Biogen Inc. (Nasdaq: BIIB) announced the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) recommended marketing authorization for SKYCLARYS® (omaveloxolone) for the treatment of Friedreich’s ataxia (FA) in people aged 16 years and older. If approved by the European Commission (EC), SKYCLARYS will be the first treatment authorized within the European Union for this rare, genetic, progressive neuromuscular disease.1 If approved, omaveloxolone will be marketed as SKYCLARYS.
“The CHMP’s recommendation for SKYCLARYS is a significant milestone toward our goal of bringing a treatment that slows the progression of FA to the patient community in the region,” said Priya Singhal, M.D., M.P.H., Head of Development at Biogen. “Upon approval of SKYCLARYS, 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 CHMP’s positive opinion for SKYCLARYS is based on efficacy and safety data from the placebo-controlled MOXIe Part 2 trial. At the end of the 48-week MOXIe Part 2 study, patients who received SKYCLARYS had less physical impairment compared to patients who received placebo, as measured by the modified Friedreich Ataxia Rating Scale (mFARS). Improvements across subscales of mFARS, including upright stability, lower limb coordination, ability to swallow and upper limb coordination, were also observed in patients treated with SKYCLARYS compared to placebo. Additional data was provided from a post hoc, propensity-matched analysis in which patients treated with SKYCLARYS in MOXIe (Parts 1 and 2) had lower mFARS score at 3 years, as compared to a matched natural history group. The most common side effects are increased liver enzymes, decreased weight and appetite, nausea, vomiting, diarrhea, headache, fatigue, oropharyngeal and back pain, muscle spasms, and influenza.
The CHMP’s recommendation for SKYCLARYS will now be reviewed by the EC for marketing authorization in the European Union with a final decision expected in the first quarter of 2024. The U.S. Food and Drug Administration (FDA) approved omaveloxolone, marketed as SKYCLARYS®, in February 2023 for the treatment of Friedreich’s ataxia in adults and adolescents aged 16 years and older.
