FDA Grants Fast Track Designation to Reata’s Omaveloxolone for Treatment of Friedreich’s Ataxia

The U.S. Food and Drug Administration granted Reata Pharmaceuticals Fast Track designation for its experimental therapy omaveloxolone for the treatment of the rare degenerative neuromuscular disorder Friedreich’s ataxia.

Friedreich’s ataxia is caused by a trinucleotide repeat expansion in the first intron of the frataxin gene, which encodes the mitochondrial protein frataxin. Pathogenic repeat expansions can lead to impaired transcription and reduced frataxin expression, which can result in mitochondrial iron overload and poor cellular iron regulation, increased sensitivity to oxidative stress, and impaired mitochondrial ATP production.

Patients with Friedreich’s ataxia experience symptoms in childhood, including progressive loss of coordination, muscle weakness, and fatigue that commonly results in motor incapacitation with patients requiring a wheelchair in their teens or early 20s. Patients with Friedreich’s ataxia may also experience visual impairment, hearing loss, diabetes, and cardiomyopathy. There are currently no approved therapies for the treatment of patients with Friedreich’s ataxia.

Omaveloxolone is an oral, once-daily, activator of Nrf2, a transcription factor that induces molecular pathways that promote the resolution of inflammation by restoring mitochondrial function, reducing oxidative stress, and inhibiting pro-inflammatory signaling. The FDA has granted Orphan Drug and Fast Track designations to omaveloxolone for the treatment of Friedreich’s ataxia. The European Commission has granted Orphan Drug designation in Europe to omaveloxolone for the treatment of Friedreich’s ataxia.

The Fast Track program is designed to accelerate the development and review of products such as omaveloxolone, which are intended to treat serious diseases and for which there is an unmet medical need. Fast Track designation enables more frequent communication with the FDA and eligibility for FDA programs such as priority review and rolling review, if relevant criteria are met.

Warren Huff, Reata’s president and CEO said the company is on track to submit a New Drug application during the first quarter of 2022 and continues working with the FDA to secure regulatory approval as quickly as possible.

Author: Rare Daily Staff