Ivacaftor (Kalydeco) with tezacaftor / ivacaftor (Symkevi) for the treatment of cystic fibrosis in people aged 12 years and over who have a homozygous F508del mutation
Ivacaftor (trade name: Kalydeco) with the fixed-dose combination of ivacaftor / tezacaftor (trade name: Symkevi) has been approved in Germany since October 2018 for the treatment of cystic fibrosis in teenagers aged 12 years and over and adults who have a homozygous F508del mutation. In 2019, the Institute for Quality and Efficiency (IQWiG, Germany) looked into whether ivacaftor in combination with tezacaftor / ivacaftor has any advantages or disadvantages in the treatment of cystic fibrosis in these people compared to the standard treatment of lumacaftor / ivacaftor.
The manufacturer provided data from an indirect comparison. The results of this kind of comparison are generally less reliable than those of a direct comparison. In this case, one study compared ivacaftor plus the fixed-dose combination of tezacaftor / ivacaftor with a placebo, and two other studies compared lumacaftor / ivacaftor at various doses with a placebo as well. Everyone in the studies had treatment for their cystic fibrosis symptoms too. Only people with a homozygous F508del mutation participated. These were the results after about half a year:
What are the advantages of ivacaftor?
Rashes: The indirect comparison suggests that ivacaftor has an advantage here.
What are the disadvantages of ivacaftor?
Hospital stays due to cystic fibrosis episodes affecting the airways: The indirect comparison suggests that ivacaftor has a disadvantage here.
Life expectancy: None of the participants passed away during the study.
There were also no differences between the two groups in terms of the following:
- Health-related quality of life
- Acute cystic fibrosis episodes affecting the airways
- Weight problems
- 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.
Institute for Quality and Efficiency in Health Care (IQWiG, Germany). Ivacaftor (combination with ivacaftor/tezacaftor, cystic fibrosis, 12 years and older, with F508del mutation, homozygous) – Benefit assessment according to §35a Social Code Book V. Dossier assessment; Commission A19-70. November 28, 2019. (IQWiG reports; Volume 844).
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