US FDA expands use of Vertex Pharma's Orkambi; revised guidance

The US Food and Drug Administration has approved Vertex Pharmaceuticals’ (Nasdaq: VRTX) Orkambi (lumacaftor/ivacaftor) for use in children with cystic fibrosis (CF) ages six through 11 who have two copies of the F508del mutation.

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 aged 12 and older with two copies of the F508delmutation. With today’s approval, about 11,000 people with CF are eligible for treatment with Orkambi in the USA.

“The ability to treat children as young as six who have the most common form of the disease is an important milestone as we pursue our goal to develop medicines for all people with CF,” said Jeffrey Chodakewitz, executive vice president and chief medical officer at Vertex.