EU clears Biogen's Skyclarys for Friedreich's ataxia

12 Feb 2024
firstwordpharma.com
Drug ApprovalAcquisitionClinical Result
Regulators in Europe approved Biogen's Skyclarys (omaveloxolone) for Friedreich's ataxia (FA) in patients 16 years and older, making it the first treatment authorised in the EU for the progressive neurodegenerative disease. The Monday decision follows a positive opinion adopted by the European Medicines Agency's (EMA) drug advisory body in December.
The Nrf2 activator, which Biogen acquired after it bought Reata Pharmaceuticals for $7.3 billion, was approved last year in the US where it has a wholesale acquisition cost of $370,000 annually.
Commenting on the EU nod, Biogen head of development Priya Singhal said, "Our team is committed to engaging with the medical community and local authorities as we work to urgently secure access for patients." Early access programmes are currently open in Germany and France, with plans to expand to more countries, the company said.
The European approval was based on efficacy and safety data from Part 2 of the mid-stage MOXIe trial, which met its primary endpoint of change in the modified Friedreich's Ataxia Rating Scale (mFARS) with a placebo-corrected difference of -2.4 points after 48 weeks. Skyclarys also fared better on tests measuring balance, coordination and swallowing compared to placebo.
In addition, the filing contained exploratory data from a post-hoc analysis in which Biogen said patients treated with Skyclarys in the extended portion of the MOXIe study had lower mFARS scores at 3 years, compared to a matched natural history group.
The most common adverse reactions in MOXIe Part 2 were elevated liver enzymes, headache, nausea, abdominal pain, fatigue, diarrhoea and musculoskeletal pain.
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