Policy & Regulation
US FDA Accepts Reata Pharmaceuticals Filing, Grants Priority Review Designation for the NDA for Omaveloxolone for the Treatment of Patients with Friedreich's Ataxia
27 May 2022 - - The US Food and Drug Administration has accepted for filing and granted Priority Review of US-based clinical-stage biopharmaceutical company Reata Pharmaceuticals, Inc's (NASDAQ: RETA) New Drug Application for omaveloxolone for the treatment of patients with Friedreich's ataxia, the company said.

The FDA indicated that at this time it has not identified any potential review issues.

The NDA is supported by the efficacy and safety data from the MOXIe Part 2 trial and additional supporting data from the MOXIe Part 1 and MOXIe Extension trials. Omaveloxolone received Fast Track Designation in November 2021 and Rare Pediatric Disease Designation in May 2022.

The FDA grants Priority Review to medicines that may offer significant improvements in the treatment, diagnosis, or prevention of a serious condition.

This Designation shortens the FDA's review of the NDA to eight months from the time of submission, versus a standard review timeline of 12 months.

The FDA has assigned a Prescription Drug User Fee Act target action date of November 30, 2022. The FDA indicated it is currently planning to hold an advisory committee meeting to discuss the application.

Friedreich's ataxia is a rare, genetic, life-shortening, debilitating, and degenerative neuromuscular disorder typically 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 typically 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 20s.

Patients with Friedreich's ataxia may also experience visual impairment, hearing loss, diabetes, and cardiomyopathy.

On average, patients with Friedreich's ataxia die in their mid 30s. 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 an estimated 4,000 patients diagnosed with Friedreich's ataxia in the United States. Currently, there are no approved therapies for the treatment of 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, Fast Track, and Rare Pediatric Disease 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, omaveloxolone and bardoxolone methyl, target the important transcription factor Nrf2 that promotes the resolution of inflammation by restoring mitochondrial function, reducing oxidative stress, and inhibiting pro-inflammatory signaling. 

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