Vertex Wins Expanded Indication for Kalydeco from EC

Vertex Pharmaceuticals got double good news from the European Commission, which has blessed an expanded indication for Kalydeco, a treatment for cystic fibrosis.

Kalydeco (ivacaftor) now may be used in children ages 2 to 5 who have one of nine gating mutations in the cystic fibrosis transmembrane conductance regulator gene. In addition, the drug now is indicated for cystic fibrosis patients ages 18 and older with an R117H mutation.

Kalydeco, a CFTR potentiator, previously was approved in the EU for cystic fibrosis patients ages 6 and older with one of nine gating mutations.

Boston-based Vertex says it will begin the country-by-country reimbursement approval processes for each new indication. Roughly 125 children in Europe with cystic fibrosis ages 2 to 5 have one of the nine gating mutations, while roughly 350 adults with cystic fibrosis have an R117H mutation.