Elexacaftor Added to Current Therapies Provides Benefit for Most Patients with Cystic Fibrosis

Two randomized, controlled studies show that elexacaftor added to tezacaftor and ivacaftor improves lung function and quality of life in patients with the most common mutation, F508del.

Current targeted drugs for cystic fibrosis are not effective for patients who are heterozygous for the common genetic defect, F508del (also called Phe508del), which impairs packaging and transport of the cystic fibrosis transmembrane conductance regulator (CFTR) protein to the membrane surface. In two multinational, industry-funded, phase 3 randomized, controlled trials, researchers evaluated combined therapy with the next-generation CFTR corrector, elexacaftor (200 mg daily), plus the two FDA-approved therapies, tezacaftor (100 mg daily) and ivacaftor (150 mg twice per day), in patients ≥12 years old with the F508del mutation and percent predicted FEV₁ of 40% to 90%. The primary outcome was the change in ppFEV₁ from baseline to 4 weeks.

In …

Reviewing Author

F. Bruder Stapleton, MD

Disclosures

Consultant/Advisory BoardCareer Physician

Editorial BoardsUpToDate

Leadership Positions in Professional SocietiesAmerican Society of Pediatric Nephrology Foundation (Chair)

F. Bruder Stapleton, MD

Disclosures

Consultant/Advisory BoardCareer Physician

Editorial BoardsUpToDate

Leadership Positions in Professional SocietiesAmerican Society of Pediatric Nephrology Foundation (Chair)

Elexacaftor Added to Current Therapies Provides Benefit for Most Patients with Cystic Fibrosis

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Reviewing Author