What is the status of development?

History of Development for SKYCLARYS

The drug development process can be thought of as a series of stages, and successful drugs must pass through each stage to become available to patients.


Several academic research labs demonstrated in both human FA cell models and mouse models that Nrf2 is downregulated. In addition, treatment of FA cell and animal models with omavoloxone demonstrated activation of Nrf2 and improvements in the mitochondrial function. Studies have shown that treatment with omav reduced pathologic levels of oxidative stress, restored antioxidative response, restored complex 1 activity, decreased lipid peroxidation, and decreased mitochondrial reactive oxygen species (ROS). In cellular assays, omav prevented cell death following pro-oxidant challenge. See links to published papers to learn more about these studies.

Working with FARA and several FA Investigators in our Collaborative Clinical Research Network (CCRN), Reata conducted a two-part, randomized, placebo-controlled Phase 2/3 Study of the Safety, Efficacy, and Pharmacodynamics of omav. Part 1 of the study was a randomized, placebo-controlled, double-blind, dose-escalation study. Part 2 of the study was conducted to establish the longer-term effectiveness of omav. Finally, Reata also conducted an open label extension study. People who participated in MOXIe part 1 or 2 were able to take omav and continue to be followed for assessment of both efficacy and safety.

June 2023: Reata reported in a press release that the United States Food and Drug Administration (FDA) has approved the Prior Approval Supplement (PAS) to update the drug substance specification for SKYCLARYS® (omaveloxolone), the first and only FDA approved drug for the treatment of Friedreich’s ataxia in adults and adolescents aged 16 years and older. With the approval of the PAS, SKYCLARYS is now available to patients with Friedreich’s ataxia in the United States

February 28, 2023: Reata Pharmaceuticals announced that the United States Food & Drug Administration (FDA) has approved their New Drug Application (NDA) for SKYCLARYS™ (aka omaveloxolone or omav) in FA for people 16 years of age and older. This marks a milestone for the FA community- the first approved treatment for FA.

February 12, 2024: Biogen Inc. announced the European Commission (EC) has authorized SKYCLARYS® (omaveloxolone) for the treatment of Friedreich’s ataxia (FA) in adults and adolescents aged 16 years and older. SKYCLARYS is the first treatment approved within the European Union for this rare, genetic, progressive neurodegenerative disease.

May 20, 2024: Biogen initiated an open-label study evaluating the safety, tolerability, and pharmacokinetics (PK) of omaveloxolone following single dose administration in pediatric patients with FA.

The first study cohort includes participants who are 12 to 15 years old with genetically confirmed FA.

Younger cohorts (between ages 6-11 and 2-5 years) will be recruited separately after safety data from the oldest cohort is reviewed.


Two-Minute Mechanism Video: Omaveloxolone

This short video explains how omaveloxolone works to potentially provide benefit to someone with FA.

Image of Video title - Omaveloxolone

News and Press Releases

| Mar 4, 2024

FA Community Update from Biogen

Industry News
| Feb 12, 2024

Biogen Received European Commission Approval for SKYCLARYS® (omaveloxolone), the First Therapy to Treat Friedreich’s Ataxia

Industry News
| Dec 15, 2023

CHMP Issues Positive Opinion for Biogen’s SKYCLARYS® (omaveloxolone), the First Therapy to Treat Friedreich’s Ataxia, a Rare Neurodegenerative Disease

Industry News
| Sep 26, 2023

Biogen Completes Acquisition of Reata Pharmaceuticals

Industry News

SKYCLARYS Clinical Trials

Improve Mitochondrial Function & Reduce Oxidative Stress
Clinical Trial | Ages 12-15

Omaveloxolone Dose Finding Study in Children With Friedreich’s Ataxia

Phase I | In-person