I know it’s been a while since I’ve shared some new information that has been in the Drug Pipeline for CF. This first one isn’t for Little Miss Khaos (her mutation is DDF508) but it is great news for those that use Kalydeco! You can learn more about all of the drugs going through studies here.
FDA Approves Ivacaftor for Five Splice Mutations
The U.S. Food and Drug Administration (FDA) approved the use of ivacaftor (Kalydeco®) today for people ages 2 and older with cystic fibrosis who have at least one of the following five splice mutations: 3849+10kbC->T, 2789+5G->A, 3272-26A->G, 711+3A->G, E831X.
This second one is for her mutation!
Positive Early Study Results for Next-Generation CFTR Modulators
New data show positive results in individuals with a single F508del mutation as well as people with two copies of F508del and support continued development of triple combination therapies.