Takeaway
- The triple-hit combination of elexacaftor-tezacaftor-ivacaftor shows efficacy in patients carrying the Phe508del variant of the cystic fibrosis gene, which confers minimal function.
- Results of this 24-week trial included better lung function, fewer exacerbations, higher BMI.
Why this matters
- Almost 9 in 10 patients with cystic fibrosis carry this variant.
Key results
- FEV1 was 13.8 points higher at 4 weeks, 14.3 points higher at 24 weeks in the drug group vs placebo.
- Pulmonary exacerbation rate was 63% lower with drug vs placebo.
- QoL scores were higher with drug vs placebo.
- Sweat chloride concentrations were lower vs placebo.
- All of these comparisons were significant, favoring the drug (P<.001).
- Side-effect profile was “acceptable,” and adverse events were largely mild-moderate, although common (at least 1 event in 93.1% with drug vs 96.0% with placebo).
- 9.9% in drug group had a serious event vs 8.0% in placebo, excluding pulmonary exacerbation.
- 1% discontinued in the drug group.
Study design
- Phase 3 randomized, placebo-controlled, double-blind trial.
- 405 patients included, age 12 years or more, at 115 sites.
- Funding: Vertex Pharmaceuticals.
Limitations
- Selected patient population in a clinical trial; effects for noncarriers of this variant unknown.
References
References
