Drugs for delF508

Question

Hello,
I have recently visited the annual meeting in Bad Soden, Germany. I have heard there, that Kalydeco® together with a potentiator is said to be licensed already in June 2014. Short-dated also a liquid for children is said to come on the market. Did I get this right? Does this mean the breakthrough?
How exactly does this drug function? Is it like this that the chloride household of the body is regulated with it and the organs do not "obstruct" any more? Many thanks for your answer.

Answer

Dear questioner,
in the above mentioned meeting it has been said, that at the moment the studies concerning delF508 are in the phase of evaluation. Hereby it deals with a combination of a potentiator (Kalydeco®) and a corrector. Depending on the study results, market authorization will be requested. First of all, however, the study results have to be waited for, that we expect for June 2014. A market authorization can happen the earliest at the end of 2014 or the beginning of 2015.
The by you mentioned liquid are micropellets for children aged 2-5 years and concern the drug Kalydeco®, that has at the moment exclusively market authorization for patients with at least one mutation G551D and is licensed only for this patients who are 6 years of age or older. An authorization for those micropellets is expected for the beginning of 2015.
The action of Kalydeco® has been discussed in detail in the workshop. In summary: Kalydeco® influences positively the probability of opening of the chloride channel and this results in an improved transport of chloride ions. That leads to better features of the airway- and gut secretions.
Best regards,
Olaf Eickmeier

21.07.2014