Ivacaftor (Kalydeco) for the treatment of cystic fibrosis in children who have a G551D mutation

Ivacaftor (trade name: Kalydeco) has been approved in Germany since July 2012 for the treatment of cystic fibrosis in children aged 6 years and over who have a minimum body weight of 25 kg and a G551D mutation. In 2019, the Institute for Quality and Efficiency in Health Care (IQWiG, Germany) looked into the advantages and disadvantages of ivacaftor compared to the standard treatment for cystic fibrosis in children. The standard treatment in this situation is best supportive care (BSC). It should be tailored to the child's individual needs, relieve symptoms and improve quality of life. BSC includes, for instance, inhalation treatments with saline (salt) solution, mucus-thinning medications, painkillers and physical therapy. For the purpose of the assessment, the manufacturer provided the results of one study involving 38 children between the ages of 6 and 11 years. One half of the participants were given one ivacaftor tablet (150 mg) every 12 hours, and the other half were given a placebo (fake medication). All of the children in the study also received BSC, but without all of the usual treatments because inhalations with saline (salt) solution were not allowed as an additional treatment. Also, the study didn’t show what sort of treatment adjustments were made in BSC. These were the results after about 11 months:

What are the advantages and disadvantages of ivacaftor?

Ivacaftor wasn't found to have any advantages or disadvantages compared to the placebo in children.

No difference

Life expectancy: None of the children passed away during the study.

Acute cystic fibrosis episodes affecting the airways: No difference was found here. 17 to 20 out of 100 children had acute cystic fibrosis episodes in each group.

There were also no differences in terms of:

  • Health-related quality of life
  • Hospital stays due to cystic fibrosis episodes affecting the airways
  • Difficulty breathing
  • Gastrointestinal (stomach and bowel) problems
  • Treatment stopped due to side effects

What remains unanswered?

The manufacturer didn’t provide any suitable data about severe side effects, rashes or dizziness.

Labels: E84, Ivacaftor, Kalydeco