The U.S. FDA approved Orkambi^® (lumacaftor/ivacaftor) for children with cystic fibrosis ages 1-2 years who have two F508del mutations.

• Manufactured by Vertex Pharmaceuticals Incorporated, Orkambi was previously approved by the FDA for use in people with CF ages 2 years and older with two copies of the 508del mutation.

• This is the third expansion of Orkambi to younger age groups since Orkambi was first approved in 2015 for people with CF ages 12 years and older with two copies of the F508del The drug is one of four medicines approved to treat the faulty CTFR protein, the underlying cause of CF.