Vertex Pharmaceuticals, Inc., announced today that the U.S. Food and Drug Administration (
The company is seeking approval of the drug for people ages 6 and older with the G551D mutation of CF.
The expedited review sets a target date of April 18, 2012, for the FDA's approval decision, four months earlier than the standard review time of 10 months.
If approved, Kalydeco (kuh-LYE-deh-koh) will be the first drug available that targets the underlying cause of CF. The FDA grants priority review for several reasons, including situations where a potential drug offers a major advance in treatment.
Kalydeco was discovered in a collaboration between Vertex and the Cystic Fibrosis Foundation, which provided substantial financial support throughout the development process.
Vertex's application for approval of Kalydeco, submitted to the FDA in October 2011, included results from Phase 3 clinical trials of the drug in people ages 6 and older with the G551D mutation of CF. The results showed that those receiving the drug had remarkable and sustained improvements in
- Read the Vertex press release.