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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.

 

 


Actual benefit

Faible

-


Improvement in actual benefit

V (absence)

-


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