FDA approval for Vertex’ Kalydeco for very young children

There was good news for US biotech firm Vertex Pharmaceuticals (Nasdaq: VRTX) yesterday, when the US Food and Drug Administration granted approval Kalydeco (ivacaftor) for use in children ages two to five with cystic fibrosis (CF) who have one of 10 mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene (G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, S549R and R117H).

Prior to this approval, Kalydeco was cleared in the USA for people aged six and older with these mutations. In 2014, Kalydeco generated sales of $464 million for Vertex, which forecasts that, with continued label and geographic expansion, net revenues for full-year 2015 will grow to $560-$580 million.

There are around 300 children in the USA aged two to five who have one of these 10 mutations, including 150 who have the R117H mutation and 150 who have one of the other nine mutations that result in a gating defect in the CFTR protein. A new weight-based oral granule formulation of Kalydeco (50mg and 75mg) that can be mixed in soft foods or liquids was created to meet the needs of children in this age group who may be unable to swallow a tablet.