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Vertex's Kalydeco seizes EU CHMP recommendation for Kalydeco in R117H+ cystic fibrosis

Published on 01/05/20 at 10:50am

The European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has given its recommendation for the approval of Kalydeco (ivacaftor) for the treatment of children and adolescents with cystic fibrosis (CF) in a new indication, Vertex has revealed.

The recommended indication specifically relates to CF patients of at least six months old who weigh at least 5 kg and have the R117H mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.

Should the European Commission agree with the Committee’s ruling, resulting approval would make Kalydeco the first and only available treatment in Europe for the underlying causes of CF in R117H-positive patients of at least six months old.

“Today’s announcement is important for young people with CF, as early intervention and treatment of this devastating and progressive disease is key to keeping patients healthier longer,” said Dr Carmen Bozic, Executive Vice President and Chief Medical Officer at Vertex. “This milestone also brings us one step closer to achieving our ultimate goal of bringing medicines forward to all people with CF.”

Kalydeco is already authorised in Europe for the treatment of R117H-positive CF patients of at least 18 years old, and in children at least six months old and weighing at least 5kg with G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N or S549R mutations in the CTFR gene.

Vertex confirmed that the therapy would be immediately available to patients in Germany in the event of approval, with availability on other European countries to follow as the company works with the relevant regulatory bodies.

Matt Fellows

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