Today, Vertex Pharmaceuticals Inc. released the initial Phase 3 clinical trial data for one of two next-generation, triple-combination modulators currently being tested.
The U.S. Food and Drug Administration approved the use of lumacaftor/ivacaftor (Orkambi®) today for children with cystic fibrosis ages 2 to 5 who have two copies of the F508del mutation.
Celtaxsys Inc. reported promising results today from an early stage clinical trial of a potential anti-inflammatory drug for people with cystic fibrosis.
A new drug intended to make it easier to clear mucus from the lungs of people with cystic fibrosis showed promising results in a Phase 2 clinical trial. The drug, called SPX-101, blocks the overactive sodium channels that line CF lung cells.
Yesterday, President Donald J. Trump signed the Right to Try bill into law. The House approved the bill last week and the Senate passed it last summer.