European Commission approves Skyclarys for Friedreich’s ataxia

Key takeaways:

• Skyclarys was approved in the U.S. for Friedreich’s ataxia in 2023.
• Approval was based on positive results from the MOXIe part 2 clinical trial.

The European Commission has authorized Skyclarys to treat Friedreich’s ataxia in adults and adolescents aged 16 years and older.

The drug was initially approved by the FDA in February 2023 for the same population.

According to a release from Biogen, Skyclarys (omavexolone) became the first treatment approved within the European Union for the rare, genetic, progressive neurodegenerative disease.

European Commission approval was based on efficacy and safety data from the 48-week, placebo-controlled MOXIe part 2 trial, which showed those who received 150 mg Skyclarys daily demonstrated significant improvement in modified Friedreich Ataxia Rating Scale (mFARS) scores relative to placebo.

The therapeutic was also shown to be generally well-tolerated, with the most common side effects being decreased weight and appetite, nausea, vomiting, diarrhea, headache, fatigue, oropharyngeal and back pain, muscle spasms and influenza.

Friedreich’s ataxia symptoms arrive early, typically in childhood, and include progressive loss of coordination, muscle weakness and fatigue, along with impaired vision, hearing loss, problems with speech and swallowing as patients grow older. Those with the condition typically require walking aids and often require a wheelchair within 10 to 20 years following diagnosis, per the release.

“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,” Priya Singhal, MD, MPH, head of development at Biogen, said in the release. “Our team is committed to engaging with the medical community and local authorities as we work to urgently secure access for patients.”

Skyclarys became commercially available in the United States in July 2023.

“On behalf of Euro-ataxia and its members, I am delighted to welcome the European Commission approval of Skyclarys a much-needed medicine for adults with Friedreich’s ataxia in EU countries,” Andreas Nadke, president of Euro-ataxia, said in the release.