What is SKYCLARYS prescribed for?
SKYCLARYS is prescribed for the treatment of Friedreich’s ataxia in adults and adolescents aged 16 years and older.
What is the name of the drug and what does it do?
SKYCLARYS is a medication for adults and teenagers aged 16 years and above who have Friedreich’s ataxia (FA).
FA is a rare genetic condition caused by complications in a gene called frataxin, which is crucial for cell function.
When this protein is lacking, it can cause problems like trouble with balance, speaking, walking, and can even lead to heart failure and death.
How does it work?
SKYCLARYS (generic name: omaveloxolone), pronounced “skye klar’ is,” works by targeting a specific gene called frataxin in the body. This gene is important for cell function.
When there’s a problem with this gene in people with Friedreich’s ataxia (FA), it can lead to issues like trouble with balance, speaking, walking, and even heart problems.
SKYCLARYS helps by addressing this gene problem, which can improve cell function and potentially lessen the symptoms of FA, like balance problems and heart issues.
What did the research discover?
The FDA approved SKYCLARYS after studying its effectiveness in a clinical trial (Study 1) involving 103 patients aged 16 to 40 with Friedreich’s ataxia (FA).
The trial took place at 11 sites in the United States, Western Europe, and Australia.
Safety data for SKYCLARYS came from two trials involving 165 FA patients, with 137 exposed for at least 48 weeks and 112 for at least 96 weeks.
In the 48-week trial, patients were randomly given SKYCLARYS or a placebo, with neither patients nor healthcare providers knowing who received what.
The study assessed the drug’s impact using the modified Friedreich’s Ataxia Rating Scale (mFARS) score, a tool used to assess the severity of symptoms and track changes in people with Friedreich’s Ataxia.
The study compared scores before and after treatment.
Patients treated with SKYCLARYS showed significantly lower mFARS scores compared to those on placebo at Week 48.
What are some of the side effects?
The side effects listed are from the product manufacturer. As health scientists, we share this information to keep you informed without causing undue worry. Many side effects diminish over time, and not all patients experience them. If prescribed medication, take it as directed and consult your healthcare professional if you have any concerning side effects.
- Elevated liver enzymes
- Increase in cholesterol
- Headache
- Nausea
- Abdominal pain
- Fatigue
- Diarrhea
- Musculoskeletal pain
- Maximum increases in liver enzymes occurred within 12 weeks after starting SKYCLARYS. Increases were generally symptom-free and reversible upon stopping the medication.
- Increases in cholesterol were seen within two weeks of starting SKYCLARYS and returned to normal within four weeks after stopping treatment.
What are the dosage recommendations and how should I take it?
Recommended dosage is 150 mg (3 capsules) taken orally once daily on an empty stomach at least one hour before eating. Swallow capsules whole.
Source:
Drug Trials Snapshots: SKYCLARYS [Internet]. U.S. Food and Drug Administration. 2023 [cited 2024 Jul 1]. Available from: https://www.fda.gov/drugs/development-approval-process-drugs/drug-trials-snapshots-skyclarys
