Omaveloxolone (Skyclarys; Reata Pharmaceuticals, Plano, TX) has been approved by the Food and Drug Administration (FDA) as the first therapy to treat Friedreich ataxia (FA) in adults and adolescents aged 16 years and older. Those who received omaveloxolone improved by −1.63 ± 1.78 points on the Modified Friedreich's Ataxia Rating Scale (mFARS), while those who received a placebo worsened by +2.52 ± 1.18 points on mFARS at week 48. In the omaveloxolone cohort, participants noted an improvement in each component of the mFARS compared with the placebo cohort. The rates of adverse events in both cohorts were similar, with most common adverse events being mild to moderate in intensity. Patients who received omaveloxolone were more likely to experience headaches, nausea, and increased alanine and aspartate aminotransferase (ALT and AST, respectively).
MOXIe (NCT02255435) was an international, double‐blind, randomized, placebo‐controlled, parallel‐group, registrational phase 2 trial that determined the safety and efficacy of omaveloxolone over 48 weeks. The trial took place at 11 healthcare facilities in the United States, Europe, and Australia. Participants were included if they were between 16 and 40 years of age, had genetically confirmed FA, baseline mFARS scores between 20 and 80, and could use a recumbent stationary bicycle for physical testing. Patients were excluded for uncontrolled diabetes, cardiac disease, active infections, laboratory abnormalities, or medical conditions that would alter the testing. Participants (n=103) were 1:1 randomized in an intent-to-treat cohort (n=51) to receive 150 mg per day of omaveloxolone or the placebo cohort (n=52). Maximal exercise testing using the stationary bike and mFARS assessments were completed at weeks 4, 12, 18, 24, 36, and 48. The primary outcome was the change in mFARS scores from baseline and 48 weeks in the omaveloxolone cohort compared with those in the placebo cohort. A post hoc analysis was performed to account for the variety of covariates present at baseline.
According to Susan Perlman, Department of Neurology, David Geffen School of Medicine, UCLA, “The approval of Skyclarys (omaveloxolone) represents an important step forward in the treatment of Friedreich's ataxia, providing physicians with the first disease-specific treatment option approved for patients living with this ultra-rare and progressive disease.”
Friedreich ataxia is a rare, inherited neuromuscular disease that causes impaired muscle coordination, reduced sensation, and increased spasticity. An estimated 1 in 50,000 individuals is affected by this chronic, progressive condition.
https://practicalneurology.com/news/omaveloxolone-approved-by-fda-as-first-therapy-for-friedreich-ataxia
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