Vertex gets EC nod for Kalydeco to treat children and adolescents with cystic fibrosis

Vertex Pharmaceuticals Incorporated announced that the European Commission has granted approval of the label extension for KALYDECO (ivacaftor) to include the treatment of children and adolescents with cystic fibrosis (CF), ages 6 months and older and weighing at least 5 kg, who have the R117H mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene – the most common residual function mutation underlying CF.

“A little over eight years ago, KALYDECO was approved as the first and only medicine to treat the underlying cause of cystic fibrosis in patients with specific mutations,” said Reshma Kewalramani, M.D., Chief Executive Officer and President at Vertex. “Since then, it’s been our goal to ensure that as many people with CF as possible are able to benefit from our treatments, and today’s label extension means that approximately 500 young patients in Europe, who have long awaited a treatment option, are now eligible for KALYDECO^®.”

Now approved, KALYDECO (ivacaftor) will be immediately available to additional eligible patients in Germany and shortly in countries where respective long-term reimbursement agreements have been previously secured. Vertex will work closely with all other relevant government authorities to secure access for eligible patients as quickly as possible.

In Europe, KALYDECO (ivacaftor) is already approved for the treatment of people with CF ages 18 and older with the R117H mutation, and in infants ages 6 months and older weighing at least 5 kg who have one of the following mutations in the CFTR gene: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N or S549R.