3 Study of VX-445 Combination Therapy in Subjects With Cystic Fibrosis Heterozygous for the F508del Mutation and a Minimal Function Mutation (F/MF)" at ClinicalTrials.gov
  10. Maddipatla, Manojna; O'Donnell, Carl (October 21, 2019). "Vertex prices cystic fibrosis combo treatment at $311,000-per-year". Reuters. Retrieved October 23, 2019.
  11. PhD, Ines Martins. "Trikafta Very Effective CF Therapy, But Still Too Costly, ICER Reports". Retrieved August 21, 2020.
  12. "Modulator Treatments for Cystic Fibrosis: Effectiveness and Value" (PDF). Institute for Clinical and Economic Review. Institute for Clinical and Economic Review. April 27, 2020. Retrieved August 21, 2020.


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