Vertex's Kalydeco approved for CF

The Food and Drug Administration approved Vertex Pharmaceuticals Inc.'s Kalydeco (ivacaftor), the first drug developed to treat the underlying cause of cystic fibrosis (CF). Kalydeco is indicated for patients aged 6 years or older who have CF and at least one copy of the G551D mutation in the CF transmembrane conductance regulator (CFTR) gene. The therapy, which was designated as an orphan drug, was approved under the FDA's priority review program ahead of the targeted Prescription...

The Food and Drug Administration approved Vertex Pharmaceuticals Inc.'s Kalydeco (ivacaftor), the first drug developed to treat the underlying cause of cystic fibrosis (CF).

Kalydeco is indicated for patients aged 6 years or older who have CF and at least one copy of the G551D mutation in the CF transmembrane conductance regulator (CFTR) gene.

The therapy, which was designated as an orphan drug, was approved under the FDA's priority review program ahead of the targeted Prescription Drug User Fee Act goal date of April 18.

The approval was supported by findings from two Phase III trials--STRIVE and ENVISION--in which patients with CF who had at least one copy of the G551D mutation were treated with Kalydeco or placebo. Data showed that the Kalydeco-treated patients experienced significant and sustained improvements in lung function and other disease measures, including weight gain and certain quality-of-life measurements, when compared with the patients who received placebo.

The patients who received Kalydeco also experienced significantly fewer pulmonary exacerbations, which are periods of worsening in the signs and symptoms of CF that often require treatment with antibiotics and hospital visits. Moreover, fewer individuals in the Kalydeco treatment groups than in the placebo groups discontinued treatment because of adverse events.

The company is seeking approval to market the drug in Europe.