The EC approval was driven by the efficacy and safety data from the placebo-controlled MOXIe Part 2 study in which treatment with Skyclarys improved patient function compared to placebo
EC approves Biogen’s Skyclarys for Friedreich’s ataxia. (Credit: CHUTTERSNAP on Unsplash)
Biogen has secured the European Commission (EC) approval for Skyclarys (omaveloxolone) to treat Friedreich’s ataxia (FA) in adults and adolescents of age 16 years and older.
Skyclarys is an oral, once-daily medication for this rare, genetic, progressive neurodegenerative disease. It was developed by Reata Pharmaceuticals which was acquired by Biogen last year.
The drug had received orphan drug, fast track, and rare paediatric disease designations from the US Food and Drug Administration (FDA), and orphan drug designation from the EC.
The EC approval was driven by the efficacy and safety data from the placebo-controlled MOXIe Part 2 study.
At the end of the 48-week treatment period, patients in the Skyclarys arm had significantly higher modified Friedreich Ataxia Rating Scale (mFARS) scores compared to placebo.
The drug outperformed placebo in every area of the mFARS examination, including upright stability, lower limb coordination, upper limb coordination, and the capacity to swallow.
A post hoc, propensity-matched analysis yielded further exploratory data, showing that patients treated with Skyclarys in MOXIe (Extension) had three-year mFARS scores that were lower than those of a matched natural history group.
Biogen development head Priya Singhal said: “Biogen is proud to add Skyclarys to our portfolio of medicines and address a significant unmet need by bringing the first treatment to people living with Friedreich’s ataxia in Europe.
“Our team is committed to engaging with the medical community and local authorities as we work to urgently secure access for patients.
“We sincerely thank the Friedreich’s ataxia community for their contributions that enabled the development of Skyclarys and made today’s approval possible.”
Skyclarys’s early access programmes are now available in Germany and France, with plans to expand to other nations.