Reata Pharmaceuticals, Inc., a biopharmaceutical company focused on developing and commercializing novel therapies for patients with severe diseases, has received approval from the U.S. Food and Drug Administration for Skyclarys™ (omaveloxolone) as a treatment for Friedreich’s ataxia in adults and adolescents aged 16 years and older.
Friedreich’s ataxia is an ultra-rare, inherited neurodegenerative disorder that is typically diagnosed during adolescence and affects approximately 5,000 patients in the U.S. With this approval, the FDA granted a rare pediatric disease priority review voucher. The WAC Cost for a one-month supply is noted at $370,000.
Skyclarys™, an oral once-daily medication that has received Orphan Drug designation, is also under review in Europe by the European Medicines Agency (EMA).
Treatment with Skyclarys can cause an elevation in hepatic transaminases (alanine aminotransferase [ALT] and aspartate aminotransferase [AST]. Skyclarys can cause changes in cholesterol and increases in B-type natriuretic peptide (BNP), a marker of cardiac function.
Women are advised not to breastfeed during treatment with Skyclarys.
Anticipated commercial drug supply of Skyclarys will be available in the second quarter of 2023.
