The U.S. Food and Drug Administration (FDA) approved the use of lumacaftor/ivacaftor (Orkambi®) today for children with cystic fibrosis ages 2 to 5 who have two copies of the most common CF gene mutation, F508del. The decision means that about 1,300 additional children in the U.S. are eligible to receive the drug, bringing the total number of those in the U.S. who could benefit from the treatment to approximately 12,300.
“This is great news for our community, as research indicates that starting treatment with CFTR modulators early may limit the onset of complications from the disease,” said Preston W. Campbell, III, M.D., president and CEO of the Cystic Fibrosis Foundation. “We are committed to building on this momentum to help bring more and better treatments to the people with CF who need them.”
A Phase 3 study evaluating the drug for children ages 12 months to less than 24 months is planned to start later this year.
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 lung damage.
The FDA first approved lumacaftor/ivacaftor in 2015 for people with CF ages 12 and older who have two copies of the F508del mutation. In 2016, the FDA expanded that approval to people with CF ages 6 to 11 with those same mutations. The drug is one of three medicines approved to treat the faulty cystic fibrosis transmembrane conductance regulator (CFTR) protein in CF.
Lumacaftor/ivacaftor was developed by Vertex Pharmaceuticals Inc. with significant clinical, scientific, and financial support from the Cystic Fibrosis Foundation.
The Foundation also supports research aimed at discovering new treatments for people with nonsense and rare mutations that do not produce the CFTR protein, an estimated 5 to 7 percent of people with CF. These individuals will be unable to benefit solely from modulators. In 2016, the Foundation launched the Nonsense and Rare Mutations Research and Therapeutics Initiative to help advance research by academic institutions and pharmaceutical companies focused exclusively on the creation of therapies to “restore” CFTR production for these individuals.