SKYCLARYS

What it is

SKYCLARYS contains 50 mg omaveloxolone per capsule. The capsule is opaque size 0 hard hydroxypropyl methylcellulose (HPMC) with "RTA 408" printed on the light green body in white ink and "50" printed on the blue cap in white ink. This medicinal product is subject to additional monitoring in Australia.

Approved indications

— Treatment of Friedreich's ataxia in adults and adolescents aged 16 years and older.

Dosing overview

The recommended dose is 150 mg omaveloxolone (3 capsules of 50 mg each) taken orally once daily. Omaveloxolone should be taken on an empty stomach at least 1 hour before or 2 hours after eating. SKYCLARYS capsules should be swallowed whole, though for patients unable to swallow whole capsules, the capsules may be opened and the entire contents sprinkled onto 2 tablespoons of apple sauce, with the entire mixture consumed immediately on an empty stomach at least 1 hour before or 2 hours after eating. Omaveloxolone should be initiated and supervised by a specialist medical practitioner (usually a Neurologist) experienced in the management of patients with Friedreich's ataxia.

Key safety warnings

Treatment with omaveloxolone has been associated with elevations in alanine aminotransferase (ALT) and aspartate aminotransferase (AST), with on-treatment aminotransferase elevations of ≥3 times the upper limit of normal (ULN) reported in 29.4% of patients, with maximal values occurring in the majority of patients within the first 12 weeks of treatment. ALT, AST, and bilirubin should be monitored prior to initiation of omaveloxolone, monthly during the first 3 months of treatment, and periodically thereafter as clinically indicated. If abnormal ALT or AST levels are present at 3 months after commencing treatment, then monitoring of ALT, AST and bilirubin should continue monthly for an additional 3 months and then continue at a reduced frequency of 3 monthly thereafter if improvement is noted. If ALT or AST increases to greater than 5 times the ULN, omaveloxolone should be immediately discontinued and liver function tests should be repeated as soon as possible. If ALT or AST increases to greater than 3 times the ULN and bilirubin increases to greater than 2 times the ULN, omaveloxolone should be immediately discontinued and liver function tests should be repeated. Treatment with omaveloxolone has been associated with increases in low-density lipoprotein (LDL) cholesterol and decreases in high-density lipoprotein (HDL) cholesterol. Lipid parameters should be assessed prior to initiation of omaveloxolone and should be monitored periodically during treatment. Treatment with omaveloxolone has been associated with increases in B-type natriuretic peptide (BNP) but without any concurrent increase in blood pressure or associated events of fluid overload or congestive heart failure. In Study 1, a total of 13.7% of patients treated with SKYCLARYS had an increase from baseline in BNP and a BNP above the ULN (100 pg/mL), compared to 3.8% of patients who received placebo. The incidence of elevation of BNP above 200 pg/mL was 3.9% in patients treated with SKYCLARYS, compared to 0% of patients who received placebo. BNP should be monitored prior to and periodically during treatment. Patients should be advised of the signs and symptoms of congestive heart failure associated with fluid overload, such as sudden weight gain (≥1.4 kg in 1 day or ≥2.3 kg in 1 week), peripheral oedema, and shortness of breath. Treatment with SKYCLARYS has been associated with mild decreases in body weight. Patients should be advised to monitor their weight regularly, and should be further evaluated if unexplained or clinically significant body weight decrease occurs.

Contraindications

Hypersensitivity to the active substance or to any of the excipients is a contraindication.

PBS listing

No information about PBS listing is available in the provided source documents.

Regulatory history

SKYCLARYS omaveloxolone 50 mg capsule was first listed on the Australian Register of Therapeutic Goods (ARTG) on 26 June 2025. In March 2025, the Pharmaceutical Benefits Scheme Advisory Committee (PBAC) recommended against listing for Friedreich's ataxia in adults and adolescents aged 16 years and older.