From the President & CEO of Reata Pharmaceuticals
Warren Huff, President and Chief Executive Officer, Reata Pharmaceuticals, said, “We welcome the opportunity to have a pre-NDA meeting regarding our omaveloxolone development program for the treatment of patients with FA. “We look forward to working with the FDA on our goal of securing the regulatory review and approval necessary to make omaveloxolone available to patients with Friedreich’s ataxia.”
Friedreich’s ataxia is a rare, inherited, life-shortening, debilitating and degenerative neuromuscular, normally diagnosed during adolescence. The disease is 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 lead to mitochondrial iron overload, poor cellular iron regulation, increased sensitivity to oxidative stress and impaired mitochondrial ATP production.
Patients with Friedreich’s ataxia experience initial symptoms in childhood which include:
- Progressive loss of coordination
- Muscle weakness
- Fatigue – causing motor incapacitation with young patients requiring a wheelchair
- Visual impairment
- Hearing loss
Based on literature and proprietary research, Friedreich’s ataxia seems to affect around 5,000 children and adults in the United States and 22,000 individuals globally. There are currently no approved therapies for the treatment of FA.
Omaveloxolone is an investigational, oral, once-daily activator of Nrf2, a transcription factor that induces molecular pathways that promote the resolution of inflammation. The resolution of inflammation occurs through 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. Also, the European Commission has granted the same product Orphan Drug designation in Europe for the treatment of Friedreich’s ataxia.
Reata Pharmaceuticals 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 most advanced clinical candidates, methyl bardoxolone and omaveloxolone, target the important transcription factor Nrf2 that promotes the resolution of inflammation through restoring mitochondrial function, reducing oxidative stress, and inhibiting pro-inflammatory signaling.
Reata possesses exclusive, worldwide rights to develop, manufacture and commercialize omaveloxolone and its other next-generation Nrf2 activators.
Bardoxolone and omaveloxolone are investigational drugs, and their safety and efficacy have not been established by any agency.
This is positive news that should halt the gossip about Reata Pharmaceuticals’ capability to treat Friedreich’s ataxia. The pre-NDA meeting, requested by the U.S. FDA, is expected to end the unwarranted devastation of this firm’s stock at the hands of critics and negative investors.
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Reata Pharmaceuticals: Good News from the FDA Regarding Omaveloxolone for Friedreich’s Ataxia
Reata Pharmaceuticals Good News
Reata Pharmaceuticals (RETA) announced it received a communication from the Division of Neurology Products of the U.S. FDA stating, that after a preliminary review of briefing materials for an upcoming Type C meeting, a pre-NDA meeting is the most appropriate format for a discussion of the development program for omaveloxolone in Friedreich’s ataxia
The Division suggested that the Company withdraw the current meeting request for a Type C meeting and instead request a pre-NDA meeting that the Division will grant upon receipt. The Division asked the Company to focus the new briefing package on questions, issues and needs applicable to a pre-NDA meeting. As requested by the FDA the Company currently plans to withdraw the current request for a Type C meeting and submit a request for a pre-NDA meeting as soon as practicable.