FDA Approves First Treatment for Rare, Degenerative Disease

Jessica Ganga

The FDA has approved omaveloxolone for the treatment of Friedreich ataxia, an inherited, degenerative disease that affects the nervous system, causing impaired coordination and walking. Although rare, Friedreich ataxia is a condition that can also cause heart disease.

The oral capsule treatment’s approval is the first-of-its-kind for the treatment of Friedreich ataxia. The approval of omaveloxolone comes after a 48-week randomized, placebo-controlled, and double-blind study and an open-label extension. In total, 103 individuals with Friedeich ataxia participated and were evaluated in the study, where they received either placebo (n = 52) or 150 mg of omaveloxolone (n = 51).

“The primary objective was to evaluate the change in the modified Friedreich’s Ataxia Rating Scale (mFARS) score compared to placebo at week 48,” according to the FDA’s news release. “The mFARS is a clinical assessment that measures disease progression, namely swallowing and speech (bulbar), upper limb coordination, lower limb coordination, and upright stability.”

The researchers found that patients taking omaveloxolone performed better on the mFARS in comparison to those who received placebo. Further, in a post hoc analysis, individuals who continued treatment for up to three years performed better on the mFARS when compared to a matched set of untreated patients.

The most common adverse events included an increase in alanine transaminase and increase of aspartate aminotransferase. These can be signs of liver damage, headache, nausea, abdominal pain, fatigue, diarrhea, and musculoskeletal pain.



FDA approves first treatment for Friedrich’s atakia. News release. US Food and Drug Administration; February 28, 2023. Accessed April 18, 2023.