Cystic Fibrosis: The Year in Review

In another exciting announcement earlier this year, Vertex Pharmaceuticals also discussed the results they’d gathered from a Phase 3 clinical trial testing ivacaftor on children between the ages of 1 and 2. Ivacaftor is a drug that is designed to help individuals who have one of the ten underlying gene mutations that cause cystic fibrosis. The results indicated that the drug may help maintain partial functioning of the pancreas in children. The study was conducted over a 24-week period and was primarily testing whether or not the drug was safe for patient so young. Thankfully, the results were positive, which means that Vertex can move forward with their application to the FDA to hopefully get this drug on the market as another treatment option.