RARE Daily

CHMP Issues Positive Opinion for Biogen’s FA Therapy

December 19, 2023

Rare Daily Staff

The Committee for Medicinal Products for Human Use of the European Medicines Agency recommended marketing authorization for Skyclarys, Biogen’s treatment for the rare and progressive neuromuscular disease Friedreich’s ataxia.

The recommendation is for use in people aged 16 years and older. If approved by the European Commission, Skyclarys will be the first treatment authorized within the European Union for this genetic condition.

Friedreich’s ataxia (FA) is a life-shortening, debilitating, and degenerative neuromuscular disorder. It is the most common inherited ataxia. Early symptoms of FA, such as progressive loss of coordination, muscle weakness, and fatigue, typically appear in childhood and can overlap with other diseases. Most people living with FA will need to use a wheelchair within 10-20 years of their first symptoms. The reported average age of death for FA patients is just 37 years old, although with appropriate and targeted care, individuals may live many years after confinement to a wheelchair.

Skyclarys is an oral, once-daily medication indicated for the treatment of Friedreich’s ataxia (FA) in adults and adolescents aged 16 years and older. The U.S. Food and Drug Administration granted Syclarys Orphan Drug, Fast Track, and Rare Pediatric Disease designations. The European Commission has granted Orphan Drug designation in Europe to Skyclarys for the treatment of FA.

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 FDA approved Skyclarys in February 2023 for the treatment of Friedreich’s ataxia in adults and adolescents aged 16 years and older.

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