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Kalydeco Approved in Europe to Treat Children with R117H Mutation

The European Commission has approved Vertex’s Kalydeco (ivacaftor) for the treatment of children and adolescents with cystic fibrosis (CF) who carry the R117H mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, which is defective in CF.

Kalydeco previously was approved in Europe for children (6 months and older) and adolescents with at least one of nine gate-closing mutations: G551D, G1244E, G1349D, S549R, G178R, S1251N, S1255P, S549N, and G551S. The therapy also is approved for CF patients with the R117H mutation who are 18 and older.

The new label extension allows patients 6 months and older with the R117H mutation and who weigh at least five kilograms (about 11 pounds) to also be treated with the therapy.

“A little over eight years ago, Kalydeco was approved as the first and only medicine to treat the underlying cause of cystic fibrosis in patients with specific mutations,” Reshma Kewalramani, MD, CEO and president at Vertex, said in a press release.

“Since then, it’s been our goal to ensure that as many people with CF as possible are able to benefit from our treatments, and today’s label extension means that approximately 500 young patients in Europe, who have long awaited a treatment option, are now eligible for Kalydeco,” Kewalramani said.

Kalydeco is known as a CFTR potentiator therapy. The CFTR gene carries the instructions for producing the CFTR protein, which is a channel on cell membranes allowing the passage of water molecules and chloride ions, working like a gate. In some CF-causing mutations, such as R117H, the CFTR gate can become fixed in the closed position, partially blocking the flow and leading to airway mucus becoming thick and sticky.

CFTR potentiators act by holding the gate open long enough to facilitate the transport of chloride ions across the channel, which helps to hydrate and clear the mucus.

With this approval, Kalydeco will me made available immediately to eligible patients in Germany, and soon in other counties based on long-term reimbursement agreements with Vertex. The company is working closely with other government authorities to ensure access to Kalydeco for eligible patients as quickly as possible.

In the U.S., Kalydeco was approved in 2012 and also is available now for use in CF patients with the R117H mutation, aged 6 months and older.

In a recent real-world, five-year analysis of CF patients in the U.S. and the United Kingdom, Kalydeco treatment led to fewer exacerbations, hospitalizations, transplants, and deaths compared to those who did not receive the therapy.

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