For consumer media only
First treatment option SKYCLARYS™ (omaveloxolone) for Australian's diagnosed with 'neurogenerative disease' Friedreich ataxia[1]
- The TGA has registered SKYCLARYS (omaveloxolone), the first treatment[2] for the rare genetic disease Friedreich ataxia (FA).[3]
- FA is a rare neurogenerative disease which causes progressive damage to the nervous system3 leading to significant disability and an average life expectancy of only 37 years.[4]
- SKYCLARYS is the first treatment registered in Australia for Friedreich ataxia (FA) in eligible individuals aged 16 years and older.1
Sydney, Australia – 21st July 2025 – Biogen Australia welcomes the TGA (Therapeutic Goods Administration) approval of SKYCLARYS™ (omaveloxolone) as the first treatment for adults and adolescents aged 16 years and older with the rare, genetic, progressive disease Friedreich ataxia (FA).1
FA is a life-shortening, neurodegenerative disease often diagnosed in childhood, with the first symptom presenting as difficulty walking.[5] Balance and coordination decline over time.[6] It usually results, within eight to ten years following initial symptoms, in an inability to walk5 with many people with FA needing to use walking aids and wheelchairs.[7]
As the disease progresses, people can also experience issues with their hearing, sight, speech and swallowing, as well as heart disease, diabetes and curvature of the spine (scoliosis),6 leading to an average life expectancy of only 37 years.4
SKYCLARYS is an oral, once daily medication[8] that activates a pathway, called Nrf2,[9] in the body which lowers inflammation to prevent tissue and cell damage. It is thought that increasing Nrf2 activity assists in slowing disease progression.[10]
"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, 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."
"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."
FA is the most common inherited ataxia[11] (loss of coordination), affecting approximately 1 in 29,000 to 42,000 people in Australia.11,[12],[13] As the condition affects many parts of the body, its overall management is complex, and a multi-disciplinary healthcare team is often needed. In Australia, key centers of excellence for FA include the Melbourne Friedreich Ataxia Clinic at Monash Medical Centre and the Brisbane Friedreich Ataxia Clinic based at the Royal Brisbane and Women's Hospital in Queensland.
"Today's news is a significant milestone in the journey to ensuring people living with FA in Australia can access a new treatment option," said David Henderson, General Manager of Biogen Australia and New Zealand. "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."
SKYCLARYS is generally well tolerated with a manageable side-effect profile. Adverse events are generally considered mild to moderate.[14]
The SKYCLARYS Consumer Medicine Information can be accessed here.
SKYCLARYS is not listed on the Pharmaceutical Benefits Scheme (PBS).
