TRANSLARNA (ataluren), RNA interference agent
RARE DISEASE - NEW MEDICINAL PRODUCT
Opinions on drugs - Posted on Feb 23 2015
Reason for request
Minor improvement in the management of Duchenne muscular dystrophy
- TRANSLARNA has Marketing Authorisation in the treatment of Duchenne muscular dystrophy (DMD) resulting from a nonsense mutation in the dystrophin gene, in ambulatory patients aged 5 years and older.
- Available data suggest a potential benefit, poorly demonstrated and modest, in terms of slowing the loss of ambulation in some children with Duchenne muscular dystrophy, with acceptable safety.
- Efficacy has not been demonstrated in patients who have lost ambulation.
Improvement in actual benefit
Évaluation des médicaments