The Food and Drug Administration has approved Kalydeco (ivacaftor) for the treatment of patients aged 12 to less than 24 months who have cystic fibrosis that is caused by any of 10 mutations in the CFTR gene and is responsive to the drug, the drug’s developer announced.
The drug was approved for patients aged 6 years and older in 2012 and in patients aged 2-5 years in 2015 and is the only approved drug that treats the underlying cause of cystic fibrosis rather than its symptoms.
The approval is based on the ongoing phase 3, open-label ARRIVAL trial (NCT02725567), which is assessing the drug’s safety in children aged 12 months to less than 24 months. The trial’s investigators have found that its safety profile in this age group is consistent with that seen in older children and adults. Most adverse events were mild to moderate; the most common (occurring in more than 30% of patients) were cough, pyrexia, elevated aspartate aminotransferase, elevated alanine aminotransferase, and runny nose. The trial found that, after 24 weeks of treatment, the mean sweat chloride levels decreased from 104.1 mmol/L (n = 14) to 33.8 mmol/L (n = 14).
Ivacaftor is contraindicated in patients taking certain antibiotics, seizure medications, or other medications; risk of drug interaction – affecting either the performance of ivacaftor or that of the other medication – is also a concern. Patients should inform their doctors if they are pregnant, planning to become pregnant, or breastfeeding; have liver or kidney problems; or drink grapefruit juice or eat grapefruit or Seville oranges. There also is a risk of high liver enzymes or cataracts. Ivacaftor is available in 150-mg tablets for adults and pediatric patients aged 6 years and older and in 50-mg and 75-mg granules for younger patients. Full prescribing information can be found on the FDA website.