The U.S. Food and Drug Administration (FDA) approved the use of lumacaftor/ivacaftor (Orkambi®) today for children with cystic fibrosis ages 6 to 11, who have two copies of the most common CF gene mutation, F508del. The decision means that about 2,400 additional children in the U.S. are eligible to receive the drug, bringing the total number of those eligible for the treatment in the U.S. to nearly 11,000.
The FDA approved lumacaftor/ivacaftor last year for people with CF ages 12 and older who have two copies of the F508del mutation. The drug is the second to treat the faulty CFTR protein in CF. The first drug, ivacaftor (Kalydeco®), was approved in 2012 for people with specific rare mutations of CF.
In people with two copies of F508del, lumacaftor/ivacaftor improves lung function and significantly reduces the rate of pulmonary exacerbations, which can lead to frequent hospitalizations and accelerated lung disease.
Lumacaftor/ivacaftor was developed by Vertex Pharmaceuticals Inc. with significant clinical, scientific and financial support from the Cystic Fibrosis Foundation.
Vertex is looking to expand access to the drug further by conducting a Phase 3 clinical trial of lumacaftor/ivacaftor in children ages 2 to 5.
For more information, see the Vertex press release.