KALYDECO Now the First FDA-Approved CFTR Modulator for Pediatric Patients with Cystic Fibrosis


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.


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. 

Cystic Fibrosis (CF)

Generally, cystic fibrosis is more present in Caucasian infants than those of other ethnicities. The condition is caused by genetic mutations. Rather than “healthy” mucus, which is normally slippery, patients with cystic fibrosis have thick, sticky mucus that accumulates in their airways. In addition to pooling mucus throughout their airways, cystic fibrosis causes blockages, traps bacteria, and inhibits breathing. Also, patients have difficulty absorbing nutrients. Complications of the condition include respiratory failure and severe lung damage.

Symptoms vary in severity, but include:

  • Frequent infections
  • Persistent coughing or wheezing
  • Chest and abdominal pain
  • Stuffy nose
  • Inability to gain weight
  • Salty skin
  • Fatigue
  • Failure to thrive
  • Shortness of breath
  • Exercise intolerance
  • Constipation
  • Infertility (males)

Learn more about cystic fibrosis here.

Jessica Lynn

Jessica Lynn

Jessica Lynn has an educational background in writing and marketing. She firmly believes in the power of writing in amplifying voices, and looks forward to doing so for the rare disease community.

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