Children with cystic fibrosis have a chance to breathe easier
The new cystic fibrosis (CF) treatment named Symkevi (tezacaftor/ivacaftor), known as Symdeko in the US, is shown to safely and effectively loosen thick, sticky mucus in children 6 - 11 years old.
Previously approved for CF patients over 12, the manufacturer submitted an application for extended approval to the U.S. Food and Drug Administration late 2018, and is planning to do the same with the European Medicines Agency later this year.
The new approval extension would include children who have two copies of the most common mutation, F508del, in the CFTR gene, or one copy of the F508del and one residual function mutation. More details can be found in the full article.