FDA approves Orkambi (lumacaftor/ivacaftor) for use in children with CF (F508del mutation) aged 6-11.- Vertex

Vertex Pharmaceuticals Incorporated announced that the 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 this 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.

Vertex also today lowered its guidance for 2016 Orkambi revenues to a range of $950 million to $990 million.