The FDA has also granted the company’s request for six-month Priority Review of Kalydeco, acts by targeting the defective protein that causes CF.
The move follows the results from two Phase 3 studies, STRIVE and ENVISION, in which people with CF who had at least one copy of the G551D mutation and were treated with Kalydeco showed sustained improvements across a variety of disease measures, including lung function.
Vertex Global Research and Development chief scientific officer and executive vice president Peter Mueller said if approved, Kalydeco will be the first treatment to target the underlying cause of CF.
"The commitments by the FDA and the EMA to expedite their reviews of our applications underscore the significant potential of KALYDECO to help people living with cystic fibrosis," Mueller added.
The company announced that its marketing authorization application (MAA) for Kalydeco has been accepted by the European Medicines Agency (EMA).