Biogen receives EC approval for Skyclarys in Friedreich’s ataxia patients

Biogen has announced that the European Commission (EC) has approved Skyclarys (omaveloxolone) to treat patients aged 16 years and older with Friedreich’s ataxia (FA), a rare and inherited neurodegenerative disorder.

The company gained access to the drug, which is now the first treatment approved in the EU for FA, in September 2023 after completing its previously-announced $7.3bn acquisition of Reata Pharmaceuticals.

FA, while rare, is the most common inherited ataxia. Early symptoms of the condition, such as progressive loss of coordination, typically appear in childhood and most patients will need to use a wheelchair within ten to 20 years of their first symptoms.

Priya Singhal, head of development at Biogen, said the company was “proud" to add Skyclarys to its portfolio of medicines and "address a significant unmet need” by bringing the first treatment to FA patients in Europe.

“Our team is committed to engaging with the medical community and local authorities as we work to urgently secure access for patients,” Singhal said.

The EC’s decision was supported by positive results from the placebo-controlled MOXIe trial, in which Skyclarys-treated patients had significantly improved modified Friedreich Ataxia Rating Scale (mFARS) scores compared to those in the placebo cohort.

All components of the mFARS assessment, including the ability to swallow, upper limb coordination, lower limb coordination and upright stability, favoured Skyclarys over placebo, Biogen said, adding that exploratory data showed that patients receiving Skyclarys in an extension of MOXIe had lower mFARS scores at three years compared to a matched natural history group.

Principal investigator of the study, Sylvia Boesch, of Medical University Innsbruck, said: “FA patients treated with Skyclarys in the clinical trial experienced important and clinically meaningful improvements for their daily lives.

“With this approval, there is optimism within the community that Skyclarys has the potential to usher in a new era in the management of FA.”

Skyclarys had already been approved by the US Food and Drug Administration when Biogen announced its intended buyout of Reata in July and an application had been filed in Europe.

Although Skyclarys was the main asset included in the acquisition, Biogen also gained access to a portfolio of products that Reata was developing for a range of neurological diseases.