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Kalydeco off label
- Question
- Hello,
my daughter has the mutations delF508 and D1152H. How great is the chance to get Kalydeco off-label? There are obviously in-vitro studies that show, that the CFTR channel is functioning nearly completely in case of intake? There is already a study from Denver about this? In the USA human beings with these mutations profit already from Kalydeco.
Many thanks for your efforts. - Answer
- Hello,
it is correct, that patients with the mutation D115H are already treated with Ivacaftor. These patients receive the medication in the frame of an on-going study, that has alreay been performed and is named "N-of-One". In case of These "N-of-One studies", patients with rare mutations have been included, as one has to judge the individual benefit in case of respective low patient numbers. In the frame of such a study, patients older than 12 years with the mutation D115H have been included.
Please ask your center about a probable "compassionate use" program. It would be helpful for the arguing to have a short description of your daughter (age, FEV1, status of germs, etc.).
Best regards,
Olaf Eickmeier - 18.06.2015
