Less physical impairment over time with SKYCLARYS

SKYCLARYS was tested against patients taking placebo in a 48-week clinical trial that included 103 patients with FA. Results were measured using a tool called the modified Friedreich Ataxia Rating Scale (mFARS).

At the end of the trial, patients without pes cavus who received SKYCLARYS had lower mFARS scores compared with patients taking placebo. Lower mFARS scores in patients who received SKYCLARYS meant less physical impairment after 48 weeks of treatment.

Result From a 3-Year Exploratory Analysis

The open-label extension

At the end of the clinical trial, all patients who participated were given the option to continue in a longer-term study called an open-label extension (OLE). Before participating in the OLE, patients were asked to temporarily stop taking their treatment. Then, all patients were given SKYCLARYS, including those who had received a placebo in the clinical trial.

Comparing SKYCLARYS to natural progression

After 3 years, researchers conducted a follow-up study. They took the results from the OLE and compared them with untreated patients in a natural history study. This kind of study is called an “exploratory analysis” and is not the same as a clinical trial. Because of that, there are some limitations to the study and the results should be interpreted cautiously.

Lower mFARS scores

were observed in patients treated with SKYCLARYS after 3 years relative to a matched set of patients from a natural history study.
See data

mFARS Understanding mFARS

The mFARS neurological exam is one of the available tests that can help your doctor determine how your FA is progressing.

FA is a progressive disease. One way to measure progression is with the mFARS neurological exam. The mFARS is commonly used in clinical trials to evaluate how well potential treatments work. The clinical trial for SKYCLARYS looked at changes in mFARS scores after 48 weeks. A lower mFARS score means less physical impairment, while a higher score indicates poorer functional ability.

The average rate of progression is:
+2 points per year
The mFARS score range is:
0-93 points
The higher the score, the worse the disease.
Average score for wheelchair use:
~65 points

It is important to understand that the rate of FA progression will vary from person to person and that mFARS scores will change on an individual basis. For example, patients who are diagnosed at a younger age typically see their mFARS scores rise more quickly than older patients.

The mFARS focuses on 4 key areas that relate to activities of daily living:

Mouth and throat function
Lower limb coordination
Upper limb coordination
Upright stability
SKYCLARYS is a once-daily oral treatment for FA. Here’s what you need to know before getting started
  • 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.