Vertex wins expanded pediatric use of CF drug Kalydeco
This article was originally published in Scrip
Executive Summary
Vertex Pharmaceuticals said on 18 March the FDA had expanded the use of the company's cystic fibrosis drug Kalydeco (ivacaftor) in children 2-5 years who have one of 10 mutations in the CF transmembrane conductance regulator (CFTR) gene: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, S549R and R117H.