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First treatment option SKYCLARYS(TM) (omaveloxolone) for Australian's diagnosed with 'neurogenerative disease' Friedreich ataxia

Biogen Australia

 

• 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 – 21^st 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).

 

People living with FA or their families seeking further information are encouraged to consult their treating healthcare professional.

 

Issued on behalf of Biogen Australia by Cube   For further information please contact Natasha Silkin via [email protected] or +61 494 408 965

 

TGA indication: SKYCLARYS is indicated for the treatment of Friedreich ataxia in adults and adolescents aged 16 years and older.

 

About SKYCLARYS™ (omaveloxolone)¹

 

Contraindications:

• Hypersensitivity to omaveloxolone or any of the ingredients

 

Precautions: 

• Your doctor will check to see how well your liver is working, your cholesterol level and your level of BNP (B-type natriuretic peptide, a blood test that is a marker of heart problems) before you start taking SKYCLARYS.
• Your doctor will be checking blood tests while you are taking SKYCLARYS.
• SKYCLARYS can reduce the effectiveness of hormonal birth control. You should use a different method of birth control and tell your doctor immediately if you become pregnant.

 

Interactions:

• Avoid eating grapefruit or grapefruit juice while taking SKYCLARYS.
• Some medicines may interfere with SKYCLARYS and affect how it works.

 

Safety:

• Contact your doctor immediately if you have sudden weight gain, swelling of legs, ankles, or feet, or shortness of breath, which may be signs or symptoms of heart problems while taking SKYCLARYS.

 

About Friedreich ataxia

Friedreich ataxia (FA) is a rare, genetic, life-shortening neurodegenerative disease that causes progressive damage to the nervous system.³ FA is the most common inherited ataxia¹¹ (loss of coordination), affecting approximately 1 in 29,000 to 42,000 people in Australia.^11,12,13

 

FA is often diagnosed in childhood,  with first symptom presenting as difficulty walking.⁵ As the disease gradually worsens, it spreads to the arms and then the trunk⁵ leading to further muscle weakness and a decline in loss of balance and coordination.⁶  As FA further progresses, people can experience issues with their hearing, sight, speech and swallowing, as well as heart disease, diabetes and curvature of the spine (scoliosis).⁶  The disease usually results, within eight to ten years following the initial symptoms, in an inability to walk⁵ with many people with FA needing to use walking aids and wheelchairs.⁷ The average life expectancy of someone with FA is 37 years.⁴

 

About Biogen

Founded in 1978, Biogen is a leading global biotechnology company that has pioneered multiple breakthrough innovations including a broad portfolio of medicines to treat multiple sclerosis, the first approved treatment for spinal muscular atrophy, globally two co-developed treatments to address a defining pathology of Alzheimer’s disease, the first treatment to target a genetic form of amyotrophic lateral sclerosis, the first oral treatment approved for postpartum depression, and the first approved treatment for Friedreich ataxia. Biogen is advancing a pipeline of potential novel therapies across neurology, neuropsychiatry, specialised immunology and rare diseases and remains acutely focused on its purpose of serving humanity through science while advancing a healthier, more sustainable and equitable world.

 

SKYCLARYS™ and Biogen^® are registered trademarks of Biogen MA Inc. Biogen Australia Pty Ltd, Macquarie Park, NSW. ©2025 Biogen-266672. Date of preparation: June 2025.

 

 

References:

Contact details:

Natasha Silkin via [email protected] or +61 494 408 965