This medicine is used for treating patients with Duchenne muscular dystrophy whose disease is caused by a type of genetic defect called a ‘nonsense mutation’ in the dystrophin gene and who are able to walk.
The initial recommendation followed the full re-evaluation of Translarna’s benefits and risks during the renewal of its marketing authorisation, which concluded that its effectiveness had not been confirmed.
As part of the re-examination requested by the company that markets Translarna, the CHMP re-assessed the data from a study carried out after authorisation as a specific obligation (study 041) and the results from a study comparing two patient registries.
The CHMP concluded that the results from study 041 failed to show that the medicine was effective in patients with a progressive decline in their ability to walk, who were expected to have greater benefit from Translarna treatment compared with other patients included in the study…
