Synthesis and Biological Profile of Omaveloxolone: The Cornerstone for Friedreich Ataxia Treatment

Cordaro, M.; Neri, G.; Ansari, S.A.M.K.; Buccheri, R.; Scala, A.; Piperno, A. Synthesis and Biological Profile of Omaveloxolone: The Cornerstone for Friedreich Ataxia Treatment. Int. J. Mol. Sci. 2025, 26, 9747. doi:10.3390/ijms26199747 

This review provides a comprehensive overview of the therapeutic potential of omaveloxone (OMA) for the treatment of Friedreich’s ataxia (FA), along with an analysis of the historical development and current status of the synthetic strategies for OMA production. OMA activates the nuclear factor-2-(erythroid-2)-related (Nrf2) pathway in vitro and in vivo, in both animal models and humans. The Nrf2 pathway plays a crucial role in the cellular response to oxidative stress. Furthermore, OMA has been shown to mitigate mitochondrial dysfunction, restore redox homeostasis and downregulate nuclear factor-κB (NF-κB), a key mediator of inflammatory responses. Through these mechanisms, OMA contributes to tissue protection and inflammation reduction in patients with FA. The review also highlights future perspective, focusing on the challenges associated with OMA reprofiling through innovative drug delivery approaches and its potential repurposing for diseases beyond FA.

Lexeo Therapeutics Stock Rallies On Discussions With FDA To Expedite Friedreich’s Ataxia Drug Approval Process

Published Oct 07, 2025. https://stocktwits.com 

The company stated that data from a planned pivotal study will be pooled with data from the ongoing Phase I/II studies of LX2006 to support an approval application to the U.S. Food and Drug Administration for the therapy. 

 Lexeo Therapeutics (LXEO) announced on Tuesday that the company is considering a smaller pivotal study for LX2006 in the treatment of Friedreich's ataxia (FA) cardiomyopathy, scheduled to begin in the first half of 2026, pending finalization of the study protocol. 

 The data from the planned pivotal study will be pooled with data from the ongoing Phase I/II studies of LX2006 to support an approval application to the U.S. Food and Drug Administration for the therapy, the company stated after discussions with the agency.

Lexeo says FDA open to speedier approval of rare disease gene therapy

Published Oct. 7, 2025. https://www.biopharmadive.com/ 

The agency will consider a submission that includes pooled data from ongoing studies, a decision analysts viewed as a notable, additional sign of regulatory flexibility for gene therapies.

According to the company, the agency has “indicated openness” to an accelerated approval filing for its treatment — a gene therapy called LX2006 for the neurodegenerative condition Friedreich’s ataxia — that’s based on pooled data from ongoing studies as well as results from a planned pivotal trial.

A25-86 Omaveloxolone (Friedreich’s ataxia) – Benefit assessment according to §35a Social Code Book V

Commission awarded on 01.07.2025 by the Federal Joint Committee (G-BA). Last updated 01.10.2025. 

Result of dossier assessment: Added benefit not proven.

Delphi study to elicit expert consensus around decision-making in the treatment of Friedreich ataxia

Delphi study to elicit expert consensus around decision-making in the treatment of Friedreich ataxia. Front. Neurol.Sec. Movement Disorders Volume 16 - 2025 | doi: 10.3389/fneur.2025.1669059

Consensus was reached on a portion of questions regarding FA diagnosis and assessment, perhaps due to the rarity of disease and panelists' varying FA experience. To improve and standardize management of FA, it is important to establish best practices and educate potential FA treaters as new therapies emerge.