Ivacaftor (Kalydeco) for the treatment of cystic fibrosis in teenagers and adults who have a G551D mutation
Ivacaftor (trade name: Kalydeco) has been approved in Germany since July 2012 for the treatment of cystic fibrosis in teenagers aged 12 years and over and adults who have a G551D mutation. In 2019, the Institute for Quality and Efficiency (IQWiG, Germany) looked into whether ivacaftor has any advantages or disadvantages compared to the standard treatment for cystic fibrosis in these groups of people. The standard treatment in this situation is best supportive care (BSC). It should be tailored to the patient'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 this assessment, the manufacturer provided one study involving 167 teenagers and adults. 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). Everyone in the study also received BSC, but without all 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 of ivacaftor?
Acute cystic fibrosis episodes affecting the airways: The study suggests that ivacaftor has an advantage here: The teenagers and adults who used ivacaftor had only half as many acute episodes as those who were given a placebo.
Difficulty breathing: The results suggest that ivacaftor has an advantage here too: Breathing improved in the ivacaftor group compared to the placebo group.
Physical wellbeing and vitality: Here the study suggests that ivacaftor has an advantage in these aspects of health-related quality of life in people whose bronchi were very narrow when the study began (FEV1 of less than 70 percent). There was no difference in people who had an FEV1 of over 70 percent.
Subjective impression of overall health: The study suggests that there was an improvement here in teenagers and adults who used ivacaftor compared to those who were given the placebo.
What are the disadvantages of ivacaftor?
Dizziness: The study suggests that ivacaftor has a disadvantage regarding this side effect. 12 out of 100 people who used ivacaftor experienced dizziness, compared to only 1 out of 100 people who were given the placebo.
Life expectancy: No participants passed away during the study.
Emotional state, body image, eating disorders, social limitations and stress resulting from the treatment: There was no difference in any of these aspects of health-related quality of life. There were also no differences concerning role functioning (the ability to carry out typical everyday activities).
There were also no differences in terms of:
- Hospital stays due to cystic fibrosis episodes affecting the airways
- Weight problems
- Gastrointestinal (stomach and bowel) problems
- Treatment stopped due to side effects
Rashes: There were no relevant differences here either.
What remains unanswered?
Severe side effects: The manufacturer didn't provide any suitable data about these problems.
Institute for Quality and Efficiency in Health Care (IQWiG, Germany). Ivacaftor (cystic fibrosis, 6 years and older, with G551D mutation) – Benefit assessment according to §35a Social Code Book V. Dossier assessment; Commission A19-65. November 28, 2019. (IQWiG reports; Volume 840).
IQWiG health information is written with the aim of helping
people understand the advantages and disadvantages of the main treatment options and health
Because IQWiG is a German institute, some of the information provided here is specific to the German health care system. The suitability of any of the described options in an individual case can be determined by talking to a doctor. informedhealth.org can provide support for talks with doctors and other medical professionals, but cannot replace them. We do not offer individual consultations.
Our information is based on the results of good-quality studies. It is written by a team of health care professionals, scientists and editors, and reviewed by external experts. You can find a detailed description of how our health information is produced and updated in our methods.