U.S. health regulators expanded the use of Vertex Pharmaceuticals Inc.’s cystic fibrosis drug Kalydeco to include patients aged between 12 months and 24 months.
The FDA approved Vertex Pharmaceuticals’ Orkambi (lumacaftor/ivacaftor) to include use in children ages 2-5 years with cystic fibrosis who have two copies of the F508del-CFTR mutation.
Vertex Pharmaceuticals Incorporated announced that the European Commission granted extension of the Marketing Authorization for Orkambi (lumacaftor/ivacaftor), the first medicine to treat the underlying cause of cystic fibrosis in people with two copies of the F508del mutation, to include children ages 6 through 11.
An anti-inflammatory drug being developed by Corbus Pharmaceuticals Holdings as an add-on treatment for cystic fibrosis demonstrated promising safety and was well tolerated in a small midstage study, according to data released by the company.
Vertex Pharmaceuticals Inc. said its Kalydeco cystic fibrosis treatment given with an experimental drug demonstrated significant improvements in lung function in a pair of late-stage trials the company plans to use to seek approval of the combination therapy.
Shares of Raptor Pharmaceutical Corp. were up after Horizon Pharma announced it had struck a deal to acquire the rare disease company for $800 million.