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Prescription of Orkambi® not possible in Ireland

Question
Hallo,

my boss in Ireland is a father of a 15-year-old girl with CF. Unfortunately Orkambi® is not licensed in Ireland. Is there a possiblity to get it in Germany for her? What does he has to do to get it? Is private insurance enough or do you have to live in Germany, paying a german health insurance? How much are the costs of treatment per year for it?

Thank you,
N.
Answer
Dear N.,

thank you for your request.
Orkambi® is licensed for CF patients (> 12years) homozygous for the deltaF508 mutation in the CF gene (=cystic fibrosis transmembrane regulator protein (CFTR)). Do you know what the girl's mutation is? If the girl is homozygous for deltaF508, the following is important: Some things have to be checked (ophthalmologic examination, liver and lung function test etc.) before doctors can prescribe Orkambi®. In this regard they should consult a physician/CF center with an expertise in prescribing it. If the girl wants to get treated, check in advance with the Irish, if they would pay for the cost generated in Germany. The costs of treatment are very high resp. more than € 300.000.- per year.

Hopefully this information will help you.

Kind regards,

Sabina Schmitt-Grohé (MD)

17.03.2017