Biogen Netherlands has received marketing authorisation from the UK’s Medicines and Healthcare products Regulatory Agency (MHRA) for its Skyclarys (omaveloxolone) to treat Friedreich’s ataxia in individuals aged 16 years and above.
This approval was granted through a national procedure.
Friedreich’s ataxia is a widespread hereditary ataxia type, affecting a minimum of one in every 50,000 individuals. It is characterised by progressive issues with balance and movement.
A clinical trial that involved 103 subjects demonstrated that individuals with Friedreich’s ataxia, aged between 16 and 40 years, showed neurological improvement when treated with the therapy versus a placebo.
After 48 weeks, those on omaveloxolone experienced a reduction in physical impairment.
The oral capsule works by activating the nuclear factor erythroid 2-related factor 2 (Nrf2) pathway.
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By GlobalDataThis protein is typically found at decreased levels in those with Friedreich’s ataxia and is crucial in aiding cellular response to oxidative stress.
UK MHRA Healthcare Quality and Access interim executive director Julian Beach said: “Patient safety is our top priority. I am pleased to confirm the first UK approval for the treatment of Friedreich’s ataxia, omaveloxolone.
“We’re assured that the appropriate regulatory standards of safety, quality, and effectiveness for the approval of this new treatment have been met. As with all products, we will keep its safety under close review.”
Skyclarys has been indicated for treating this condition in the adult and adolescent population aged 16 years and above, not only in the UK but also in the European Union, Canada, and the US.
The US Food and Drug Administration (FDA) has granted the fast track, orphan drug, and rare paediatric disease designations to the therapy, which also gained orphan drug designation from the European Commission for treating Friedreich’s ataxia.
In Canada, Skyclarys was authorised for marketing under the Health Canada Priority Review process.
