Omaveloxolone for the treatment of Friedreich ataxia: clinical trial results and practical considerations

ABSTRACT

Introduction

Omavaloxolone, an NRF2 activator, recently became the first drug approved specifically for the treatment of Friedreich ataxia (FRDA). This landmark achievement provides a background for a review of the detailed data leading to the approval.

Areas covered

The authors review the data from the 4 major articles on FRDA in the context of the authors’ considerable (>1000 patients) experience in treating individuals with FRDA. The data is presented in the context not only of its scientific meaning but also in the practical context of therapy in FRDA.

Expert opinion

Omaveloxolone provides a significant advance in the treatment of FRDA that is likely to be beneficial in a majority of the FRDA population. The data suggesting a benefit is consistent, and adverse issues are relatively modest. The major remaining questions are the subgroups that are most responsive and how long the beneficial effects will remain significant in FRDA patients.

KEYWORDS:

• Antioxidant
• KEAP1
• clinical trial
• ferritin
• mFARS
• Friedrech ataxia

Article highlights

• Omaveloxolone is now approved for use in Friedreich ataxia (FRDA).

• Approval reflects consistent benefit with relatively few adverse events over several studies.

• Response to omaveloxolone persists for several years following initiation, but it is not curative.

• Monitoring of transaminases is needed during clinical therapy.

• The use of omaveloxolone in more diverse FRDA populations will help aid in the understanding of the complete clinical profile of omaveloxolone and its utility in different subgroups.

Declaration of interest

DR Lynch and S Perlman have received grant funding from Reata Pharmaceuticals for their participation in the MOXIe study. The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed.

Reviewer disclosures

Peer reviewers on this manuscript have no relevant financial or other relationships to disclose.

Additional information

Funding

This paper was not funded.