Expanded label for Vertex' Kalydeco approved in EUExpanded label for Vertex' Kalydeco approved in EU

The European Commission has approved expansion of the indication for Kalydeco (ivacaftor) from US biotech firm Vertex Pharmaceuticals (Nasdaq: VRTX).

The approved uses for Kalydeco now include children ages two to five 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) and to include people with CF ages 18 and older who have an R117H mutation.

Ivacaftor, the first medicine to treat the underlying cause of CF in people with specific mutations in the CFTR gene, was previously approved in the European Union for use in people with CF ages six and older who have one of nine gating mutations. In Europe, around 125 children with CF ages two to five have one of the nine gating mutations included in today’s approval and approximately 350 adults with CF have an R117H mutation. Vertex will now begin the country-by-country reimbursement approval processes for each new indication.