Drug updated on 9/4/2024
| 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 | CFTR potentiators and correctors |
| 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 patient’s 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
- Trikafta (elexacaftor, ivacaftor, and tezacaftor) is 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 patient’s 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.
- This summary is based on the review of eight systematic review(s)/meta-analysis(es). [1-8]
- FEV1% Improvement: ETI treatment led to an increase in FEV1% by +10.47% (95% CI, 6.88-14.06) in one meta-analysis, with increases of 9.23% after 4 weeks and 12.57% after 24 weeks reported in another meta-analysis.
- Reduction in Pulmonary Exacerbations: ETI treatment reduced acute pulmonary exacerbations with a risk difference (RD) of -0.16 (95% CI, -0.28 to -0.04) and showed decreased exacerbation rates in combined therapy.
- Quality of Life Enhancement: ETI treatment improved quality of life with a reported RD of +14.93 (95% CI, 9.98-19.89) and CFQ-R score improvements of 21.46 points after 4 weeks and 19.29 points after 24 weeks.
- Adverse events during ETI treatment did not significantly differ from control groups (RD, -0.03; 95% CI, -0.08 to 0.01). Mild to moderate adverse events were reported, with common side effects including headache and rash. Severe adverse events had an incidence rate of 0.066 (95% CI, 0.028-0.104).
- Increases in systolic and diastolic blood pressure were associated with lumacaftor-ivacaftor but not with ETI or TEZ/IVA. Real-world studies reported a higher frequency of discontinuation and mental health-related adverse events with CFTR modulators.
- Significant improvements in lung function and quality of life were observed in participants aged ≥12 years and children aged 6-11 years with the Phe508del mutation and/or advanced lung disease, with the greatest benefits in lung function and reduction in exacerbations noted in patients with the F508del mutation.
Product Monograph / Prescribing Information
| Document Title | Year | Source |
|---|---|---|
| Trikafta (elexacaftor, ivacaftor, and tezacaftor) 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 |