Ataluren (Translarna®)

Assessment Status NCPE Assessment Process Complete
Drug Ataluren
Brand Translarna®
Indication For the treatment of DMD resulting from a nonsense mutation in the dystrophin gene, in ambulatory patients aged 5 years and older. The presence of a nonsense mutation in the dystrophin gene should be determined by genetic testing.
Assessment Process
Rapid review commissioned 18/11/2014
Rapid review completed 23/12/2014
Rapid review outcome Full Pharmacoeconomic Evaluation Recommended
Full submission received from Applicant 29/10/2015
NCPE assessment completed 29/04/2016
NCPE assessment outcome Reimbursement not Recommended


The HSE has approved reimbursement following confidential price negotiations, May 2019.