CF

Vertex Pharmaceuticals has received approval from the US Food and Drug Administration (FDA) for its Kalydeco (ivacaftor) to treat cystic fibrosis (CF) in children aged between two and five years.

The approval allows the drug to treat children with CF who have one of ten mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene (G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, S549R and R117H).

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Earlier, the drug was approved in the US to treat children aged six and older with these mutations. It is also approved in Europe, Canada, Australia and New Zealand to treat people with CF who have specific genetic mutations in the CFTR gene.

"Children with cystic fibrosis can begin to experience meaningful lung function decline and struggle to gain weight at a very young age, underscoring the importance of starting treatment early in life."

Vertex Pharmaceuticals executive vice-president and chief medical officer Dr Jeffrey Chodakewitz said: "Children with cystic fibrosis can begin to experience meaningful lung function decline and struggle to gain weight at a very young age, underscoring the importance of starting treatment early in life."

The approval was based on an open-label Phase III 24-week study, which was designed to assess the safety and pharmacokinetics of weight-based dosing of ivacaftor (50mg or 75mg twice-daily) in children aged two to five.

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In the US, Ivacaftor is available as 50mg and 75mg oral granules for patients aged two and below six years, while it is available as 150mg tablets in countries where it is approved for patients aged six years and older.

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European Medicines Agency (EMA) has validated a marketing authorisation application (MAA) line extension for ivacaftor in children aged two to five with specific mutations in the CFTR gene, and is currently under review by the Committee for Medicinal Products for Human Use (CHMP).

Image: Clubbing of the fingers is a feature of cystic fibrosis. Photo: courtesy of Jerry Nick, MD.