Cystic fibrosis is caused by mutations in the CFTR gene, which makes a protein that moves salt and water across cell surfaces. CFTR modulators are oral drugs that help that defective protein work better — “correctors” help it fold and reach the cell surface, and “potentiators” help the channel open. The triple therapy elexacaftor/tezacaftor/ivacaftor (Trikafta) is effective for most people with at least one F508del mutation and has substantially improved lung function, weight, and quality of life; ivacaftor (Kalydeco) alone treats certain gating mutations. Eligibility depends on a person’s specific CFTR mutations.
Taken by mouth every day, on an ongoing basis. Doses are usually taken with fat-containing food to help absorption. Which modulator is prescribed depends on the person’s CFTR genotype, and treatment is monitored by the CF care team.
Modulators are a long-term, disease-modifying therapy — not a one-time treatment or a cure. Most eligible patients see improved lung function and nutrition, but the CF care team continues to monitor liver tests, eye exams (in children), and drug interactions, and other daily CF therapies are still needed.