VX-809 research

Question

Hello,
in the research news dated 01.01.2013
(here the link to a German website:
http://muko.info/forschung/news-detail-und-archiv/detailansicht/article/zulassungsprogramm-fuer-korrektur-des-f508del-soll-2013-starten/17.html ).
it has been said, that the market authorization for "VX-809" should start even in the year 2013, unfortunately, until today no furhter news appeared on this. My question is, how advanced is the study at the moment and when can one expect further information?
Best regards,
H.

Answer

Dear questioner,
in case of the mentioned "authorization", it deals with a "research authorization", when jargonizing it can be called phase-3 study. Hereby it is investigated (before market authorization of the substance as a drug) with patients finally if the drug is effective and if there are no (severe) side effects. Officially, according to clinicaltrials.gov (see adress below), data is collected until march 2016, threfore unfortunately until the final judegment of the results and with that until a successfull market authorization, some time will still go by.

The by you mentioned VX-809 study is indeed a study with 2 drugs: VX-770 and VX-809. Hereby at the moment, research is going on for the final dosage of both preparations. The study for market authorization for the combination preparation is composed of 3 parts, out of those 2 are already finished.
Please find more under:
www.clinicaltrials.gov/ct2/show/record/NCT01931839?term=vx-770+vx-809&rank=10

(aktiv)
and
www.clinicaltrials.gov/ct2/show/record?term=vx-770+vx-809&rank=7

www.clinicaltrials.gov/ct2/show/record?term=vx-770+vx-809&rank=6

(fertig)

It remains to await, if Vertex will present in advance data from this study at the north American CF congress in autumn 2014 (a success, e.g. in the finding the right dosage, will certainly be judged positively by the heads of the study).
Best regards,
Frauke Stanke

09.07.2014