Vertex : FDA Okays Kalydeco For Children With Cystic Fibrosis Ages 2 To 5

(RTTNews) - Vertex Pharmaceuticals Inc. (VRTX) announced Wednesday that the U.S. Food and Drug Administration approved Kalydeco for use in children ages 2 to 5 with cystic fibrosis or CF who have one of 10 mutations in the cystic fibrosis transmembrane conductance regulator or CFTR gene (G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, S549R and R117H).

Prior to today's approval, Kalydeco was approved in the United States for people ages 6 and older with these mutations.

There are approximately 300 children in the United States ages 2 to 5 who have one of these 10 mutations, including 150 who have the R117H mutation and 150 who have one of the other nine mutations that result in a gating defect in the CFTR protein.

A new weight-based oral granule formulation of Kalydeco (50 mg and 75 mg) that can be mixed in soft foods or liquids was created to meet the needs of children in this age group who may be unable to swallow a tablet.

The approval is based on previously announced results of an open-label Phase 3 24-week 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.

Cystic fibrosis is caused by a defective or missing CFTR protein resulting from mutations in the CFTR gene.

In Europe, an MAA line extension for ivacaftor in children ages 2 to 5 with specific mutations in the CFTR gene has been validated by the European Medicines Agency (EMA) and is currently under review by the Committee for Medicinal Products for Human Use (CHMP).

Kalydeco is approved in the U.S., Europe, Canada, Australia and New Zealand to treat people with CF who have specific genetic mutations in the CFTR gene.

Vertex said it retains worldwide rights to develop and commercialize Kalydeco.