TRANSLARNA (ataluren), RNA interference agent
NEUROLOGY - Update
Opinions on drugs - Posted on Oct 04 2017
Reason for request
Re-assessment of the actual benefit and the improvement in actual benefit
No clinical benefit demonstrated in the treatment of Duchenne muscular dystrophy, resulting from a nonsense mutation in the dystrophin gene, in children ≥ 5 years
- TRANSLARNA has marketing authorisation in the treatment of Duchenne muscular dystrophy, resulting from a nonsense mutation in the dystrophin gene, in ambulatory patients aged ≥ 5 years.
- In the absence of demonstration of its efficacy compared with the placebo on the decline in walk distance, but considering the severity of the disease and the absence of any therapeutic alternative with a valid marketing authorisation and given its acceptable safety, TRANSLARNA may be added to the current therapeutic regimen without modifying it.
Improvement in actual benefit
Évaluation des médicaments