Omaveloxolone

What it is and how it works

Mechanism of Action 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. The Nrf2 pathway is involved in the cellular response to oxidative stress.

Mechanism class: Cytochrome P450 3A4 Inducers · Cytochrome P450 2C8 Inducers

Indications

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.

Dosage and administration (summary)

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. Administer SKYCLARYS on an empty stomach, at least 1 hour before or 2 hours after eating. 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. Moderate and Severe Hepatic Impairment: The recommended dosage of SKYCLARYS is 100 mg once daily for patients with moderate hepatic impairment.

Contraindications

None. None.

Warnings and precautions

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 ) Elevation of Aminotransferases Treatment with SKYCLARYS can cause an elevation in hepatic transaminases (ALT and AST).

Pregnancy

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 SkyclarysPregnancy…

Storage and supply

Storage and Handling Store at 20°C to 25°C (68°F to 77°F), excursions permitted between 15°C and 30°C (59°F to 86°F) .

Source: Omaveloxolone (Skyclarys) — US FDA prescribing information, 2026-04-06, DailyMed. Summarised and shortened; the full label governs. Iranian registration, indications and labelling may differ.