Brand Name
Skyclarys
Generic Name
Omaveloxolone
View Brand Information FDA approval date: February 28, 2023
Form: Capsule
What is Skyclarys (Omaveloxolone)?
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.
Approved To Treat
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An Open-Label, Phase 1, Study to Evaluate the Pharmacokinetics, Safety, Tolerability, Pharmacodynamics, and Efficacy of Omaveloxolone in Participants ≥2 to <16 Years of Age With Friedreich's Ataxia
Summary: In this study, researchers will learn more about BIIB141, also known as omaveloxolone or SKYCLARYS®. This drug has been approved, or made available for doctors to prescribe, for people with Friedrich's Ataxia (FA) who are at least 16 years old. But, it is not yet available for children and teens with FA who are younger than 16 years old. The main objective of this study is to learn how BIIB141 is ...
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Summary: In this study, researchers will learn more about BIIB141, also known as omaveloxolone or SKYCLARYS®. This drug has been approved, or made available for doctors to prescribe, for people with Friedrich's Ataxia (FA) who are at least 16 years old. This is called a drug-drug interaction study. In this kind of study, researchers are looking at what happens when 2 or more medicines affect each other in ...
A Phase 3, 2-Part, Randomized, Double-Blind, Placebo-Controlled Study (Part 1) and Open-Label Extension (Part 2) to Evaluate the Efficacy, Safety, Pharmacokinetics, and Pharmacodynamics of Omaveloxolone (BIIB141) in Participants With Friedreich's Ataxia Aged 2 to < 16 Years
Summary: In this study, researchers will learn more about the effects and safety of BIIB141, also known as omaveloxolone or SKYCLARYS®. This drug has been approved, or made available for doctors to prescribe, for people with Friedreich's Ataxia (FA) who are at least 16 years old. But, it is not yet available for children and teens with FA who are younger than 16 years old. The main objective of this study ...
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Brand Information
SKYCLARYS (omaveloxolone)
1INDICATIONS AND USAGE
SKYCLARYS is indicated for the treatment of Friedreich's ataxia in adults and adolescents aged 16 years and older.
2DOSAGE FORMS AND STRENGTHS
SKYCLARYS capsules contain 50 mg of omaveloxolone, and are supplied as opaque hard capsules having a light green body and blue cap, imprinted with “RTA 408” in white ink on the body and “50” in white ink on the cap.
3CONTRAINDICATIONS
None.
4ADVERSE REACTIONS
The following clinically significant adverse reactions are described in greater detail in other labeling sections:
- Elevation of aminotransferases
- Elevation of BNP
- Lipid abnormalities
4.1Clinical 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.
4.2Postmarketing Experience
The following adverse reactions have been identified during post approval use of SKYCLARYS. Because these reactions are reported voluntarily from a population of uncertain size, it is not always possible to reliably estimate their frequency or establish a causal relationship to drug exposure.
Immune System Disorders: hypersensitivity (urticaria, rash)
5DESCRIPTION
SKYCLARYS contains omaveloxolone in immediate release capsules for oral administration available in a 50 mg strength. The chemical name of omaveloxolone is N-(2-cyano-3,12-dioxo-28-noroleana-1,9(11)-dien-17-yl)-2,2-difluoro-propanamide. Omaveloxolone is a white to off-white amorphous solid. The molecular formula is C
Omaveloxolone has a pKa of 7.26 and is practically insoluble in water across the physiological pH range. Capsule contents include the following inactive ingredients: croscarmellose sodium, magnesium stearate, pregelatinized starch, and silicified microcrystalline cellulose. The hard capsule shells contain FD&C Blue #1, ferric oxide yellow, hypromellose, titanium dioxide, and white ink.
6CLINICAL STUDIES
The efficacy of SKYCLARYS was evaluated in a 48-week, randomized, double-blind, placebo-controlled study in patients 16 to 40 years of age with Friedreich's Ataxia (Study 1; NCT02255435).
A total of 103 patients were randomized (1:1) to receive SKYCLARYS 150 mg once daily (n=51) or placebo (n=52).
Enrolled patients had to have a stable modified Friedreich's Ataxia Rating Scale (mFARS) score between 20 and 80, be able to complete maximal exercise testing, and have a left ventricular ejection fraction of at least 40%. In Study 1, 53% of enrolled patients were male, 97% were White, and the mean age was 24 years at study entry. Patients with or without pes cavus were included in Study 1. Pes cavus was defined as having a loss of lateral support and was determined if light from a flashlight could be seen under the patient's arch when barefoot and weight bearing.
The prespecified primary analysis was the change from baseline in the mFARS score compared to placebo at Week 48 in the Full Analysis Population of patients without pes cavus (n=82). The mFARS is a clinical assessment tool to assess patient function, which consists of 4 domains to evaluate bulbar function, upper limb coordination, lower limb coordination, and upright stability. The mFARS has a maximum score of 99, with a lower score on the mFARS signifying lesser physical impairment.
Treatment with SKYCLARYS resulted in statistically significant lower mFARS scores (less impairment) relative to placebo (see
The All Randomized Population (N=103), which included all patients regardless of pes cavus status, demonstrated similar results to the Full Analysis Population of lower mFARS scores in patients treated with SKYCLARYS compared to placebo, with a nominally significant least squares mean difference between treatment groups of ‑1.94 (95% CI: -3.71, -0.16, p=0.0331).
In a post hoc, propensity-matched analysis, lower mFARS scores were observed in patients treated with SKYCLARYS after 3 years relative to a matched set of untreated patients from a natural history study. These exploratory analyses should be interpreted cautiously given the limitations of data collected outside of a controlled study, which may be subject to confounding.
7PATIENT COUNSELING INFORMATION
Advise the patient to read the FDA-approved patient labeling (Patient Information).
8PRINCIPAL DISPLAY PANEL
73179-250-90
