MHRA approves Biogen's Skyclarys for Friedreich's ataxia

Biogen’s Skyclarys (omaveloxolone) has received approval from the Medicines and Healthcare products Regulatory Agency (MHRA) as the first available treatment for individuals aged 16 and older suffering from Friedreich’s ataxia (FA), a rare neurodegenerative movement disorder. Friedreich’s ataxia is noted as the most prevalent hereditary ataxia, a cluster of disorders impacting coordination, balance, and speech, with a prevalence rate of at least one in every 50,000 individuals in the UK.

The early manifestations of this life-shortening disorder, such as a gradual loss of coordination and muscle strength, usually emerge during childhood. Within a decade or two following the onset of these initial symptoms, the majority of patients will require the use of a wheelchair. As the disease progresses, patients might also encounter hearing loss, vision problems, speech and swallowing difficulties, scoliosis, diabetes, and severe cardiac conditions.

Skyclarys is administered orally in capsule form and works by activating the Nrf2 pathway, which aids cells in addressing conditions that could cause cell and tissue harm. The MHRA’s decision to approve the drug was based on encouraging results from the MOXIe trial, which was placebo-controlled. Patients treated with Skyclarys demonstrated superior performance in neurological function tests compared to those in the placebo group.

Biogen had previously reported that all elements of the modified FA Rating Scale (mFARS) assessment, which includes swallowing capability, coordination of upper and lower limbs, and maintaining upright stability, showed a preference for Skyclarys over the placebo. Julian Beach, the MHRA interim executive director of healthcare quality and access, expressed his satisfaction with the approval of Skyclarys as the first treatment for Friedreich’s ataxia in the UK. He emphasized that the drug has met the necessary regulatory standards concerning safety, quality, and effectiveness, and that its safety will be continuously monitored.

Biogen acquired access to Skyclarys in September 2023 following its $7.3 billion acquisition of Reata Pharmaceuticals. The drug had already been sanctioned by the US Food and Drug Administration when Biogen announced its planned takeover of Reata in July. Additionally, Skyclarys secured approval from the EU in February of the previous year. While Skyclarys was the primary asset in the acquisition, Biogen also obtained access to a collection of products that Reata was developing for various neurological conditions.