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.