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FDA Expands Labeling for Kalydeco (Ivacaftor) to Include Children Ages 2 to 5 With Cystic Fibrosis

Drug previously approved for patients 6 years of age and older

The FDA has approved Kalydeco (ivacaftor, Vertex Pharmaceuticals) for use in children aged 2 to 5 years with cystic fibrosis (CF) who have one of 10 mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene (G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, S549R, and R117H). Before the new approval, Kalydeco was indicated in the U.S. for CF patients 6 years of age and older with these mutations.

Approximately 300 children aged 2 to 5 years in the U.S. have one of these 10 mutations, including 150 who have the R117H mutation and 150 who have one of the other nine mutations, which 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 the 2- to 5-year age group who may be unable to swallow a tablet.

The new approval was based on results from an open-label, phase III, 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 aged 2 to 5 years.

In the U.S., ivacaftor is now indicated for patients aged 2 years and older who have one of the following mutations in the CFTR gene: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, or S549R. In addition, ivacaftor is indicated for the treatment of CF in patients 2 years of age and older who have an R117H mutation in the CFTR gene.

Ivacaftor is not effective in CF patients with two copies of the F508del mutation (F508del/F508del) in the CFTR gene. The safety and efficacy of ivacaftor in children younger than 2 years of age with CF have not been studied. The use of ivacaftor in children under the age of 2 years is not recommended.

CF is a rare, life-threatening genetic disease affecting approximately 75,000 people in North America, Europe, and Australia. The median predicted age of survival for a person with CF is between 34 and 47 years, but the median age of death remains in the mid-20s.

CF is caused by a defective or missing CFTR protein resulting from mutations in the CFTR gene. Children must inherit two defective CFTR genes — one from each parent — to develop CF. There are more than 1,900 known mutations in the CFTR gene. Some of these mutations, which can be determined by genotyping, lead to CF by creating non-working or too- few CFTR proteins at the cell surface. The defective function or absence of CFTR proteins in individuals with CF results in poor flow of salt and water into and out of cells in several organs. In the lungs, this leads to the buildup of abnormally thick, sticky mucus that can cause chronic lung infections and progressive lung damage.

Source: Vertex Pharmaceuticals; March 18, 2015.

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