following a full submission assessed under the orphan medicine process:
omaveloxolone (Skyclarys®) is not recommended for use within NHSScotland.
Indication under review: for the treatment of Friedreich's ataxia in adults and adolescents aged 16 years and older.
In a randomised, double-blind, phase II study, omaveloxolone improved the modified Friedreich’s ataxia rating scale (mFARS) score from baseline to week 48 compared with placebo; this was statistically significant and clinically relevant.
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 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:
- omaveloxolone (Skyclarys)
- SMC ID:
- SMC2845
- Indication:
For the treatment of Friedreich’s ataxia (FA) in adults and adolescents aged 16 years and older.
- Pharmaceutical company
- Biogen
- BNF chapter
- Central nervous system
- Submission type
- Full
- Status
- Not recommended
- Date advice published
- 09 March 2026