Kalydeco® also before 6th birthday?

Question

My son, 4, CF, has the mutations G551D and dF508.
Until now he is doing quite well concerning his general condition, even if he already had to fight strongly in his first year of life.
Now I am asking myself if Kalydeco® can be prescribed for him even before his 6th birthday.
Finally it is especially in CF so important, to preserve the lung from greater damage. Therefore the sooner the better.

Answer

Hello,
I think the usage of Kalydeco® is especially reasonable before the onset of longterm damage of the lung. Therefore, I am fully of your opinion, that CF patients with at least one mutation G551D, as your son, especially profit if the drug is used early. Before each market authorization of a new drug however, clinical studies of the corresponding age group are demanded. Here, finding of dosage, profile of side effects and the effectiveness of the substance is checked. This is especially difficult in the age group of your son. The actual dosage form of the drug is one tablet and therefore in the age group under 6 years often problematic in the application. In spite of this, there is a study for children between 2 and 6 years of age with CF and at least one mutation G551D in the recruiting phase. Here micro-tablets have been developed, that can be taken with milk or apple purree. However, this study is according to my knowledge not conducted in Germany.

Yours sincerely,
Olaf Eickmeier

10.05.2013