Omaveloxolone approved for patients aged 16 years and older with Friedreich ataxia (FRDA): A therapeutics bulletin of the American College of Medical Genetics and Genomics (ACMG)

Arthur Lenahan, Sho Yano, Brett Graham, Kuntal Sen, Omaveloxolone approved for patients aged 16 years and older with Friedreich ataxia (FRDA): A therapeutics bulletin of the American College of Medical Genetics and Genomics (ACMG), Genetics in Medicine Open, Volume 1, Issue 1, 2023, 100832, ISSN 2949-7744, doi:10.1016/j.gimo.2023.100832. 

Omaveloxolone (trade name: SKYCLARYS) is a Nrf2 activator that increases cellular resilience to oxidative stress that has been FDA approved for patients with FRDA who are 16 years and older. Omaveloxolone received orphan drug, fast track, priority review, and rare pediatric disease designations. Omaveloxolone is a semisynthetic triterpenoid administered orally once daily.