SKYCLARYS is used for the treatment of Friedreich ataxia (FA) in adults and children aged 16 years and older

Talk to your doctor to see if SKYCLARYS is right for you. It is not known if SKYCLARYS is safe and effective for use in children younger than 16 years of age

SKYCLARYS is the first and only FDA-approved prescription medicine to treat FA for patients 16 years and older

Understanding FA

What is FA?

FA is a rare, genetic, progressive disease that damages the nervous system, causing you to lose control of your muscles over time.

What causes FA?

FA happens when a specific gene, called the frataxin gene (or FXN), does not function properly and cannot help nerve cells work the way they should. Ultimately, this leads to irreversible cell death.

How is FA diagnosed?

FA can be confirmed with a genetic test. It is important that the test includes a Friedreich Ataxia Repeat Expansion Analysis. Your doctor can provide additional information about the specific test needed.

What happens as FA progresses?

As you get older, FA continues to damage nerve cells, which leads to greater difficulty with things like:

  • Standing
  • Walking
  • Talking
  • Fine motor skills

How can FA progression be measured?


The severity of FA can be measured in different ways—one way is the modified Friedreich Ataxia Rating Scale (mFARS).


Most people with FA will need to use a wheelchair around 10 to 15 years after the onset of symptoms. In addition to difficulty moving and talking, FA may cause a serious heart condition called cardiomyopathy. With cardiomyopathy, the muscles in the heart become damaged, making it harder for the heart to pump blood.

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  • SKYCLARYS is used for the treatment of Friedreich ataxia in adults and children aged 16 years and older. It is not known if SKYCLARYS is safe and effective for use in children younger than 16 years of age

What are the possible side effects of SKYCLARYS?

SKYCLARYS may cause serious side effects, including:

  • Increase in blood liver enzymes: Some people taking SKYCLARYS have had an increase in the level of liver enzymes in their blood. Your healthcare provider will do liver function tests
    • before you start taking SKYCLARYS
    • every month for the first 3 months after starting your treatment with SKYCLARYS
    • during certain times as needed while taking SKYCLARYS

    If your liver enzymes increase, your healthcare provider may change your dose, stop treatment for some time, or completely stop treatment with SKYCLARYS.

  • Increase in a blood protein called B-Type Natriuretic Peptide (BNP). BNP tells how well your heart is working. Your healthcare provider will check your BNP levels before your treatment with SKYCLARYS. Tell your healthcare provider if you have signs and symptoms of your heart not working well such as too much fluid in your body (fluid overload). Signs and symptoms may include:
    • sudden weight gain (3 pounds or more of weight gain in 1 day, or 5 pounds or more of weight gain in 1 week)
    • swelling in your arms, hands, legs, or feet (peripheral edema)
    • fast heartbeat (palpitations)
    • shortness of breath

    If you have symptoms of fluid overload that is considered a side effect of SKYCLARYS, your healthcare provider may stop treatment with SKYCLARYS.

  • Changes in cholesterol levels. Increases in low density lipoprotein cholesterol (LDL-C) or bad cholesterol and decreases in high density lipoprotein cholesterol (HDL-C) or good cholesterol have happened during treatment with SKYCLARYS. Your healthcare provider will check your cholesterol levels before and during your treatment with SKYCLARYS

The most common side effects of SKYCLARYS include: increased liver enzymes (ALT/AST), headache, nausea, stomach pain, tiredness, diarrhea, and muscle pain.

Before taking SKYCLARYS, tell your healthcare provider about all of your medical conditions, including if you:

  • have liver problems
  • have a history of heart problems, including heart failure
  • have a high level of fat in your blood (high blood cholesterol)
  • are pregnant or plan to become pregnant. It is not known if SKYCLARYS will harm your unborn baby. Women who use hormonal birth control should use another form of birth control such as a non-hormonal intrauterine system or an extra non-hormonal birth control such as condoms while using SKYCLARYS and for 28 days after stopping SKYCLARYS
  • are breastfeeding or plan to breastfeed. It is not known if SKYCLARYS passes into your breast milk. Talk to your healthcare provider about the best way to feed your baby if you take SKYCLARYS

Tell your healthcare provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements such as St. John’s Wort.

  • Taking SKYCLARYS with other medicines can cause serious side effects
  • SKYCLARYS may affect the way other medicines work, and other medicines may affect how SKYCLARYS works
  • Know the medicines you take. Keep a list of them to show to your healthcare provider and pharmacist when you get a new medicine

What should I avoid while taking SKYCLARYS?

  • Do not drink grapefruit juice or eat grapefruit. These may change the amount of SKYCLARYS in your blood

These are not all the possible side effects of SKYCLARYS. For more information, ask your healthcare provider or pharmacist.

Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088.

Please see full Prescribing Information, including Patient Information.