24 November 2021 - - The US Food and Drug Administration has granted Fast Track Designation for omaveloxolone for the treatment of Friedreich's ataxia, US-based biopharmaceutical company Reata Pharmaceuticals, Inc. (NASDAQ: RETA) said.

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.

Friedreich's ataxia is a rare, genetic, life-shortening, debilitating, and degenerative neuromuscular disorder, which is normally diagnosed during adolescence.

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.

Based on literature and proprietary research, we believe Friedreich's ataxia affects approximately 5,000 children and adults in the United States and 22,000 individuals globally.

There are currently no approved therapies for the treatment of patients with 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 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.

Reata is a clinical-stage biopharmaceutical company that develops novel therapeutics for patients with serious or life-threatening diseases by targeting molecular pathways involved in the regulation of cellular metabolism and inflammation.

Reata's two most advanced clinical candidates, bardoxolone methyl and omaveloxolone, target the important transcription factor Nrf2 that promotes the resolution of inflammation by restoring mitochondrial function, reducing oxidative stress, and inhibiting pro-inflammatory signaling.

Bardoxolone and omaveloxolone are investigational drugs, and their safety and efficacy have not been established by any agency.