Recent omaveloxolone updates include progress in the phase 3 BRAVE study, development of a liquid-ready formulation and evolving reimbursement discussions across multiple regions
Phase 3 BRAVE study advances pediatric development in Friedreich’s ataxia
Biogen is continuing to expand the clinical development and global availability of omaveloxolone (SKYCLARYS®; Biogen, Cambridge, MA, USA) for Friedreich’s ataxia (FA), with recent activity focused on pediatric evaluation, treatment accessibility and long-term evidence generation.1–4
Omaveloxolone became part of Biogen’s rare disease portfolio following the company’s acquisition of Reata Pharmaceuticals in September 2023. Biogen has since continued development of the therapy, which is approved for adults and adolescents aged 16 years and older with FA in several regions, including the USA and European Union.1,2
A key ongoing initiative is the global phase 3 BRAVE study, which is evaluating the efficacy, safety, pharmacokinetics and pharmacodynamics of omaveloxolone in approximately 255 children aged 2 to <16 years with FA.2,3 The 52-week, randomized, double-blind, placebo-controlled study uses change from Baseline in Upright Stability Score, a subscale of the modified Friedreich Ataxia Rating Scale, as its primary endpoint. Participants are randomized 2:1 to receive once-daily omaveloxolone or placebo, followed by an open-label extension lasting up to 104 weeks.2,3
New liquid-ready omaveloxolone formulation aims to improve treatment accessibility
In an April 2026 community update shared by the Friedreich’s Ataxia Research Alliance (FARA), Biogen reported that the BRAVE study had reached 50% enrolment.4 The same update confirmed the conclusion of the MOXIe open-label extension study and highlighted an ongoing bioequivalence study evaluating a tablet formulation of omaveloxolone that can be suspended in liquid relative to the currently available capsule formulation.4 This approach may help address swallowing difficulties experienced by some patients with FA.
Global reimbursement discussions continue across Canada, Australia and England
International reimbursement and access discussions are also progressing. In Canada, Quebec’s Institut national d’excellence en santé et en services sociaux recognized the therapeutic value of omaveloxolone and established reimbursement criteria for eligible patients, representing a step towards potential public reimbursement within the province.5 Additional reporting suggested that the recommendation may bring provincial coverage closer for eligible individuals living with FA in Quebec.6
In Australia, the Therapeutic Goods Administration registered omaveloxolone for adults and adolescents aged 16 years and older with FA.1 In England, however, the National Institute for Health and Care Excellence (NICE) was unable to make a recommendation on omaveloxolone for treating FA in people aged 16 years and over after Biogen withdrew its evidence submission during the appraisal process. NICE stated that the appraisal could resume if the company submits new evidence in the future.7
Long-term MOXIe data support sustained slowing of disease progression
Long-term findings continue to support the role of omaveloxolone in slowing disease progression. Data from the MOXIe open-label extension study, presented at the 2025 International Congress of Parkinson’s Disease and Movement Disorders, suggested that patients treated with omaveloxolone for approximately 4 years experienced slower disease progression compared with matched natural history cohorts.8 The safety profile remained broadly consistent with previous analyses and included manageable liver enzyme elevations and gastrointestinal adverse events.8
PASS registry to generate long-term real-world evidence in Friedreich’s ataxia
Real-world evidence generation is also underway through the omaveloxolone PASS registry, which is designed to collect long-term safety, effectiveness and patient experience data over a 5-year period.9 Together, these developments indicate that Biogen’s current FA strategy is focused on expanding omaveloxolone to younger patient populations, improving practical treatment accessibility and supporting long-term evidence generation.
References
1. Biogen. Friedreich ataxia. Available at: https://www.biogen.com/disease-areas/friedreich-ataxia.html (accessed June 15, 2026).
2. Biogen. Biogen initiates phase 3 pediatric study of omaveloxolone for the treatment of Friedreich ataxia [Press release]. June 18, 2025. Available at: https://investors.biogen.com/news-releases/news-release-details/biogen-initiates-phase-3-pediatric-study-omaveloxolone-treatment (accessed June 15, 2026).
3. Friedreich’s Ataxia Research Alliance. Press release and community statement: Biogen initiates phase 3 pediatric study of omaveloxolone for the treatment of Friedreich ataxia. Available at: https://www.curefa.org/news/press-release-and-community-statement-biogen-initiates-phase-3-pediatric-study-of-omaveloxolone-for-the-treatment-of-friedreich-ataxia/ (accessed June 15, 2026).
4. Friedreich’s Ataxia Research Alliance. Community statement: Update from Biogen. Available at: https://www.curefa.org/news/community-statement-update-from-biogen/ (accessed June 15, 2026).
5. Biogen Canada Inc. *New progress toward public reimbursement of SKYCLARYS™ for people living with Friedreich ataxia in Quebec* [Press release]. June 8, 2026. Available at: https://www.newswire.ca/news-releases/new-progress-toward-public-reimbursement-of-skyclarys-tm-for-people-living-with-friedreich-ataxia-in-quebec-875121400.html (accessed June 15, 2026).
6. Friedreich’s Ataxia News. Ataxia treatment SKYCLARYS closer to reimbursement in Quebec. 2026. Available at: https://friedreichsataxianews.com/news/ataxia-treatment-skyclarys-closer-reimbursement-quebec/ (accessed June 15, 2026).
7. National Institute for Health and Care Excellence. Omaveloxolone for treating Friedreich’s ataxia in people 16 years and over. NICE technology appraisal guidance TA1061. Available at: https://www.nice.org.uk/guidance/ta1061 (accessed June 15, 2026).
8. NeurologyLive. Long-term data suggest Friedreich ataxia agent omaveloxolone slows disease progression over 4 years. Available at: https://www.neurologylive.com/view/long-term-data-suggest-friedreich-ataxia-agent-omaveloxolone-slows-disease-progression-over-4-years (accessed June 15, 2026).
9. NeurologyLive. Expanding clinical care in Friedreich ataxia through the SKYCLARYS PASS registry. Available at: https://www.neurologylive.com/view/expanding-clinical-care-friedreich-ataxia-through-omaveloxlone-skyclarys-pass-registry (accessed June 15, 2026).
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Cite: Latest developments in omaveloxolone for Friedreich’s ataxia: Pediatric studies and global access. touchNEUROLOGY. 15 May 2026.
Editor: Katey Gabrysch, Editorial Director.
Disclosures: The content was developed and edited by human editors. No fees or funding were associated with its publication. touchNEUROLOGY utilize AI as an editorial tool (ChatGPT (GPT-4o) [Large language model]. https://chat.openai.com/chat).
This content has been developed independently by Touch Medical Media for touchNEUROLOGY. Views expressed are the speaker’s own and do not necessarily reflect the views of Touch Medical Media.
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