On 28 March 2025, the European Commission issued a decision to not renew the conditional marketing authorisation for Translarna (ataluren) in the European Union (EU). The marketing authorisation holder for the medicine was PTC Therapeutics International Limited. The non\-renewal of the marketing authorisation follows the opinion issued by EMA’s human medicines committee, CHMP, which recommended to not renew the authorisation because the effectiveness of the medicine has not been confirmed. Further information is available in the [news announcement](https://www.ema.europa.eu/en/news/translarna-ema-re-confirms-non-renewal-authorisation-duchenne-muscular-dystrophy-medicine) published at the time of the CHMP opinion. Translarna was granted marketing authorisation in the EU on 31 July 2014 for the treatment of Duchenne muscular dystrophy. The European Public Assessment Report (EPAR) for Translarna will be updated to indicate that the marketing authorisation is no longer valid.
Therapeutic Indication
Translarna is indicated for the treatment of Duchenne muscular dystrophy resulting from a nonsense mutation in the dystrophin gene, in ambulatory patients aged 2 years and older. Efficacy has not been demonstrated in non\-ambulatory patients. The presence of a nonsense mutation in the dystrophin gene should be determined by genetic testing.
Therapeutic Area (MeSH)
ATC Code
M09AX03
ATC Item
N/A
Pharmacotherapeutic Group
Other drugs for disorders of the musculo-skeletal system
Active Substance (Summary)
INN / Common Names
EMA Name
Translarna
Medicine Name
Translarna
Aliases
N/ANo risk management plan link.
