Texas-based Reata Pharmaceuticals announced this week that the FDA approved its rare disease drug, omavaloxolone, for the treatment of Friedreich’s ataxia in adults and adolescents aged 16 years and older.
The drug, to be sold as Skyclarys, is not only the company’s first approval ever, but also the first therapy specifically indicated for the treatment of Friedrich’s ataxia. The inherited neurodegenerative disorder is usually diagnosed in adolescence, and affects roughly 5,000 people annually in the U.S. Patients with Friedrich’s ataxia suffer from a progressive loss of coordination and muscle weakness with a typical progresssion to motor incapacitation by early adulthood and eventually death.
Skyclarys’ approval was supported by efficacy and safety data from the MOXIe Part 2 trial and a post hoc Propensity-Matched Analysis of the open-label MOXIe extension trial, in which the drug showed to reduce patients’ impairment relative to placebo at week 48.
Wall Street also echoed the feelings of celebration with Reata’s stock soaring 198.9% and closing at 93.17 on the day the approval news was shared.
