following a reassessment through the ultra-orphan framework:
ataluren (Translarna®) is not recommended for use within NHSScotland.
Indication under review: treatment of Duchenne muscular dystrophy resulting from a nonsense mutation in the dystrophin gene, in ambulatory patients aged 2 years and older. The presence of a nonsense mutation in the dystrophin gene should be determined by genetic testing.
In a phase III double-blind study in patients with Duchenne muscular dystrophy with a nonsense mutation, the average rate of decline in 6-minute walking distance was numerically lower in the ataluren group than the placebo group.
The submitting company’s justification of the treatment’s cost in relation to its health benefits was not sufficient and in addition the company did not present a sufficiently robust clinical and economic analysis to gain acceptance by SMC.
This advice takes account of the views from a Patient and Clinician Engagement (PACE) meeting.
Medicine details
- Medicine name:
- ataluren (Translarna)
- SMC ID:
- SMC2827
- Indication:
Treatment of Duchenne muscular dystrophy resulting from a nonsense mutation in the dystrophin gene, in ambulatory patients aged 2 years and older.
The presence of a nonsense mutation in the dystrophin gene should be determined by genetic testing.
- Pharmaceutical company
- PTC Therapeutics
- BNF chapter
- Musculoskeletal and joint diseases
- Submission type
- Ultra-orphan reassessment
- Status
- Not recommended
- Date advice published
- 08 June 2026