FDA Grants Special Designation to Friedreich’s Ataxia Treatment Based on Findings from Study Led by CHOP Neurologist

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Friedreich’s Ataxia is a progressive neurological disorder associated with the loss of the ability to walk, loss of hand coordination and loss of speech. Patients also develop heart disease that can be fatal. The disorder results from a relative lack of the protein frataxin, which causes abnormal mitochondrial function and lack of energy production and thus gives rise to the features of the disease. Recent evidence suggests that omaveloxolone may reverse some of these features, leading to stabilization of disease progression. This drug represents a potential new therapy for Friedreich’s Ataxia.

Omaveloxolone is an investigational, oral, once-daily, activator of Nrf2, a transcription factor that induces molecular pathways that promote the resolution of neuroinflammation by restoring mitochondrial function, reducing oxidative stress, and inhibiting pro-inflammatory signaling. David Lynch, MD, PhD, Director of the Friedreich’s Ataxia Program at Children’s Hospital of Philadelphia (CHOP), has been studying the efficacy of omaveloxolone for years in collaboration with others around the world and with Reata Pharmaceutical, a clinical-stage biopharmaceutical company.

Dr. Lynch and colleagues recently published results of a clinical trial — MOXIe Part 1, Part 2 and Extension studies of omaveloxolone — showing that omaveloxolone has a potentially beneficial effect in slowing the progression of Friedreich’s ataxia out to more than two years.

According to Dr. Lynch, the results from the trial show:

  1. Omaveloxolone modifies the long-term behavior of the disease.
  2. When analyzed in a delayed-start fashion, it doesn’t matter when you start omaveloxolone to see a benefit. Each cohort in the trial benefited almost equally once they started the drug.

In short, it appears that this drug truly provides benefit to patients at all stages.

The trial was sponsored by Reata Pharmaceuticals. Using the efficacy and safety data from the trial, Reata submitted a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for omaveloxolone for patients aged 16 and older.

The FDA recently granted Fast Track Designation and Orphan Drug Designation to omaveloxolone for the treatment of Friedreich’s Ataxia, making it the first medication for Friedreich’s Ataxia. The European Commission has also granted Orphan Drug Designation in Europe to omaveloxolone for the treatment of Friedreich’s Ataxia.

Omaveloxolone: potential new agent for Friedreich ataxia. Lynch DR, Johnson J. Neurodegener Dis Manag. 2021;11(2):91-98.

Safety and efficacy of omaveloxolone in Friedreich ataxia (MOXIe Study). Lynch DR, Chin MP, Delatycki MB, Subramony SH, Corti M, Hoyle JC, Boesch S, Nachbauer W, Mariotti C, Mathews KD, Giunti P, Wilmot G, Zesiewicz T, Perlman S, Goldsberry A, O'Grady M, Meyer CJ. Ann Neurol. 2021;89(2):212-225.

Safety, pharmacodynamics, and potential benefit of omaveloxolone in Friedreich ataxia. Lynch DR, Farmer J, Hauser L, Blair IA, Wang QQ, Mesaros C, Snyder N, Boesch S, Chin M, Delatycki MB, Giunti P, Goldsberry A, Hoyle C, McBride MG, Nachbauer W, O'Grady M, Perlman S, Subramony SH, Wilmot GR, Zesiewicz T, Meyer C. Ann Clin Transl Neurol. 2018;6(1):15-26.