FDA approves broader use of CF drug Orkambi

The US Food and Drug Administration as approved Orkambi (lumacaftor/ivacaftor) to include use in children ages two through five years with cystic fibrosis (CF) who have two copies of the F508del-CFTR mutation.

This makes Orkambi the first medicine approved to treat the underlying cause of CF in this population, says the drug’s marketer, US biotech firm Vertex Pharmaceuticals (Nasdaq: VRTX), which has come under fire in some European countries, particularly the UK, over the high price of the drug, which in 2017 generated sales of $1.32 billion.

Vertex’ shares were up 1.49% at $177.51 by close of trading on Tuesday following the announcement,