Kalydeco® for children

Question

Dear expert team,
is there any concrete information about the time of licensing of Kalydeco® for children from 2-5 years?
How successful were the studies?
My daughter has G551D, can the health insurance in spite of this refuse to pay the costs?
Are there any reasons that speak against Kalydeco® in spite of a suitable mutation?
Many thanks in adavance

Answer

Dear questioner,
Kalydeco® is at the moment licensed in Germany for children from the age of 6 years on with one of the following gating mutations (class III) in the CFTR gene: G551D, G1244E, G1349D, G178R,G551S, S1251N, S1255P, S549N or S549R. Therefore your daughter would fall into the area of indication when she is 6 years old.
On the question about the coverage from the health insurance, I would like to cite a paragraph from an informational page of the German CF patient organization "Mukoviszidose eV":
"The public health insurances do at the moment cover the costs for Ivacaftor. A new law governs the introduction of new drugs in Germany like this, that in the first year after licensing, the public health insurances cover the costs. In the first year, there is also a benefit assessment of the new drug done. On this basis there is finally a conference between the health insurance and the pharmaceutical company about the price after the first year. The German patient organization represents the interests of the patients in this process. Accordingly we will further report, if something concerning this topic has changed. "
The complete, interesting article about Kalydeco® can be found on the website of the Muko.eV (German Website):
muko.info/forschung/news-detail-und-archiv/detailansicht/browse/1/article/ivacaftor-kalydecoR-vx-770-in-deutschland-zugelassen/17.html


Reasons, that speak against Kalydeco® in spite of a suitable mutation, can be found in detail on the leaflet of the drug; caution is in order in case of a strongly reduced kidney function; in case of moderate liver impairment, the dosage has to be adapted, in case of a strongly impaired liver function, the drug should only be taken after a detailed risk-effect analysis. Furthermore there is only few data for patients, whose lung function is under 40%. Furhtermore, drug interactions have of course to be taken into account.
Furthermore it should be considered, that Klaydeco® does not show the same effect in all patients, in spite of an identical mutation. Therefore it is for sure going to play a role when considering the question, if the therapy is continued and also if the costs are paid by the insurance, if in the course of time a positive effect on the lung function of the patient can be shown.
On the study data it can be said, that there are 2 published phase III studies; one tested the efficacy and tolerability of the drug in CF patients older than 12 years, the other in patients at the age of 6-11 years. Due to the positive results, Kalydeco® has been licensened actually for these age groups. At the moment a study is ongoing, thats data has already been collected and that is evaluated right now. Here patients over 6 years of age can participate, in order to test the long term tolerability of Kalydeco®.
In the register of all actually running studies (link: https://clinicaltrials.gov/ct2/results?term=VX+770&pg=1), data of a study is evaluated at the moment, that is aiming to test the security of Kalydeco® in patients with CF at the age of 2-5 years. This is a phase III study, that is done in the USA, Canada and UK, with patients participating who have one CFTR gating mutation (therefore also others than G551D) and are 2-5 years old. This study is intended to investigate the security and metabolism of the drug and also to find the right dosage for this age group. In March 2014 the data has been collected and is evaluated at the moment, so that in a short time frame one can expect some information. How long it is going to last then until there will be a licensing for this age group in Germany, I am not able to tell you, but things are on the way.

I hope to have helped you with my remarks,
Best regards,
Dr. Daniela d'Alquen

03.01.2015