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, as they may not be as reliable as a clinical trial.
Lower mFARS scores
See data
were observed in patients treated with SKYCLARYS after 3 years relative to a matched set of patients from a natural history study.
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
