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Ataluren
- Question
- When will Ataluren be available in Germany?
- Answer
- Hello,
Dr. Jutta Bend of Mukoviszidose e.V. (the German CF patient association) provided the following answer to your question:
“Unfortunately, the news on this topic is not all that great.
At the ECFS congress in Dublin, results of the phase III Ataluren (=PTC124) study were presented. No significant improvement in lung function (FEV1) was found throughout the study population, though apparently there is a positive tendency. A significant effect could only be shown through a sub-group analysis (i.e., patients were grouped by antibiotics therapy and then analyzed): It could be proven that Ataluren was effective in those patients who had not been given inhalative aminoglycosides (e.g., Gentamicin and Tobramycin are aminoglycoside antiobiotics). But again, this effect could only be shown in a sub-group analysis and not in the entire group of study patients.
Therefore, unfortunately, the overall result of this phase-III study is not as clear as would have been wished for, and other studies would have to prove the drug’s effectiveness better. After all, given these current results, one can not be certain that the drug is actually effective.
Given these disappointing results, it is not possible to tell when (and if) the drug will ever hit the market.”
Kind regards
Annette Pfalz for ECORN-CF - 02.08.2012
