CHMP recommends Kalydeco (ivacaftor) for children 2 to 5 with cystic fibrosis- Vertex

The CHMP has issued a positive opinion recommending the indication of Kalydeco (ivacaftor) from Vertex, be expanded to include children ages 2 to 5 with CF, in addition to the current approved use in people ages 6 and older, who have one of nine gating mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene (G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N and S549R). A new weight-based oral granule formulation of ivacaftor (50 mg and 75 mg) that is mixed in soft foods or liquids was created for these younger children. The CHMP opinion for the use of ivacaftor in children ages 2 to 5 included a recommendation to extend weight-based dosing of ivacaftor to children ages 6 to 11 who weigh less than 25 kg using the new oral granule formulation. In addition, the CHMP issued a positive opinion recommending ivacaftor for use in people with CF ages 18 and older who have the R117H mutation. In Europe , approximately 125 children ages 2 to 5 have one of the nine gating mutations included in the positive opinion, and approximately 250 adults have the R117H mutation.