Vertex: Health Canada Grants Market Authorization For ORKAMBI In Young Children

(RTTNews) - Vertex Pharmaceuticals Inc. (VRTX) announced that Health Canada has granted Market Authorization for ORKAMBI (lumacaftor/ivacaftor) to include use in children ages 2 through 5 years with cystic fibrosis who have two copies of the F508del CFTR mutation. Approval is based on a Phase 3 open-label safety study in 60 patients that showed treatment with ORKAMBI was generally well tolerated for 24 weeks, with a safety profile similar to that in patients ages 6-11 years.

ORKAMBI was approved by the U.S. FDA in August for use in patients ages 2 to 5 years who have two copies of the F508del CFTR mutation.