Posted by Michael Wonder on 07 Aug 2018
FDA approves Orkambi (lumacaftor/ivacaftor) as first medicine to treat the underlying cause of cystic fibrosis for children ages 2-5 years with most common form of the disease
7 August 2018 - Approximately 1,300 people in the U.S. ages 2 through 5 years have two copies of the F508del mutation, the most common genetic form of the disease.
Vertex Pharmaceuticals today announced the U.S. FDA has approved Orkambi (lumacaftor/ivacaftor) to include use in children ages 2 through 5 years with cystic fibrosis (CF) 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 (lumacaftor 100mg/ivacaftor 125mg and lumacaftor 150mg/ivacaftor 188mg) for weight-based dosing. Orkambi oral granules should be available for fulfillment within 2 to 4 weeks.
