Drug updated on 11/1/2024
| Dosage Form | Tablet (oral; 150 mg); Granule (oral; 5.8 mg, 13.4 mg, 25 mg, 50 mg, 75 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 1 month and older who have at least one mutation in the CFTR gene that is responsive to ivacaftor based on clinical and/or in vitro assay data
- If the patients genotype is unknown, an FDA-cleared CF mutation test should be used to detect the presence of a CFTR mutation followed by verification with bi-directional sequencing when recommended by the mutation test instructions for use.
Latest News
Summary
- This summary is based on the review of eight systematic review(s)/meta-analysis(es). [1-8]
- FEV1% Improvement: Elexacaftor/Tezacaftor/Ivacaftor (ETI) increased FEV1% by +10.47% (95% CI (confidence interval), 6.88-14.06) and showed significant improvements for F508del/MF and F508del/F508del genotypes; both dual (Lumacaftor-Ivacaftor, Tezacaftor-Ivacaftor) and triple therapies significantly improved lung function.
- Pulmonary Exacerbations and Quality of Life: ETI reduced pulmonary exacerbations (RD, -0.16; 95% CI, -0.28 to -0.04) and improved quality of life (RD, +14.93; 95% CI, 9.98-19.89); dual and triple therapies consistently lowered exacerbation rates and increased quality of life scores.
- Body Mass Index (BMI) and Other Outcomes: ETI increased BMI (+1.07 kg/m²; 95% CI, 0.90-1.25), with Ivacaftor and triple therapy also showing BMI gains; CFTR modulators, including Ivacaftor, reduced sweat chloride concentration, Pseudomonas aeruginosa prevalence, and healthcare utilization.
- Elexacaftor-Tezacaftor-Ivacaftor (ETI) showed no significant difference in adverse events compared to control (RD, -0.03; 95% CI, -0.08 to 0.01), while Ivacaftor maintained a consistent safety profile. Higher rates of respiratory-related adverse events and discontinuations were observed in real-world studies with Lumacaftor-Ivacaftor.
- Neurocognitive and mental health adverse events were reported across all CFTR (cystic fibrosis transmembrane conductance regulator) modulators, with mild to moderate adverse events common with ETI. Triple therapy adverse events were similar to placebo and control groups, and Tezacaftor-Ivacaftor had a better safety profile compared to Lumacaftor-Ivacaftor.
- There is no population types or subgroups information available in the reviewed studies.
Product Monograph / Prescribing Information
| Document Title | Year | Source |
|---|---|---|
| Kalydeco (ivacaftor) Prescribing Information. | 2023 | Vertex Pharmaceuticals Incorporated, Boston, MA |