EU expands approval of Kalydeco (ivacaftor) to include nine gating mutations in the cystic fibrosis (CFTR) gene (G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N and S549R)- Vertex Pharmaceuticals

The European Commission has approved expansion of the indication for Kalydeco (ivacaftor), from Vertex Pharmaceuticals, to include children ages 2 to 5 with cystic fibrosis (CF) 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). Ivacaftor was previously approved in the European Union (EU) for use in people with CF ages 6 and older who have one of nine gating mutations.

The European Commission�s Decision is based on previously announced results of a 24-week open-label Phase III study that was designed to evaluate the safety and pharmacokinetics of weight-based dosing of ivacaftor (50 mg or 75 mg twice daily) in children ages 2 to 5. A weight-based oral granule formulation of ivacaftor that is mixed in soft foods or liquids was created for these younger children and is available in two strengths, 50 mg and 75 mg. The approval also includes an extension of weight-based dosing of ivacaftor to children ages 6 to 11 who weigh less than 25 kg, using the new oral granule formulation.