EMA Reaffirms Translarna Ruling

The European Medicines Agency’s human medicines committee (CHMP) has upheld its decision not to renew the conditional marketing authorization for Translarna (ataluren), a treatment for certain patients with Duchenne muscular dystrophy (DMD) caused by a “nonsense mutation” in the dystrophin gene. Despite multiple re-evaluations requested by the manufacturer, including reviews of additional real-world data and new analyses, the CHMP concluded that Translarna’s effectiveness remains unproven. The initial negative opinion was issued in September 2023, reaffirmed in January 2024, and again in June 2024 after further review by the EMA.¹

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References

1. EMA recommends non-renewal of authorisation of Duchenne muscular dystrophy medicine Translarna | European Medicines Agency (EMA). (2023, September 15). European Medicines Agency (EMA). https://www.ema.europa.eu/en/news/ema-recommends-non-renewal-authorisation-duchenne-muscular-dystrophy-medicine-translarna

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