Skip to main content

Thank you for visiting nature.com. You are using a browser version with limited support for CSS. To obtain the best experience, we recommend you use a more up to date browser (or turn off compatibility mode in Internet Explorer). In the meantime, to ensure continued support, we are displaying the site without styles and JavaScript.

  • Comment
  • Published:

Approval of omaveloxolone for Friedreich ataxia

Nature Reviews Neurology (2024)Cite this article

Subjects

The recent approval of omaveloxolone for the treatment of Friedreich ataxia in the USA and Europe represents an important milestone in the field of rare neurological diseases. However, many challenges lie ahead, including the translation of trial results into clinical practice, and the management of patients’ expectations.

This is a preview of subscription content, access via your institution

Access options

Buy this article

  • Purchase on Springer Link
  • Instant access to full article PDF
Buy now

Prices may be subject to local taxes which are calculated during checkout

References

  1. Boesch, S. & Indelicato, E. Experimental drugs for Friedrich’s ataxia: progress and setbacks in clinical trials. Expert Opin. Investig. Drugs 32, 967–969 (2023).

    Article  CAS  PubMed  Google Scholar 

  2. Indelicato, E. et al. Onset features and time to diagnosis in Friedreich’s ataxia. Orphanet J. Rare Dis. 15, 198 (2020).

    Article  PubMed  PubMed Central  Google Scholar 

  3. Indelicato, E. et al. Predictors of survival in Friedreich’s ataxia: a prospective cohort study. Mov. Disord. 39, 510–518 (2024).

    Article  CAS  PubMed  Google Scholar 

  4. Indelicato, E. & Bösch, S. Emerging therapeutics for the treatment of Friedreich’s ataxia. Expert Opin. Orphan Drugs 6, 57–67 (2018).

    Article  CAS  Google Scholar 

  5. Lynch, D. R. et al. Safety and efficacy of omaveloxolone in Friedreich ataxia (MOXIe Study). Ann. Neurol. 89, 212–225 (2021).

    Article  CAS  PubMed  Google Scholar 

  6. Lynch, D. R. et al. Efficacy of omaveloxolone in Friedreich’s ataxia: delayed-start analysis of the MOXIe extension. Mov. Disord. 38, 313–320 (2023).

    Article  CAS  PubMed  Google Scholar 

  7. Lynch, D. R. et al. Propensity matched comparison of omaveloxolone treatment to Friedreich ataxia natural history data. Ann. Clin. Transl. Neurol. 11, 4–16 (2024).

    Article  CAS  PubMed  Google Scholar 

  8. Karthikeyan, G. et al. Reduction in frataxin causes progressive accumulation of mitochondrial damage. Hum. Mol. Genet. 12, 3331–3342 (2003).

    Article  CAS  PubMed  Google Scholar 

  9. Singh, I. et al. Investigation of mitochondrial DNA variations among Indian Friedreich’s ataxia (FRDA) patients. Mitochondrion 25, 1–5 (2015).

    Article  CAS  PubMed  Google Scholar 

  10. Indelicato, E. et al. Skeletal muscle proteome analysis underpins multifaceted mitochondrial dysfunction in Friedreich’s ataxia. Front. Neurosci. 17, 1289027 (2023).

    Article  PubMed  PubMed Central  Google Scholar 

Download references

Acknowledgements

S.B. and E.I. are members of the European Reference Network for Rare Neurological Diseases — project ID 739510.

Author information

Authors and Affiliations

  1. Center for Rare Movement Disorders Innsbruck, Department of Neurology, Medical University of Innsbruck, Innsbruck, Austria

    Sylvia Boesch & Elisabetta Indelicato

Authors
  1. Sylvia Boesch
    View author publications

    You can also search for this author in PubMed Google Scholar

  2. Elisabetta Indelicato
    View author publications

    You can also search for this author in PubMed Google Scholar

Corresponding author

Correspondence to Sylvia Boesch.

Ethics declarations

Competing interests

S.B. reports consultancy activity for VICO Therapeutics, REATA Pharmaceutics and Biogen, advisory board activity for REATA Pharmaceutics and Biogen, and honoraria from Ipsen, Merz Pharma, Abbvie, REATA Pharmaceutics and Biogen. E.I. declares no competing interests.

Additional information

Related links

Friedreich’s Ataxia Treatment Pipeline: https://www.curefa.org/research/research-pipeline

Rights and permissions

Reprints and permissions

About this article

Check for updates. Verify currency and authenticity via CrossMark

Cite this article

Boesch, S., Indelicato, E. Approval of omaveloxolone for Friedreich ataxia. Nat Rev Neurol (2024). https://doi.org/10.1038/s41582-024-00957-9

Download citation

  • Published:

  • DOI: https://doi.org/10.1038/s41582-024-00957-9

Search

Advanced search

Quick links

Nature Briefing

Sign up for the Nature Briefing newsletter — what matters in science, free to your inbox daily.

Get the most important science stories of the day, free in your inbox. Sign up for Nature Briefing