Vertex announces FDA approval of ORKAMBI to treat cystic fibrosis
Vertex announced the U.S. FDA has approved ORKAMBI to include use in children ages 2 through 5 years with cystic fibrosis who have two copies of the F508del-CFTR mutation, making it the first medicine approved to treat the underlying cause of CF in this population. ORKAMBI oral granules are available in two dosage strengths for weight-based dosing. ORKAMBI oral granules should be available for fulfillment within 2 to 4 weeks. ORKAMBI was already approved in the U.S. for the treatment of CF in patients ages 6 and older who have two copies of the F508del-CFTR mutation. A Marketing Authorization Application line extension for ORKAMBI in children ages 2 through 5 years has been submitted to the European Medicines Agency with a decision anticipated in the first half of 2019.