EU label expansion lowers age range for Vertex's Kalydeco

11 December 2019
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US drugmaker Vertex Pharmaceuticals (Nasdaq: VRTX) has announced that the European Commission has granted a label extension for Kalydeco (ivacaftor) to allow treatment of certain cystic fibrosis (CF) patients aged from six to 12 months.

These children will be deemed suitable if they weigh 5kg or more and if they have at least one of the following nine mutations in their CF transmembrane conductance regulator (CFTR) gene: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N or S549R.

This approval makes Kalydeco the first and only approved medicine in Europe to treat the underlying cause of CF in children this young. It was previously available for certain patients aged from 12 months upwards.

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