The US Food and Drug Administration (FDA) has approved omaveloxolone (Skyclarys™) as the first drug indicated for the treatment of Friedreich ataxia (FA) in adults and adolescents aged 16 years and older, Reata Pharmaceuticals announced.
“The approval of Skyclarys, the first therapy specifically indicated for the treatment of FA, is an important milestone for patients affected by this disease as well as their families and caregivers,” said Warren Huff, Reata’s chief executive officer.
The approval of omaveloxolone is supported by the efficacy and safety data obtained through the MOXIe Part 2 trial and a post hoc propensity-matched analysis of the open-label MOXIe extension trial.
During MOXIe Part 2, a randomized, double-blind, placebo-controlled study, patients with genetically confirmed FA and baseline-modified FA Rating Scale (mFARS) scores between 20 and 80 were administered daily doses of placebo or 150 mg of omaveloxolone.
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According to the results, treatment with omaveloxolone resulted in lower mFARS scores indicating less impairment compared with placebo at week 48. Elevated liver enzymes (AST/ALT), headache, nausea, abdominal pain, fatigue, diarrhea, and musculoskeletal pain were reported as the most common adverse reactions.
The post hoc propensity-matched analysis compared the mFARS progression of MOXIe extension trial patients treated with omaveloxolone and the progression of propensity score-matched untreated patients in the largest natural history study of FA, called Clinical Outcome Measures in FA (FA-COMS). Patients from the MOXIe extension study with at least one post-baseline assessment were matched 1:1 with patients from the FA-COMS study.
The analysis revealed an mFARS score improvement in patients treated with omaveloxolone after 3 years relative to the matched set of untreated patients from the FA-COMS natural history study. However, “these exploratory analyses should be interpreted cautiously given the limitations of data collected outside of a controlled study, which may be subject to confounding,” Reata said in a press release.
FA, an inherited neurodegenerative disorder typically diagnosed during adolescence, affects approximately 5000 diagnosed patients in the US. Reata is a biopharmaceutical company focused on developing novel therapies for patients with severe diseases.
Reata Pharmaceuticals announces FDA approval of SKYCLARYS™ (omavaloxolone), the first and only drug indicated for patients with Friedreich’s ataxia. News Release. Reata Pharmaceuticals; February 28, 2023.