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Genetic therapy heterozygous DF508

Question
Hello,
how far is research concerning DF508-heterozygous patients? Are there first study results, when can one expect a licensing in case of promising results?
Thanks!
Answer
Dear questioner,

at the moment, two drug combinations are tested for patients with the CF mutation F508del-CFTR: the combination therapy Lumacaftor/Ivacaftor as well as the combination therapy VX-661/Ivacaftor (please see: muko.info/forschung/klinische-studien/studienliste.html [German website]).
With this, the studies about Lumacaftor/Ivacaftor are advanced to phase III, while for the drugs VX-661/Ivacaftor patients are recruited for a so-called phase II study.
The company Vertex, that produces such drugs, planns according to the available information in the internet a licensing procedure for the "...4th quartile 2014 in Europe as well as in the USA. In case this request would be supported, Lumacaftor/Ivacaftor could probably already soon be available, namely in the course of the year 2015 or at the beginning of 2016. This would be true for patients with two F508del mutations over the age of 12 on the German market. For the treatment of patients with only one F508del mutation or other mutations, further (mutation specific) therapies have to be developed. And also the correction in case of two F508del mutation will have to be further improved...." (here it deals with a citation of the information of the German patient organization Mukoviszidose eV, please find the full text under muko.info/forschung/news-detail-und-archiv/detailansicht/article/ergebnisse-der-vertex-studien-mit-der-kombination-von-lumacaftor-und-ivacaftor-bei-f508del-homozyg/17.html)
For the combination therapy with VX-661/Ivacaftor the research is not advanced enough for licensing.
Best regards,
Frauke Stanke
12.06.2015