ATTENTION

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MOXIe clinical trial design
MOXIe trial results
3-year post hoc analysis results
4-year open-label extension results

At 4 years, SKYCLARYS showed continued treatment effect1

mFARS scores for SKYCLARYS-treated patients increased by 4.40 points on average
over ~4 years (average of +1.06 per year) in the MOXIe OLE1

Mean change in mFARS score over 4 years1

Data from the open-label extension study are supportive and should be interpreted cautiously in the context of its open-label, non-controlled study design.

mFARS=modified Friedreich Ataxia Rating Scale; OLE=open-label extension; SE=standard error.

Consider the natural history of Friedreich ataxia (FA) progression
A large longitudinal study of untreated patients found that FA progresses on average ~2 points per year on the mFARS2

mFARS component subscores: mean change over 4 years

The mFARS is made up of 4 components that assess functional abilities. At 4 years,
consistent bulbar function and upper limb coordination were observed, while lower
limb coordination and upright stability function progressed.1

CI=confidence interval.

Review the warnings and precautions

for SKYCLARYS

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INDICATION

  • SKYCLARYS® (omaveloxolone) is indicated for the treatment of Friedreich ataxia in adults and adolescents aged 16 years and older

 

Important Safety Information

 

WARNINGS AND PRECAUTIONS

Elevation of Aminotransferases

  • Treatment with SKYCLARYS can cause an elevation in hepatic transaminases (alanine aminotransferase [ALT] and aspartate aminotransferase [AST]). 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. 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
  • 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. If transaminase levels stabilize or resolve, SKYCLARYS may be reinitiated with an appropriate increased frequency of monitoring of liver function
 

Elevation of B-Type Natriuretic Peptide

  • Treatment with SKYCLARYS can cause an increase in B-type natriuretic peptide (BNP), a marker of cardiac function. A total of 14% of patients treated with SKYCLARYS had an increase from baseline in BNP value above the ULN (100 pg/mL), compared to 4% of patients who received placebo. The incidence of elevation of BNP above 200 pg/mL was 4% in patients treated with SKYCLARYS. Cardiomyopathy and cardiac failure are common in patients with Friedreich ataxia. Whether the elevations in BNP are related to SKYCLARYS or cardiac disease associated with Friedreich ataxia is unclear
  • Elevations in BNP may indicate cardiac failure and should prompt an evaluation of cardiac function. Check BNP prior to initiation of SKYCLARYS. Monitor patients for the signs and symptoms of fluid overload, such as sudden weight gain (3 pounds or more of weight gain in one day, or 5 pounds or more of weight gain in a week), peripheral edema, palpitations, and shortness of breath. If signs and symptoms of fluid overload develop, worsen, or require hospitalization, evaluate BNP and cardiac function, and manage appropriately. Management of fluid overload and heart failure may require discontinuation of SKYCLARYS
 

Lipid Abnormalities

  • Treatment with SKYCLARYS can cause changes in cholesterol. In Study 1, 29% of patients treated with SKYCLARYS reported elevated cholesterol above ULN at one or more time points. Mean increases were observed within 2 weeks of initiation of SKYCLARYS and returned to baseline within 4 weeks of discontinuing treatment. A total of 16% of patients treated with SKYCLARYS had an increase in low-density lipoprotein cholesterol (LDL-C) from baseline, compared to 8% of patients who received placebo. The mean increase in LDL-C for all SKYCLARYS-treated patients was 23.5 mg/dL at 48 weeks. A total of 6% of patients treated with SKYCLARYS had decreases in high-density lipoprotein cholesterol (HDL-C) from baseline compared to 4% of patients who received placebo. The mean decrease in HDL-C for all SKYCLARYS-treated patients was 5.3 mg/dL at 48 weeks
  • Assess lipid parameters prior to initiation of SKYCLARYS and monitor periodically during treatment. Manage lipid abnormalities according to clinical guidelines
 

ADVERSE REACTIONS

  • 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
 

DRUG INTERACTIONS

  • Avoid concomitant use of SKYCLARYS with moderate or strong CYP3A4 inhibitors. If use cannot be avoided, dosage modifications are recommended
  • Avoid concomitant use of SKYCLARYS with moderate or strong CYP3A4 inducers
  • Refer to the prescribing information for dosing instructions for concomitant use of CYP3A4 and CYP2C8 substrates and monitor for lack of efficacy of the concomitant treatment
  • Advise patients to avoid concomitant use with combined hormonal contraceptives, implants, and progestin only pills
 

SPECIFIC POPULATIONS
Pregnancy

  • 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
  • There are no adequate data on the development risks associated with the use of SKYCLARYS in pregnant women
 

Lactation

  • There are no data on the presence of omaveloxolone or its metabolites in human milk. The effects on milk production and the breastfed infant are unknown. The developmental and health benefits of breastfeeding should be considered along with the mother’s clinical need for SKYCLARYS and any potential adverse effects on the breastfed infant from SKYCLARYS or from the underlying maternal condition
 

Hepatic Impairment

  • Avoid treatment with SKYCLARYS in patients with severe hepatic impairment, including those who develop severe hepatic impairment
  • Reduced dosage in patients with moderate hepatic impairment with close monitoring for adverse reactions is recommended
 

Please see full Prescribing Information.

References

1. Zesiewicz T, Lynch DR, Delatycki MB, et al. Long-term efficacy and safety of omaveloxolone in patients with Friedreich ataxia: 4-year data from the ongoing MOXIe open-label extension. Presented at: International Congress of Parkinson’s Disease and Movement Disorders (MDS); October 5-9, 2025; Honolulu, HI. 2. Patel M, Isaacs CJ, Seyer L, et al. Progression of Friedreich ataxia: quantitative characterization over 5 years. Ann Clin Transl Neurol. 2016;3(9):684-694. doi:10.1002/acn3.332

Intended for a US HCP audience.
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