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Ataluren and research
- Question
- Hello,
Some time ago I asked a question concerning Ataluren on this platform. Our two daughters (28 and 31) have the genotype Delta F508 and R1162X. Our CF physician mentioned at the last control, that recently quite promising results have been reported for Ataluren at a CF congress in London.
According to Prof. Tümmler, who I have contacted already some time ago, R1162X would be suitable for therapy with Ataluren, in case it would be licensed on the market.
Can you report about the latest developments concerning Ataluren?
Many thanks!
- Answer
- Dear questioner,
You can find the developments of the last years concerning Ataluren in the web under:
1. Informations on the results of the Ataluren (PTC) phase 3 study - [site in German]:
https://muko.info/forschung/news-detail-und-archiv/detailansicht/article/ataluren-erste-ergebnisse-der-phase-3-studie.html
(I think you will already know the opinion of Mrs Bend from the answer to one of your last questions)
2. After this, there are only few new things to report – however two other studies with this drug have been completed and are at the moment analysed.
(https://muko.info/forschung/klinische-studien/studienliste.html, dort: PTC124-021: Ataluren vs. Plazebo und PTC124-023: Ataluren Langzeitwirkung).
3. The company itself writes about Ataluren and CF word by word:
(www.ptcbio.com/en/pipeline/ataluren-translarna/ataluren-faq/): “An application for approval of ataluren for nonsense mutation cystic fibrosis (nmCF) has not been submitted and therefore, ataluren for nmCF is only available through clinical trials." That means that the company has actually not provided an application for approval of ataluren in CF. The reason for this are some data from the phase 3 study (1.) It remains open, if PTC is now applying for approval after finishing and analyzing the following studies (2.).
In summary: actually there are unfortunately no new news on the topic Ataluren, I hope nevertheless to have helped you a bit with this information
Best regards,
Dr. Frauke Stanke
- 05.12.2016
