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Vertex scores EU approval for cystic fibrosis drug Kalydeco in R117H+ children and adolescents

Published on 10/06/20 at 12:33pm

Vertex has revealed that it has secured a new approval from the European Commission for its therapy Kalydeco (ivacaftor) in the treatment of cystic fibrosis (CF) in children and adolescents.

The approval relates specifically to patients at least six months old who weigh at least 5kg whose cystic fibrosis transmembrane conductance regulator (CFTR) gene features the most common residual function mutation, R117H.

The decision means that the therapy will now be made available to patients in this population with immediate effect in Germany, with access to follow promptly in countries “where respective long-term reimbursement agreements have been previously secured”. Vertex confirmed it will now be liaising with government authorities to provide access to all other eligible patients across the EU as soon as possible.

The ruling adds this latest approval to those Kalydeco already has under its belt in Europe, including for R117H patients over the age of 18, and in patients at least six months old, weighing at least 5kg, with either the G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N or S549R mutations in their DFTR gene.

“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,” said Dr Reshma Kewalramani, Chief Executive Officer and President at Vertex. “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.”

Matt Fellows

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