NCHR Testimony at the FDA Panel on Ataluren

September 28, 2017. We agree with FDA scientists that the data today do not indicate significant benefit in randomized, double-blind, placebo-controlled trials, such as Study 7. Only after making many post-hoc changes did ataluren show it was effective for ADP patients, but this was not replicated in Study 20. As you know, those post-hoc manipulations do not provide clear evidence of efficacy. For both studies, 90% and 79% of patients were White. But the CDC reports that Hispanic males are disproportionately likely to have these conditions. It is essential that an adequate number of Hispanic males be analyzed to determine if they can benefit from a treatment such as ataluren.

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