On September 30, 2021 Reata Pharmaceuticals, Inc. (Nasdaq: RETA) ("Reata," the "Company," or "we"), a clinical-stage biopharmaceutical company, reported that it has completed its pre-New Drug Application ("NDA") meeting with the United States Food and Drug Administration ("FDA") for omaveloxolone for the treatment of patients with Friedreich’s ataxia and reaffirmed its plan to submit an NDA in the first quarter of 2022 (Press release, Reata Pharmaceuticals, SEP 30, 2021, View Source [SID1234590580]).
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The purpose of the pre-NDA meeting was to discuss the content of Reata’s planned NDA submission. We plan to submit the NDA seeking standard approval for omaveloxolone for the treatment of Friedrich’s ataxia. We are not planning to conduct a second pre-approval clinical study prior to the submission. The FDA indicated that the appropriate approval pathway would be a matter of review after submission of the NDA. In response to our questions about the contents of the filing and because of the seriousness of the indication, the FDA exercised its discretion subject to review to permit us to submit the results of certain nonclinical and clinical studies after approval.
"We are pleased with the outcome of our recent pre-NDA meeting and that we have a path to submit our NDA in the first quarter of 2022," said Warren Huff, Reata’s President and Chief Executive Officer. "Friedreich’s ataxia is a severe, ultra-rare disease that affects approximately 5,000 patients in the United States. We remain committed to our goal of working with the FDA to secure regulatory approval for omaveloxolone as quickly as possible for patients with this devastating disease."
"Omaveloxolone could be the first drug approved for the treatment of Friedreich’s ataxia—actually the first drug approved for any ataxia," said Dr. Susan Perlman, MD, Professor, Department of Neurology, David Geffen School of Medicine, University of California, Los Angeles, CA. "The MOXIe Part 2 study with omaveloxolone is the first to demonstrate a significant improvement in neurological function in patients with Friedreich’s ataxia. While not a cure, if approved, Friedreich’s ataxia would finally become a treatable disease, something the Friedreich’s ataxia community has been working towards for a long time."
About Friedreich’s Ataxia
Friedreich’s ataxia is a rare, inherited, 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 resulting in motor incapacitation with patients requiring a wheelchair by 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.
About Omaveloxolone
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 designation 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.