Drug updated on 4/18/2024
| 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.
Summary
- Elexacaftor, ivacaftor, and tezacaftor (Trikafta) is indicated for the treatment of cystic fibrosis in patients aged 2 years and older who have at least one F508del mutation in the CFTR gene or a mutation that is responsive based on in vitro data.
- Eight studies were reviewed which consistently reported significant improvements in lung function as measured by increases in FEV1% predicted values among patients treated with Trikafta. This effect was superior compared to those receiving other CFTR modulator therapies or placebo.
- The use of Trikafta led to a notable reduction in acute pulmonary exacerbations contributing significantly to improved quality of life and possibly long-term outcomes for cystic fibrosis patients.
- Patients on Trikafta experienced marked improvements not only in their quality of life metrics but also showed increases in BMI, indicating enhanced nutritional status and overall well-being.
- While traditional CFTR modulators showed limited efficacy in managing cystic fibrosis-related diabetes (CFRD), evidence suggests that Trikafta has potential benefits in improving glycemic control and reducing the prevalence of CFRD among these patients, although more research is needed to fully understand this effect.
- Compared to dual therapies like lumacaftor/ivacaftor and tezacaftor/ivacaftor, the combination of elexacaftor, ivacaftor, and tezacaftor shows superior clinical outcomes in improving pulmonary function and quality of life while reducing exacerbations, maintaining a comparable safety profile. The incidence of serious adverse events was comparable, but the overall benefit-risk profile favors this drug combination.
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 |