Elexacaftor-Tezacaftor-Ivacaftor: The First Triple-Combination Cystic Fibrosis Transmembrane Conductance Regulator Modulating Therapy
Elexacaftor-tezacaftor-ivacaftor is a recently approved triple-combination therapy for cystic fibrosis that includes two correctors and a potentiator of the CFTR channel. It is indicated for individuals aged 12 and older who carry at least one F508del mutation in the CFTR gene. This combination offers a new treatment option for many patients who were previously ineligible for CFTR modulation therapies due to their genetic mutations. Clinical trials have shown that it outperforms existing therapies in improving lung function, quality of life, reducing sweat chloride levels, and decreasing exacerbations. The most commonly reported side effects in trials were rash and headache, and liver function monitoring is recommended. Ongoing patient data evaluation is essential to confirm the long-term safety and efficacy of this treatment. While Elexacaftor-tezacaftor-ivacaftor represents a significant advancement for cystic fibrosis care, about 10% of the CF population cannot benefit from this or other CFTR modulation therapies.