CHMP positive opinion for Kalydeco for the treatment of infants with cystic fibrosis ages 1 month and older – Vertex Pharma

Vertex Pharmaceuticals announced that the European Medicines Agency’s (EMA’s) Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion for the label expansion of Kalydeco (ivacaftor) for the treatment of infants with cystic fibrosis (CF) ages 1 month to less than 4 months old who have one of the following mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene: R117H, G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N or S549R.

“Cystic fibrosis is a chronic, progressive disease that is present from birth, which is why it is important to treat the underlying cause of this disease as early as possible to potentially slow disease progression,” said Fosca De Iorio, Vice President, International Medical Affairs at Vertex. “If the label expansion for Kalydeco is approved, this would allow eligible infants with CF to receive a treatment that targets the underlying cause of their disease at the very start of their lives.”

In the European Union, Kalydeco is already approved for the treatment of people with CF ages 4 months old and above with specific mutations in the CFTR gene.