Friedreich's ataxia
Rare disease · 2025–2025
Every decision
| Medicine | Meeting | Outcome | Consumer input |
|---|---|---|---|
| omaveloxolone Treatment of Friedreich's ataxia in adults and adolescents aged 16 years and older. | Mar 2025 | Not recommended | 77 individuals Friedreich's Ataxia Research Alliance · Friedreich Ataxia Research Association |
| omaveloxolone Treatment of Friedreich's ataxia in people aged 16 years and older with a frataxin (FXN) gene mutation and mFARS score of 20 to 80. | Nov 2025 | Recommended with restriction | 123 individuals |
Who spoke
Friedreich's Ataxia Research Alliance · Friedreich Ataxia Research Association
Appraised elsewhere
NICE, in England and Wales, has appraised 1 technology for this condition, recommending 0 . A different committee, a different population and a different price: this is what has been looked at there, not a verdict on what happened here.
| Technology | Appraisal | Outcome | Date |
|---|---|---|---|
| omaveloxoloneOmaveloxolone for treating Friedreich's ataxia in people 16 years and over (terminated appraisal) | TA1061 | terminated | 2025-05 |
Conditions are classified by a language model from the indication text of each submission, so an individual label here can be wrong. Consumer input is read from the document's own consumer comments section: 2 of 2 decisions since 2014 carried some. Silence means no comment reached the committee through that facility, not that nobody was affected. More on how this was built.