Translarna™ (ataluren) Market Authorisation Not Renewed by European Commission.
The European Commission (EC) has announced that it will not renew the market authorisation of Translarna™ (ataluren) for treating nonsense mutation Duchenne muscular dystrophy. After re-examining the data, the EMA’s human medicines committee (CHMP) concluded that the effectiveness of Translarna could not be confirmed.
EMA Recommend Against Renewing Translarna for DMD
The Committee for Medicinal Products for Human Use (CHMP), part of the European Medicines Agency, has again advised against renewing approval for Translarna (ataluren), a drug used to treat Duchenne muscular dystrophy (DMD) caused by a nonsense mutation in the dystrophin gene.
7 September is World Duchenne Awareness Day
On 7 September the World Duchenne Organisation will launch a documentary that portrays the lives of people living with Duchenne muscular dystrophy across the globe, it will share their journeys, challenges and successes, and captures the resilience and determination of the Duchenne community. People are encouraged to share it with their wider communities.
CHMP Issues Negative Opinion on Translarna™ Following European Commission Request for Review
EMA’s human medicines committee (CHMP) has recommended non-renewal of the conditional marketing authorisation of Translarna™ (ataluren) for the treatment of nonsense mutation Duchenne muscular dystrophy in Europe. This opinion follows the return of the previously issued negative opinion by the European Commission for re-review. Click here to read PTC Therapetics press release.
European Commission Returns Translarna™ Opinion to CHMP For Re-evaluation
We are pleased to announce that the European Commission (EC) has decided not to adopt the Committee for Medicinal Products for Human Use's (CHMP) negative opinion on the renewal of the conditional marketing authorization of Translarna™ (ataluren).
EMA confirms recommendation not to renew marketing authorisation for Translarna
The CHMP agreed to re-examine its decision on request of the manufacturer PTC. This took place in January and despite efforts from the manufacturer and patient community the EMA announced on Friday, 26 January 2024 recommendation for non-renewal of authorisation of Translarna. The process found that while there was evidence of benefit from the treatment, it’s not statistically significant. Importantly there was no concerns about the safety of the treatment.
Round Up On Research Developments By Condition
Neuromuscular conditions can be difficult to diagnose and manage, so research is vital for families and for quality of life. In this issue, we include a round-up of some research developments that have taken place in recent months
If you or your son is currently taking Translarna, and you would like to express your views on the recent EMA announcement please contact MDI
PTC therapeutics have asked for a re-examination of the data with a final decision expected in January 2024. Muscular Dystrophy Ireland is working collaboratively with clinicians in Ireland and other muscular dystrophy patient organisations from Europe to support people during this time.