In Europe, about 12,000 people with CF in this age group have two copies of F508del, the most common CF mutation.
Orkambi® targets the underlying cause of the disease -- a defective
In July, the U.S. Food and Drug Administration approved Orkambi® for individuals with two copies of the F508del mutation ages 12 and older, representing about 8,500 people or nearly one-third of those with CF in the United States.
The drug is the second to treat the faulty
Orkambi® is a combination of ivacaftor and lumacaftor. In people with two copies of F508del, the drug improves
Orkambi® was developed by Vertex Pharmaceuticals Inc. with significant clinical, scientific and funding support from the CF Foundation.