After thorough reconsideration, the European Medicines Agency’s human medicines committee (CHMP) has upheld its initial decision to decline the conditional marketing authorization renewal for Translarna (ataluren). This medication is prescribed for individuals with Duchenne muscular dystrophy caused by a specific genetic anomaly known as a ‘nonsense mutation’ in the dystrophin gene, who retain the ability to walk.
The original verdict stemmed from a comprehensive re-evaluation of Translarna’s pros and cons during the renewal process, concluding that its efficacy remained unverified. In response to a request from the drug’s marketing company, the CHMP conducted a re-examination, scrutinizing data from a post-authorization study (study 041) and findings from a comparative analysis of two patient databases.
The CHMP determined that the outcomes from study 041 did not demonstrate the medicine’s effectiveness in patients experiencing a gradual decline in walking ability, a group anticipated to derive substantial benefits from Translarna compared to others in the study. Over 18 months of treatment, these patients experienced an average decrease of about 82 meters in their six-minute walking distance in the Translarna group versus 90 meters in the placebo group. However, this variance lacked statistical significance, suggesting it could be attributed to chance.
Furthermore, the committee highlighted that additional studies failed to confirm Translarna’s mechanism of action, revealing only a minimal impact on dystrophin protein production.
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