The European Medicines Agency has recommended Kalydeco (ivacaftor), an orphan-designated medicine, for the treatment of cystic fibrosis in patients age 6 years and older who have a G551D mutation in the cystic fibrosis transmembrane regulator (CFTR) gene. The Agency’s Committee for Medicinal Products for Human Use (CHMP) reviewed Kalydeco under accelerated assessment, in 150 days. Accelerated assessment is one of the Agency’s tools to speed up access by patients to new medicines that are of major public health interest.
Kalydeco offers an innovative therapeutic approach for patients with cystic fibrosis: it is the first treatment that targets the underlying mechanism of the disease, by restoring the function of the mutated CFTR protein. Currently available therapies for patients with cystic fibrosis only address the consequences of the disease, not the underlying defect. Clinical studies showed that Kalydeco improved pulmonary function in cystic fibrosis patients with the specific G551D-CFTR mutation.