Posted by Michael Wonder on 29 Sep 2016
U.S. FDA approves Orkambi (lumacaftor/ivacaftor) for use in children with cystic fibrosis ages 6 through 11 who have two copies of the F508del mutation
28 September 2016 - Approximately 2,400 children ages 6 through 11 have two copies of the F508del mutation in the U.S.
Vertex Pharmaceuticals today announced that the U.S. FDA approved Orkambi (lumacaftor/ivacaftor) for use in children with cystic fibrosis (CF) ages 6 through 11 who have two copies of the F508del mutation. People with this mutation represent the largest population of those with CF, a rare, life-threatening disease.
Orkambi is the first and only medicine to treat the underlying cause of CF for people with this mutation. It was previously approved by the FDA for use in people ages 12 and older with two copies of the F508del mutation. With today's approval, approximately 11,000 people with CF are eligible for treatment with Orkambi in the United States.
Orkambi will be available for eligible children ages 6 through 11 in the United States as soon as possible.
