Vertex reports pediatric Orkambi data, earnings

Vertex Pharmaceuticals Inc. (NASDAQ:VRTX) reported earnings Wednesday and said Orkambi ivacaftor/lumacaftor met the primary safety endpoint but missed a secondary efficacy endpoint in a Phase III trial to treat cystic fibrosis in patients aged 6-11 with two copies of the F508 mutation of the CFTR gene.

Next quarter, the company plans to submit an sNDA to FDA that would expand Orkambi's label to include the age group. Vertex said about 2,400 U.S. children aged 6-11 are homozygous for the mutation. The drug is approved to treat CF in patients aged 12 and older homozygous for the mutation. ...