Following successful clinical results, Vertex Pharmaceuticals Inc. recently gained approval from the U.S. Food and Drug Administration (FDA) to treat more than 600 cystic fibrosis (CF) patients with its proprietary Kalydeco (ivacaftor) product.
Each of the individuals concerned — all of whom were at least 2 years of age — demonstrated one of five specific residual function mutations impacting the gene responsible for CF with “a splicing defect.”
"In the five years since Kalydeco became the first approved medicine to treat the underlying cause of cystic fibrosis, we have been relentless in our efforts to bring this important medicine to all who may benefit," Dr. Jeffrey Chodakewitz, executive vice president and chief medical officer at Vertex, said. "We will continue to pursue this goal until all people with CF have a medicine that treats their form of this serious and life-shortening disease."
The FDA had previously approved Boston-based Vertex’s Kalydeco for 23 other such mutations in May, based on in vitro data. CF derives from defective or missing cystic fibrosis transmembrane conductance regulator (CFTR) proteins.
Kalydeco is administered orally in 150 mg tablet doses for adults and pediatric patients age 6 years and older and also dispensed as 50 mg and 75 mg granules for pediatric patients ages 2 to 5.
Cystic fibrosis is a rare but life-threatening disorder primarily affecting individuals in North America, Europe and Australia.