Drug updated on 10/29/2024
| Dosage Form | Tablet (oral; 50 mg elexacaftor, 25 mg tezacaftor, and 37.5 mg ivacaftor, co-packaged with 75 mg ivacaftor; 100 mg elexacaftor, 50 mg tezacaftor, and 75 mg ivacaftor, co-packaged with 150 mg ivacaftor); Oral granules (oral; 100 mg elexacaftor, 50 mg tezacaftor, and 75 mg ivacaftor, co-packaged with 75 mg ivacaftor; 80 mg elexacaftor, 40 mg tezacaftor, and 60 mg ivacaftor, co-packaged with 59.5 mg ivacaftor) |
| Drug Class | Cystic fibrosis transmembrane conductance regulator (CFTR) potentiators |
| Ongoing and Completed Studies | ClinicalTrials.gov |
Indication
- Indicated for the treatment of cystic fibrosis (CF) in patients aged 2 years and older who have at least one F508del mutation in the CFTR gene or a mutation in the CFTR gene that is responsive based on in vitro data
- If the patients genotype is unknown, an FDA-cleared CF mutation test should be used to confirm the presence of at least one F508del mutation or a mutation that is responsive based on in vitro data.
Latest News
Summary
- This summary is based on the review of nine systematic review(s)/meta-analysis(es). [1-9]
- The studies focused on patients aged 6 years and older with cystic fibrosis, specifically those with at least one Phe508del mutation, as well as non-F508del CFTR (cystic fibrosis transmembrane conductance regulator) variants, including N1303K and G85E, demonstrating significant effectiveness of elexacaftor-tezacaftor-ivacaftor (Trikafta) in improving lung function, reducing sweat chloride levels, and enhancing quality of life.
- The safety profile of elexacaftor-tezacaftor-ivacaftor (Trikafta) indicated that adverse events (AEs) did not differ significantly from control groups, with a risk difference of -0.03 (95% CI: -0.08 to 0.01), and the overall incidence of AEs was 0.824 (95% CI: 0.769-0.879), including severe AEs at 0.066 (95% CI: 0.028-0.104); common side effects included headache and rash, and no deaths were reported.
- Specific populations studied included patients aged 6 years and older, with particular attention to those with at least one Phe508del mutation and non-F508del CFTR variants, showing mild to moderate AEs without significant differences in safety outcomes compared to other CFTR modulators.
- The population types and subgroup considerations indicate that elexacaftor-tezacaftor-ivacaftor is effective for patients aged 12 years and older with at least one Phe508del mutation, as well as for individuals with non-F508del CFTR variants, particularly those with N1303K and G85E; high efficacy was also noted in patients with F508del/minimal function and F508del/F508del variants, although further randomized controlled trials are needed for those under 12 years.
Product Monograph / Prescribing Information
| Document Title | Year | Source |
|---|---|---|
| Trikafta (elexacaftor, tezacaftor, ivacaftor) Prescribing Information. | 2023 | Vertex Pharmaceuticals Inc., Boston, MA |
Systematic Reviews / Meta-Analyses
Clinical Practice Guidelines
| Document Title | Year | Source |
|---|---|---|
| Canadian clinical consensus guideline for initiation, monitoring and discontinuation of CFTR modulator therapies for patients with cystic fibrosis. | 2021 | Cystic Fibrosis Canada |