EMA confirms recommendation for non-renewal of ...
EMA's CHMP has recommended not renewing Translarna's EU marketing authorisation, citing unconfirmed effectiveness for Duchenne muscular dystrophy patients with a nonsense mutation. Despite re-examination, including study 041 and patient registry data, Translarna's benefits were not statistically significant, leading to a negative benefit-risk balance conclusion.
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EMA confirms recommendation for non-renewal of ...
EMA's CHMP has recommended not renewing Translarna's EU marketing authorisation, citing unconfirmed effectiveness for Duchenne muscular dystrophy patients with a nonsense mutation. Despite re-examination, including study 041 and patient registry data, Translarna's benefits were not statistically significant, leading to a negative benefit-risk balance conclusion.