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How does Kalydeco work?

Medically reviewed by Carmen Pope, BPharm. Last updated on May 4, 2023.

Official answer

by Drugs.com

Kalydeco specifically targets the underlying cause of cystic fibrosis (CF) and is approved for adults, children, and infants aged 4 months and older with CF with one or more specific mutations in their CFTR gene that is responsive to Kalydeco. It allows the defective CFTR protein to work better and increases salt and water movement within the airways. This thins the mucus and makes it easier to cough out.

Research demonstrates that Kalydeco markedly improves lung function, reduces salt levels in sweat, and helps people with CF gain weight. It is too early to say whether it will cure people with CF.

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