Omaveloxolone

The precise mechanism by which omaveloxolone exerts its therapeutic effect in patients with Friedreich's ataxia is unknown. Omaveloxolone has been shown to activate the Nuclear factor (erythroid-derived 2)-like 2 (Nrf2) pathway in vitro and in vivo in animals and humans.

• SKYCLARYS is indicated for the treatment of Friedreich's ataxia in adults and adolescents aged 16 years and older. SKYCLARYS is indicated for the treatment of Friedreich's ataxia in adults and adolescents aged 16 years and older.

• None. None.contraindicated

Obtain alanine aminotransferase (ALT), aspartate aminotransferase (AST), bilirubin, B-type natriuretic peptide (BNP), and lipid parameters prior to initiating SKYCLARYS and during treatment. ( 2.1 , 5.1 , 5.2 , 5.3 ) Recommended dosage is 150 mg (3 capsules) taken orally once daily. ( 2.2 ) Administer SKYCLARYS on an empty stomach, at least 1 hour before or 2 hours after eating. ( 2.2 ) Swallow SKYCLARYS capsules whole or open and sprinkle the entire contents of both halves of the capsule onto applesauce and mix. Do not crush or chew capsules. ( 2.2 ) Moderate and Severe Hepatic Impairment: The recommended dosage of SKYCLARYS is 100 mg once daily for patients with moderate hepatic impairment. If adverse reactions emerge, further reduce the dosage to 50 mg once daily. Avoid use in patients with severe hepatic impairment. ( 2.5 , 8.6 , 12.3 ) 2.1 Recommended Testing Before Initiating SKYCLARYS and Monitoring to Assess Safety Obtain ALT, AST, bilirubin, BNP, and lipid parameters prior to initiating SKYCLARYS and during treatment [see Warnings and Precautions ( 5.1 , 5.2 , 5.3 )] . 2.2 Recommended Dosage The recommended dosage of SKYCLARYS is 150 mg (3 capsules) taken orally once daily. Administer SKYCLARYS on an empty stomach, at least 1 hour before or 2 hours after eating [see Clinical Pharmacology ( 12.3 )]. Swallow SKYCLARYS capsules whole. Do not crush or chew.

Elevation of Aminotransferases: Monitor ALT, AST, and total bilirubin prior to initiation, every month for the first 3 months of treatment, and periodically thereafter. ( 2.1 , 5.1 ) Elevation of B-type Natriuretic Peptide (BNP): Advise patients of signs and symptoms of fluid overload. ( 2.1 , 5.2 ) Lipid Abnormalities: Monitor cholesterol periodically during treatment. ( 2.1 , 5.3 ) 5.1 Elevation of Aminotransferases Treatment with SKYCLARYS can cause an elevation in hepatic transaminases (ALT and AST). In Study 1 [see Clinical Studies ( 14 )] , the incidence of elevations of ALT or AST above 5 times and 3 times the upper limit of normal (ULN) was 16% and 31%, respectively, in patients treated with SKYCLARYS. There were no cases of concomitant elevation of transaminases and total bilirubin observed in Study 1. Maximum increases in ALT and AST occurred within 12 weeks after starting SKYCLARYS. Increases in serum aminotransferases were generally asymptomatic and reversible following discontinuation of SKYCLARYS. Patients with clinically significant liver disease were excluded from Study 1. Monitor ALT, AST, and total bilirubin prior to initiation of SKYCLARYS, every month for the first 3 months of treatment, and periodically thereafter. If transaminases increase to levels greater than 5 times the ULN, or greater than 3 times the ULN with evidence of liver dysfunction (e.g., elevated bilirubin), immediately discontinue SKYCLARYS and repeat liver function tests as soon as possible.

The following clinically significant adverse reactions are described in greater detail in other labeling sections: Elevation of aminotransferases [see Warnings and Precautions ( 5.1 )] Elevation of BNP [see Warnings and Precautions ( 5.2 )] Lipid abnormalities [see Warnings and Precautions ( 5.3 )] Most common adverse reactions (incidence ≥20% and greater than placebo) are elevated liver enzymes (AST/ALT), headache, nausea, abdominal pain, fatigue, diarrhea, and musculoskeletal pain. ( 6.1 ) To report SUSPECTED ADVERSE REACTIONS, contact Biogen at 1-844-987-3224- or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch. 6.1 Clinical Trials Experience Because clinical trials are conducted under widely varying conditions, adverse reaction rates observed in the clinical trials of a drug cannot be directly compared with rates in clinical trials of another drug and may not reflect the rates observed in clinical practice. The safety of SKYCLARYS 150 mg once daily has been evaluated in 165 patients with Friedreich's ataxia, including 137 patients exposed for at least 48 weeks, and 125 patients exposed for at least 96 weeks. The most common adverse reactions in Study 1 (≥20% and greater than placebo) were elevated liver enzymes (AST/ALT), headache, nausea, abdominal pain, fatigue, diarrhea, and musculoskeletal pain. Table 3 shows the adverse reactions that occurred in 10% or more of patients treated with SKYCLARYS and greater than placebo.

Pregnancy: Based on animal data, may cause fetal harm. ( 8.1 ) 8.1 Pregnancy Pregnancy Exposure Registry There is a pregnancy exposure registry that monitors pregnancy outcomes in women exposed to SKYCLARYS during pregnancy. Healthcare providers are encouraged to enroll pregnant patients, or pregnant women may register themselves in the program by calling 1-866-609-1785 or by sending an email to SkyclarysPregnancySurveillance@ppd.com. Risk Summary There are no adequate data on the developmental risks associated with the use of SKYCLARYS in pregnant women. In animal studies, administration of omaveloxolone during pregnancy or throughout pregnancy and lactation produced evidence of developmental toxicity (embryofetal mortality and growth impairment, and mortality, growth impairment, and neurobehavioral deficits in offspring) at plasma exposures similar to or less than exposures in humans (see Animal Data ). In the U.S. general population, the estimated background risk of major birth defects and miscarriage in clinically recognized pregnancies is 2% to 4% and 15% to 20%, respectively. The background risk of major birth defects and miscarriage for the indicated population is unknown.

Metabolism

Absorption The median (range) time to achieve peak plasma concentration (T max ) was 7 to 14 (1 to 24) hours. The total plasma omaveloxolone exposure based on area under the concentration-time curve (AUC) increased in a dose-dependent and dose proportional manner over a dose range of 50 mg (0.33 times the recommended dosage) to 150 mg, but maximum omaveloxolone plasma concentration (C max ) increased in a less than dose proportional manner over the dose range in healthy fasted subjects.

• Feb 28, 2023NDANDA216718Biogen Us

1. 1Fatigue1338.6%
2. 2Hepatic Enzyme Increased1207.8%
3. 3Nausea1187.7%
4. 4Product Dose Omission Issue1006.5%
5. 5Headache926.0%
6. 6Diarrhoea855.5%
7. 7Blood Cholesterol Increased744.8%
8. 8Muscle Spasms714.6%
9. 9Fall603.9%
10. 10Urinary Tract Infection603.9%
11. 11Abdominal Pain Upper563.6%
12. 12Alanine Aminotransferase Increased543.5%
13. 13Atrial Fibrillation533.4%
14. 14Vomiting533.4%
15. 15Death473.1%

The 10 most recently updated of 25 ClinicalTrials.gov registrations naming Omaveloxolone as an intervention. Registration is not evidence of efficacy or safety — reference crosswalk only.

• A Study to Learn More About the Effects and Long-Term Safety of Omaveloxolone (BIIB141) in Children and Teens With Friedreich's Ataxia
  Recruiting · Phase 3 · Interventional · 255 enrolled · Biogen
  NCT06953583updated 2026-06-03
• A Study to Learn How the Body Processes BIIB141 (Omaveloxolone) When Taken as a Capsule or as a Tablet Dissolved in Liquid, and to Learn About Its Safety in Healthy Adults Ages 18 to 55
  Completed · Phase 1 · Interventional · 70 enrolled · Biogen
  NCT07297199updated 2026-05-14
• 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
  Active not recruiting · Phase 1 · Interventional · 33 enrolled · Biogen
  NCT06054893updated 2026-05-11
• Efficacy of Omaveloxolone Treatment for Dysphagia in French Patients With Friedreich's Ataxia
  Recruiting · Observational · 40 enrolled · Centre Hospitalier Universitaire de Nice
  NCT07013292updated 2026-04-29
• 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
  Recruiting · Observational · 300 enrolled · Biogen
  NCT06623890updated 2026-04-28
• Cardiac Output and Fatigue in Friedreich's Ataxia
  Not yet recruiting · Interventional · 30 enrolled · Scott Barbuto
  NCT07444333updated 2026-03-02
• A Study to Learn About the Effects and Safety of RTA 408 (Omaveloxolone) in People Aged 16 to 40 With Friedreich's Ataxia
  Completed · Phase 2 · Interventional · 172 enrolled · Biogen
  NCT02255435updated 2026-01-23
• A Study to Learn if Taking BIIB141 (Omaveloxolone) Affects How Omeprazole is Processed in the Body and About BIIB141's Safety in Healthy Adults Aged 18 to 55
  Completed · Phase 1 · Interventional · 22 enrolled · Biogen
  NCT07149415updated 2025-10-23
• A Study to Learn How BIIB141 (Omaveloxolone) is Processed in the Body When Taken as Capsules Compared to Sprinkled on Yogurt in Healthy Adults Aged 18 to 55
  Completed · Phase 1 · Interventional · 52 enrolled · Biogen
  NCT07019064updated 2025-09-22
• 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
  Recruiting · Observational · 20 enrolled · Biogen
  NCT06628687updated 2025-06-26

Frequently asked questions

How does Omaveloxolone work?

The precise mechanism by which omaveloxolone exerts its therapeutic effect in patients with Friedreich's ataxia is unknown. Omaveloxolone has been shown to activate the Nuclear factor (erythroid-derived 2)-like 2 (Nrf2) pathway in vitro and in vivo in animals and humans.

What is Omaveloxolone used for?

According to FDA labeling, Omaveloxolone carries indications including: SKYCLARYS is indicated for the treatment of Friedreich's ataxia in adults and adolescents aged 16 years and older. SKYCLARYS is indicated for the treatment of Friedreich's ataxia in adults and adolescents aged 16 years and older.. This is a reference summary of labeled uses, not medical advice or a treatment recommendation.

What class of drug is Omaveloxolone?

Omaveloxolone is classified as Other nervous system drugs, Cytochrome P450 2C8 Inducers, Cytochrome P450 3A4 Inducers.

What are the brand names for Omaveloxolone?

Omaveloxolone is marketed under brand names including Skyclarys.

What are the contraindications for Omaveloxolone?

Omaveloxolone labeling lists contraindications including: None. None.. Always consult the full prescribing information and a clinician.