Drug updated on 12/11/2024
| Dosage Form | Tablet (oral; tezacaftor/ivacaftor and ivacaftor: 50 mg/75 mg and 75 mg); Tablet (oral; tezacaftor/ivacaftor and ivacaftor: 100 mg/150 mg and 150 mg) |
| 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 age 6 years and older who are homozygous for the F508del mutation or who have at least one mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene that is responsive to tezacaftor/ivacaftor based on in vitro data and/or clinical evidence.
Latest News
Summary
- This summary is based on the review of five systematic review(s)/meta-analysis(es). [1-5]
- Elexacaftor-Tezacaftor-Ivacaftor (Trikafta) Triple Therapy significantly improved pulmonary function (forced expiratory volume in 1 second (FEV₁)) and quality of life (QoL) in cystic fibrosis (CF) patients aged 12 years and older with F508del variants, leading to reduced exacerbation rates.
- Ivacaftor (IVA) Monotherapy resulted in notable weight gain in children and adults with at least one G551D mutation, while no significant body mass index (BMI) changes were observed in adults with the R117H mutation or children homozygous for F508del receiving IVA with Tezacaftor.
- Combination Therapies demonstrated varying effectiveness, with Elexacaftor-Tezacaftor-Ivacaftor showing superior improvements in QoL and lung function compared to both Lumacaftor-Ivacaftor and Tezacaftor-Ivacaftor, which had moderate improvements.
- Common Adverse Effects of Elexacaftor-Tezacaftor-Ivacaftor include headache and rash, while Lumacaftor-Ivacaftor is associated with early transient breathlessness and increased blood pressure.
- Elexacaftor-Tezacaftor-Ivacaftor demonstrated comparable adverse events to placebo or control, whereas Tezacaftor-Ivacaftor reported fewer adverse events compared to Lumacaftor-Ivacaftor.
- Studies included individuals aged 12 years and older, with significant benefits observed in the F508del mutation cohort from Elexacaftor-Tezacaftor-Ivacaftor, and notable weight gain in individuals with at least one G551D mutation from Ivacaftor; effectiveness and safety for younger populations (<12 years) were not well established.
Product Monograph / Prescribing Information
| Document Title | Year | Source |
|---|---|---|
| Symdeko (tezacaftor/ivacaftor) Prescribing Information. | 2023 | Vertex Pharmaceuticals Incorporated, Boston, MA |
Systematic Reviews / Meta-Analyses
| Document Title | Year | Source |
|---|---|---|
| Newly Discovered Cutting-Edge Triple Combination Cystic Fibrosis Therapy: A Systematic Review | 2022 | Cureus |
| Effect of CFTR Modulators on Anthropometric Parameters in Individuals with Cystic Fibrosis: An Evidence Analysis Center Systematic Review | 2021 | Journal of the Academy of Nutrition and Dietetics |
| Real-World Safety of CFTR Modulators in the Treatment of Cystic Fibrosis: A Systematic Review | 2020 | Journal of Clinical Medicine |
| Corrector therapies (with or without potentiators) for people with cystic fibrosis with class II CFTR gene variants (most commonly F508del) | 2020 | The Cochrane Database of Systematic Reviews |
| From Ivacaftor to Triple Combination: A Systematic Review of Efficacy and Safety of CFTR Modulators in People with Cystic Fibrosis | 2020 | International Journal of Molecular Sciences |
Clinical Practice Guidelines
| Document Title | Year | Source |
|---|---|---|
| Brazilian guidelines for the pharmacological treatment of the pulmonary symptoms of cystic fibrosis. Official document of the Sociedade Brasileira de Pneumologia e Tisiologia (SBPT, Brazilian Thoracic Association) | 2023 | Jornal Brasileiro de Pneumologia |