On February 28, 2023, the U.S. Food and Drug Administration (FDA) approved the first treatment for Friedreich’s ataxia. Friedreich’s ataxia is a very rare, inherited and debilitating disease caused by a genetic mutation, resulting in abnormal production of a protein called frataxin. Defective or missing fraxatin can lead to oxidative stress that damages cells in the nervous system, causing them to not signal properly. Sufferers experience losses in balance, coordination and muscle strength. Initially diagnosed during adolescence, the disease worsens gradually over time and can also lead to complications such as heart disease and diabetes. Skyclarys® (omaveloxolone) capsules are approved for the treatment of Friedreich’s ataxia in adults and adolescents 16 years and older. The exact mechanism of how Skyclarys® works is unclear, but it has been shown to activate the pathway involved in cellular response to oxidative stress. Skyclarys® was found to be effective and safe to treat Friedreich’s ataxia in a 48-week randomized, placebo-controlled, double-blind study. Part 2 of the MOXie study enrolled 103 individuals with Friedreich’s ataxia who were randomized to receive either placebo or Skyclarys® 150 mg for 48 weeks. The primary objective was to assess the change in disease progression compared to placebo at week 48. Patients receiving Skyclarys® demonstrated a slower decline in functioning compared to those receiving placebo. After the study was completed, participants could continue treatment with Skyclarys® in an extension study for up to three years. Those who continued with treatment performed better clinically compared to a matched set of untreated patients. The most common side effects noted during the study were elevated liver enzymes, headache, nausea and fatigue. Skyclarys® received orphan drug, fast track, priority review and rare pediatric disease designations from the FDA. Skyclarys® is expected to be available in the second quarter of 2023 through an integrated specialty pharmacy and patient service program developed by the manufacturer.