On September 25, biotechnology company Vertex Pharmaceuticals Incorporated (“Vertex”) announced FDA-approval of KALYDECO (ivacaftor) for pediatric patients with cystic fibrosis (CF). Specifically, KALYDECO can treat infants between 4-6 months old with a CFTR gene mutation. While the therapy is already approved for treating patients older than 6 months old, this approval represents the therapy’s ability to meet an unmet patient need. Additionally, KALYDECO is the first (and only) FDA-approved CFTR modulator.
KALYDECO
The FDA approved KALYDECO using data from the Phase 3 ARRIVAL trial. Overall, the trial enrolled 6 pediatric patients with cystic fibrosis. There are multiple CFTR genetic mutations resulting in cystic fibrosis. For the purpose of this trial, all participants were required to have one of 10 specific mutations. Ultimately, KALYDECO was shown to reduce symptoms, improve patient outcomes, and modify disease progression.
However, there are a few factors to consider in regards to KALYDECO. It should not be given to patients under 4 months old. Patients taking rifampin, rifabutin, phenytoin, phenobarbital, or St. John’s wort should not take KALYDECO. Currently, there is no data on how KALYDECO might affect pregnant women or fetuses. Side effects include dizziness, high liver enzymes, jaundice, cataracts, headache, respiratory tract infections, nausea, diarrhea, or abdominal discomfort.
