Dutch CF Registry 2017 - Treatment
In recent years, more and more new drugs have been introduced that combat the cause of CF. These drugs are called CFTR modulators, because they correct the wrongly-created CFTR protein, the chloride transport channel in the cells. These drugs reduce the symptoms throughout the body. Of the people who are eligible for treatment with Kalydeco, 94% used it in 2017. Of those people who are allowed to use Orkambi, the medicine was actually prescribed to 77%.
Figure 1 below shows how many people used CFTR modulators in 2017: Kalydeco, Orkambi or Symkevi. Symkevi is the latest CFTR modulator that was not yet available in the Netherlands at the time of writing (September 2018). The people who use the drug do so because they participated in a clinical study.