- The FDA approved Reata Pharmaceuticals Inc's RETA Skyclarys (omaveloxolone) for Friedreich's ataxia in adults and adolescents aged 16 and older.
- With this approval, the FDA granted a rare pediatric disease priority review voucher.
- The company says Skyclarys is the first therapy specifically indicated for Friedreich's ataxia.
- Related: Why Reata Pharmaceuticals Shares Are Falling Monday?
- Friedreich's ataxia is an ultra-rare, inherited neurodegenerative disorder typically diagnosed during adolescence.
- Patients with Friedreich's ataxia experience progressive loss of coordination, muscle weakness, and fatigue, which commonly progresses to motor incapacitation and wheelchair reliance by their teens or early twenties, eventually death.
- Friedreich's ataxia affects approximately 5,000 diagnosed patients in the U.S.
- The approval of Skyclarys is supported by the efficacy and safety data from the MOXIe Part 2 trial and a post hoc Propensity-Matched Analysis of the open-label MOXIe Extension trial.
- Treatment with Skyclarys resulted in statistically significantly lower mFARS scores (less impairment) than placebo at Week 48.
- Further, in a post hoc Propensity-Matched Analysis, lower (improved) mFARS scores were observed in patients treated with Skyclarys after three years relative to the matched set of untreated patients from the FA-COMS natural history study.
- The company is completing commercial drug product manufacturing and anticipates a commercial drug supply of Skyclarys to be available in the second quarter of 2023.
- Price Action: RETA shares are up 166.12% at $82.95 during the premarket session on the last check Wednesday.
© 2024 Benzinga.com. Benzinga does not provide investment advice. All rights reserved.
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