Omaveloxolone: First Approval
Omaveloxolone (SKYCLARYS™) is an orally active, small molecule semi-synthetic triterpenoid drug that increases antioxidant activity, which is being developed by Reata Pharmaceuticals, Inc. for the treatment of Friedreich’s ataxia. In patients with Friedreich’s ataxia, the nuclear factor (erythroid-d...
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| Veröffentlicht in: | Drugs (New York, N.Y.) N.Y.), 2023-06, Vol.83 (8), p.725-729 |
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| creator | Lee, Arnold |
| description | Omaveloxolone (SKYCLARYS™) is an orally active, small molecule semi-synthetic triterpenoid drug that increases antioxidant activity, which is being developed by Reata Pharmaceuticals, Inc. for the treatment of Friedreich’s ataxia. In patients with Friedreich’s ataxia, the nuclear factor (erythroid-derived 2)-like 2 (Nrf2) pathway is suppressed, which is associated with oxidative stress, mitochondrial dysfunction and damage to cells, including central and peripheral neurones. The Nrf2 pathway may be activated by omaveloxolone as it blocks the ubiquitination and degradation of Nrf2. Omaveloxolone was approved in February 2023 in the USA for the treatment of Friedreich's ataxia. This article summarizes the milestones in the development of omaveloxolone leading to this first approval for the treatment of Friedreich's ataxia in adults and adolescents aged 16 years and older. |
| doi_str_mv | 10.1007/s40265-023-01874-9 |
| format | Article |
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In patients with Friedreich’s ataxia, the nuclear factor (erythroid-derived 2)-like 2 (Nrf2) pathway is suppressed, which is associated with oxidative stress, mitochondrial dysfunction and damage to cells, including central and peripheral neurones. The Nrf2 pathway may be activated by omaveloxolone as it blocks the ubiquitination and degradation of Nrf2. Omaveloxolone was approved in February 2023 in the USA for the treatment of Friedreich's ataxia. 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Springer Nature or its licensor (e.g. a society or other partner) holds exclusive rights to this article under a publishing agreement with the author(s) or other rightsholder(s); author self-archiving of the accepted manuscript version of this article is solely governed by the terms of such publishing agreement and applicable law.</rights><rights>2023. The Author(s), under exclusive licence to Springer Nature Switzerland AG.</rights><rights>Copyright Springer Nature B.V. 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| ispartof | Drugs (New York, N.Y.), 2023-06, Vol.83 (8), p.725-729 |
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| subjects | AdisInsight Report Adolescent Adolescents Adult Antioxidants Antioxidants - therapeutic use Ataxia Drug dosages Enzymes Friedreich Ataxia - complications Friedreich Ataxia - metabolism Friedreich's ataxia Humans Internal Medicine Laboratories Medicine Medicine & Public Health Mitochondria NF-E2-Related Factor 2 - metabolism NF-E2-Related Factor 2 - therapeutic use Oxidative stress Pharmaceuticals Pharmacology/Toxicology Pharmacotherapy Teenagers Triterpenes - pharmacology Triterpenes - therapeutic use Ubiquitination Workloads |
| title | Omaveloxolone: First Approval |
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