Slowing Friedreich
Not an actual patient.
Less physical impairment over time with SKYCLARYS
SKYCLARYS was tested against untreated patients 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 who received SKYCLARYS showed an improvement in their mFARS scores compared with untreated patients. Lower mFARS scores in patients who received SKYCLARYS meant less physical impairment over time.
Patients who took SKYCLARYS showed improvement in mFARS scores over those who went untreated regardless of age, sex, or ability to walk*
Patients on SKYCLARYS improved in all 4 physical criteria* evaluated with mFARS, such as:
-
Upright
stability
-
Lower limb coordination
-
Mouth and throat function
-
Upper limb coordination
A -4.21–point difference was observed at week 48 in patients 16–18
years of age who received SKYCLARYS versus untreated patients.†
There is limited data available for non-ambulatory patients in the MOXIe trial, and the impact of SKYCLARYS on this patient population has not been demonstrated.
†The MOXIe adolescent population, n=20; P=0.057.
The long-term results from SKYCLARYS clinical trials
After the 48-week trial, 98% of patients joined the long-term clinical trial.
After 3 years, researchers conducted a follow-up study. They took the results from the long-term trial 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.
The natural history study is an observational study and is being conducted in a wide range of patients that may not be representative of the patients studied in the SKYCLARYS clinical trials.
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.
See datamFARS Understanding mFARS
The mFARS neurological exam is one of the available tests that can help your doctor determine how your FA is progressing.
mFARS is commonly used in clinical trials to evaluate how well potential treatments work. A higher score means more severe symptoms.
The mFARS score range is
0-93points:
The higher the score, the worse the disease.
The average rate of progression observed in a natural history study is
~2 points per year.
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.
It 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 prescription medicine. Here’s what you need to know before getting started
What is SKYCLARYS?
SKYCLARYS is used for the treatment of Friedreich ataxia in adults and children 16 years of age and older.
Before taking SKYCLARYS, tell your healthcare provider about all 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. Some medications interact with SKYCLARYS. Keep a list of your medicines to show to your healthcare provider and pharmacist when you get a new medicine.
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 during treatment, 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
- muscle pain
These are not all the possible side effects of SKYCLARYS. Call your doctor for medical advice about side effects.
You are encouraged to report negative side effects of prescription drugs to the FDA.
Visit www.fda.gov/medwatch or call 1-800-FDA-1088. You may also report side effects to Reata Pharmaceuticals, Inc., at 1-800-314-3934.
For additional information about SKYCLARYS, please see the full Prescribing Information and Patient Product Information.
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