TGA approves first treatment in rare, neurogenerative disease

The Therapeutic Goods Administration (TGA) has approved the first treatment for Friedreich ataxia (FA), marking a turning point for adults and adolescents aged ≥16 living with this rare neurodegenerative disease.

Skyclarys (omaveloxolone) was acquired by Biogen through its $7.3 billion acquisition of Reata Pharmaceuticals in 2023. FA, which often manifests in childhood, severely impacts mobility and leads to an average life expectancy of only 37 years.

“This TGA registration represents an important step forward for this very challenging disease as, until now, there have been no approved treatments available for those living with Friedreich ataxia in Australia,” said Professor Martin Delatycki, Co-Director at the Bruce Lefroy Centre, Murdoch Children’s Research Institute (MCRI) and clinical geneticist at the Victorian Clinical Genetics Services (VCGS).

“This is a complex degenerative disease with major impact on quality of life and lifespan which affects largely young Australians – so this announcement is very significant. The focus now must be on early genetic testing to identify affected individuals so they can receive the best possible treatment.”

The approval was based on data from the pivotal MOXIe Part 2 trial, in which patients treated with Skyclarys showed statistically significant improvement on the modified Friedreich’s Ataxia Rating Scale (mFARS) compared to placebo at 48 weeks. Clinical gains were seen across key domains, including coordination of upper and lower limbs, swallowing, and upright stability.

“Friedreich ataxia (FA) is a progressive condition that can significantly impact a person’s ability to perform everyday activities – things many of us may take for granted, like jumping out of bed, eating breakfast, or running for the train. As the disease progresses, these challenges often become more pronounced,” said Brad Hyde, CEO of the Friedreich Ataxia Research Association (FARA).

“Most people are diagnosed in childhood or adolescence, which can alter life plans and expectations. The FA community remains driven by hope and determination, and today’s milestone represents an exciting step forward.”

David Henderson, General Manager of Biogen Australia and New Zealand, echoed that sentiment.

“Today’s news is a significant milestone in the journey to ensuring people living with FA in Australia can access a new treatment option,” he said.

“Friedreich ataxia is a debilitating neurological condition that transforms everyday activities into overwhelming challenges. Biogen is deeply committed to the local communities impacted by rare and neuromuscular diseases, and we have a strong sense of urgency in our focus on bringing treatments to the people, and their families, who need them.”

FA affects an estimated 1 in 29,000 to 42,000 Australians. National centres of excellence include the Melbourne Friedreich Ataxia Clinic at Monash Medical Centre and the Brisbane Friedreich Ataxia Clinic at the Royal Brisbane and Women’s Hospital.

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