European medicines panel recommends marketing of Skyclarys for Friedreich’s ataxia
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A committee of the European Medicines Agency has recommended marketing authorization for Skyclarys to treat Friedreich’s ataxia in patients aged 16 years and older.
According to a release from Biogen Inc., if approved by the European Commission, Skyclarys (omavexolone) will be the first treatment authorized within the European Union to address the rare, genetic, progressive neuromuscular disease.
In the U.S., the FDA approved Skyclarys in February for patients with Friedreich’s aged 16 years and older.
The EMA committee’s recommendation for the oral, once-daily therapeutic was based on safety and efficacy data from the placebo-controlled MOXIe part 2 trial. By week 48, participants administered Skyclarys demonstrated less physical impairment compared with those given placebo, as measured by the modified Friedreich Ataxia Rating Scale (mFARS).
Improvements measured by mFARS subscales for those treated with the drug included better upright stability, lower limb coordination, ability to swallow as well as upper limb coordination.
In addition, data from a post hoc, propensity-matched analysis found those treated with Skyclarys in parts 1 and 2 of MOXIe recorded lower mFARS score at 3 years, compared with a matched natural history group, per the release.
“The ... recommendation for Skyclarys is a significant milestone toward our goal of bringing a treatment that slows the progression of [Friedreich’s ataxia] to the patient community in the region,” Priya Singhal, MD, MPH, head of development at Biogen, said in the release. “We look forward to leveraging Biogen’s rare disease expertise and capabilities to bring this groundbreaking treatment to patients in the European Union living with this debilitating disease.”
The recommendation for the marketing of Skyclarys will be reviewed by the European Commission, with a final decision expected in the first quarter of 2024, according to the release.