Omaveloxolone
- Generic Name
- Omaveloxolone
- Brand Names
- Skyclarys
- Drug Type
- Small Molecule
- Chemical Formula
- C33H44F2N2O3
- CAS Number
- 1474034-05-3
- Unique Ingredient Identifier
- G69Z98951Q
- Background
Omaveloxolone (RTA-408) is a semisynthetic oleanane triterpenoid with antioxidant and anti-inflammatory properties. Omaveloxolone acts as an activator of nuclear factor (erythroid-derived 2)-like 2 (Nrf2), a transcription factor that mitigates oxidative stress. In patients with Friedreich's ataxia, a genetic disease involving mitochondrial dysfunction, the Nrf2 pathway is impaired, and Nrf2 activity is lower. Therefore, the use of Nrf2 activators such as omaveloxolone represents a therapeutic advantage in this group of patients. In February 2023, omaveloxolone was approved by the FDA for the treatment of Friedreich's ataxia in adults and adolescents aged 16 years and older. The use of omaveloxolone for the treatment of conditions involving mitochondrial dysfunction and oxidative stress has also been evaluated.
- Indication
Omaveloxolone is indicated for the treatment of Friedreich's ataxia in adults and adolescents aged 16 years and older.
- Associated Conditions
- Friedreich's Ataxia
- Associated Therapies
- -
New Postmarketing Registry to Evaluate Real-World Safety and Patient Experience With Omaveloxolone
At the 2024 ICAR, the SKYCLARYS PASS registry (NCT06623890) was presented to collect long-term safety and patient experience data on omaveloxolone, an FDA-approved treatment for Friedreich ataxia. The registry will follow 300 omaveloxolone-naïve patients for up to 5 years, focusing on potential risks like drug-induced liver injury and congestive heart failure, with annual reports to the European Medicines Agency and FDA interim analyses. The study also explores health-related quality of life and healthcare resource utilization.
Biogen's Outlook Dims As Analyst Projects Flat Revenue Until 2026
Needham downgrades Biogen, projecting flat revenue until 2026 due to slow growth of Alzheimer’s drug Leqembi and high market expectations. Leqembi's Q3 sales were $67 million globally, $39 million in the U.S. CHMP recommended approval for early Alzheimer’s disease. Skyclarys Q4 sales expected at $116 million, $608 million in 2025. Revenues to remain flat or decline slightly until 2026. Zurzuvae sales reached $22 million in Q3. Needham downgrades stock from Buy to Hold.
Gastrointestinal Disorders and Aminotransferase Elevations Cited as Most Noted Adverse
The phase 3 MOXIe Part 2 trial analysis of omaveloxolone (Skyclarys) for Friedreich ataxia (FA) showed most treatment-emergent adverse events (TEAEs) occurred within the first 12 weeks, with gastrointestinal disorders and aminotransferase elevations (AST/ALT) being the most common. Elevated AST/ALT occurred in 29.4% of omaveloxolone-treated patients within the first 12 weeks and 9.8% after 12 weeks, compared to only 1 case in the placebo group. The median time for side effects to appear was shorter in the omaveloxolone group, with a median duration of elevated ALT and AST of 33 days. The study concluded that these findings guide patient treatment expectations and emphasize the importance of dosing compliance.
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Ethical Considerations of Friedreich's Ataxia Treatment
Kwa and Kendal examine ethical issues in treating Friedreich’s ataxia, including resource allocation, balancing benefits and risks of gene therapies, and informed consent. They highlight barriers to equitable access, such as high costs and strict eligibility criteria, emphasizing the need for transparent, accessible healthcare strategies in precision medicine.
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PTC to ask for FDA approval of vatiquinone for FA by year's end
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Drug Development Updates
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