If Approved, Omaveloxolone Would Become the First Therapy Indicated for the Treatment of Patients with Friedreich’s Ataxia
Friedreich’s Ataxia Affects Approximately 5,000 Patients in the United States with an Estimated 4,000 Diagnosed Patients
Reata Pharmaceuticals, Inc. (Nasdaq: RETA), (“Reata,” the “Company,” “our,” “us,” or “we”), a clinical-stage biopharmaceutical company, today announced the completion of the rolling submission of a New Drug Application (“NDA”) to the U.S. Food and Drug Administration (“FDA”) for omaveloxolone for the treatment of patients with Friedreich’s ataxia. This NDA is supported by the efficacy and safety data from the MOXIe Part 1, Part 2, and MOXIe Extension studies. The FDA has granted Fast Track Designation and Orphan Drug Designation to omaveloxolone for the treatment of Friedreich’s ataxia.
“This NDA submission marks an important step toward making a treatment available for patients with Friedreich’s ataxia, a serious degenerative neuromuscular disorder with no approved therapies,” said Warren Huff, Reata’s Chief Executive Officer. “I want to thank all those who made this possible, especially our patients with Friedreich’s ataxia, their families, and investigators, as well as our employees, for their tenacity and unwavering belief in the omaveloxolone clinical program. We look forward to the next steps on the path to making omaveloxolone available as a first-in-class therapy for Friedreich’s ataxia, pending approval.”
About Friedreich's Ataxia
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 teens or early 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 the mid-thirties. 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 FA 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 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, omaveloxolone and bardoxolone methyl (“bardoxolone”), 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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