Today, the Commission granted EU conditional marketing authorisation for givinostat, a medicine to treat Duchenne muscular dystrophy. There is currently no cure for this rare, genetic disease, characterised by progressive muscle weakness and breakdown, primarily in children.
The authorisation is based on the positive scientific assessment from the European Medicines Agency (EMA). The EMA concluded that while not all data is yet available, the benefits to patients of the immediate availability of this medicine outweigh the risks. In order to confirm the efficacy of givinostat, the company has been requested to conduct two further studies, based on patient data.
The conditional marketing authorisation is granted pending this additional data and is valid for one year, after which it may be renewed on a yearly basis. The Commission decision is online.
More information is available online.
(For more information: Eva Hrnčířová – Tel.: +32 2 298 84 33; Anna Gray – Tel.: +32 2 298 08 73)
- 6 June 2025
Excerpt from the official page. The original is authoritative.