Vertex Gets Euro CHMP Positive Opinion for Kalydeco Expanded Pediatric Use

Vertex Pharmaceuticals Inc. on Friday said the European Medicines Agency’s Committee for Medicinal Products for Human Use recommended expanded approval of Kalydeco to include the treatment of children and adolescents with the most common cystic fibrosis genetic mutation.
The European Commission, which generally follows the CHMP’s recommendations, will now review the positive opinion, which covers patients ages six months and older who have the R117H mutation in the cystic fibrosis transmembrane conductance regulator gene, the Boston drug maker said.
Vertex said Kalydeco, if approved, will be the first and only medicine in Europe to treat the underlying cause of cystic fibrosis in patients with the R117H mutation, the most common residual function mutation in the progressive, genetic disease that causes persistent lung infections.
Kalydeco is already approved in Europe for adults with the R117H mutation and for children with several other mutations.

https://www.marketscreener.com/VERTEX-PHARMACEUTICALS-11321/news/Vertex-Pharmaceuticals-Gets-European-CHMP-Positive-Opinion-for-Kalydeco-Expanded-Pediatric-Use-30510612/