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
A Study to Learn More About the Effects and Long-Term Safety of BIIB141 (Omaveloxolone) in Participants With Friedreich's Ataxia Aged 2 to 15 Years Old
- First Posted Date
- 2025-05-01
- Last Posted Date
- 2025-05-01
- Lead Sponsor
- Biogen
- Target Recruit Count
- 255
- Registration Number
- NCT06953583
- Locations
- 🇺🇸
UCLA Neurology Outpatient Clinic at Westwood, Los Angeles, California, United States
🇺🇸Norman Fixel Institute for Neurological Diseases UF Health, Gainesville, Florida, United States
🇺🇸USF Health Morsani College of Medicine Department of Neurology, Tampa, Florida, United States
A Study to Learn How BIIB141 (Omaveloxolone) Affects the Health of Participants With Friedrich's Ataxia Who Took it During Pregnancy and/or During Breastfeeding and About the Health of Their Babies
- First Posted Date
- 2024-10-08
- Last Posted Date
- 2025-02-18
- Lead Sponsor
- Biogen
- Target Recruit Count
- 20
- Registration Number
- NCT06628687
- Locations
- 🇺🇸
Evidera, Morrisville, North Carolina, United States
A Study to Learn More About the Long-Term Safety of BIIB141 (Omaveloxolone) in Participants With Friedreich's Ataxia Who Are Prescribed it by Their Own Doctors
- First Posted Date
- 2024-10-02
- Last Posted Date
- 2025-04-10
- Lead Sponsor
- Biogen
- Target Recruit Count
- 300
- Registration Number
- NCT06623890
- Locations
- 🇺🇸
UCLA Neurology, Los Angeles, California, United States
🇺🇸University of Colorado, Aurora, Colorado, United States
🇺🇸University of Florida, Gainesville, Florida, United States
A Study to Find Out How BIIB141 (Omaveloxolone) Moves From the Blood Into the Breastmilk of Healthy Women Who Are Breastfeeding or Pumping Milk
- First Posted Date
- 2024-09-25
- Last Posted Date
- 2025-04-22
- Lead Sponsor
- Biogen
- Target Recruit Count
- 12
- Registration Number
- NCT06612879
- Locations
- 🇺🇸
Fortrea Madison WI, CRU, Madison, Wisconsin, United States
A Study to Find Out How BIIB141 (Omaveloxolone) is Processed in the Body and to Learn More About Its Safety in Participants With Friedreich's Ataxia Aged 2 to 15 Years Old
- First Posted Date
- 2023-09-26
- Last Posted Date
- 2025-05-21
- Lead Sponsor
- Biogen
- Target Recruit Count
- 35
- Registration Number
- NCT06054893
- Locations
- 🇺🇸
Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, United States
A TQTc Study for Omaveloxolone
- First Posted Date
- 2023-07-03
- Last Posted Date
- 2024-02-02
- Lead Sponsor
- Reata, a wholly owned subsidiary of Biogen
- Target Recruit Count
- 30
- Registration Number
- NCT05927649
- Locations
- 🇺🇸
Celerion, Inc., Tempe, Arizona, United States
An Open-label DDI Study of Omaveloxolone in Healthy Subjects
- First Posted Date
- 2023-06-18
- Last Posted Date
- 2024-02-02
- Lead Sponsor
- Reata, a wholly owned subsidiary of Biogen
- Target Recruit Count
- 20
- Registration Number
- NCT05909644
- Locations
- 🇺🇸
Celerion, Inc., Tempe, Arizona, United States
A Clinical Drug-Drug Interaction (DDI) Study With Omaveloxolone
- Conditions
- Healthy Adult Subjects
- Interventions
- First Posted Date
- 2019-07-05
- Last Posted Date
- 2024-02-02
- Lead Sponsor
- Reata, a wholly owned subsidiary of Biogen
- Target Recruit Count
- 61
- Registration Number
- NCT04008186
- Locations
- 🇺🇸
Covance Clinical Research Unit (CRU) Inc., Dallas, Texas, United States
A Pharmacokinetic Study of Omaveloxolone in Subjects With Hepatic Impairment and Normal Hepatic Function
- First Posted Date
- 2019-04-03
- Last Posted Date
- 2024-02-02
- Lead Sponsor
- Reata, a wholly owned subsidiary of Biogen
- Target Recruit Count
- 48
- Registration Number
- NCT03902002
- Locations
- 🇺🇸
Orange County Research Center, Tustin, California, United States
🇺🇸Clinical Pharmacology of Miami, LLC, Miami, Florida, United States
🇺🇸Orlando Clinical Research Center, Orlando, Florida, United States
A Pharmacokinetic Study of Omaveloxolone in Healthy Volunteers
- First Posted Date
- 2018-09-10
- Last Posted Date
- 2024-02-02
- Lead Sponsor
- Reata, a wholly owned subsidiary of Biogen
- Target Recruit Count
- 32
- Registration Number
- NCT03664453
- Locations
- 🇺🇸
Medpace Clinical Pharmacology Unit, Cincinnati, Ohio, United States
Drug Development Updates
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