Government reimbursement brings first targeted treatment to Australian adults living with rare genetic form of MND¹

• From 1 October 2026, QALSODY™ (tofersen) will be available through the Pharmaceutical Benefits Scheme (PBS) for eligible adults living with SOD1-ALS.^1,2
• ALS is the most common form of motor neurone disease (MND)³ and SOD1-ALS is a rare subtype of the disease⁴ estimated to affect around 70 Australians.⁵
• QALSODY is the first targeted disease-modifying treatment in Australia for people living with SOD1-ALS.⁶

Adelaide, Australia, 11 September 2026 - Biogen Australia welcomes today’s Government announcement that eligible Australians living with a rare genetic form of amyotrophic lateral sclerosis - SOD1-ALS - will have subsidised access to the first targeted disease-modifying treatment for their condition. ^1,2

From 1 October 2026, QALSODY™ (tofersen) will be listed on the Pharmaceutical Benefits Scheme (PBS) for the treatment of patients with amyotrophic lateral sclerosis who have a superoxide dismutase 1 (SOD1) gene pathogenic variant.^1,2

ALS, the most common form of MND, is a progressive, life-limiting, neurological condition affecting motor neurons, nerve cells that carry messages from the brain to the muscles.^3,7 Weak muscles, stiff limbs and overactive reflexes are key features of ALS, and it most commonly starts in either an arm or leg.^3,7 As the disease progresses, people can lose their ability to move, speak, eat and eventually breathe independently.^3,7,8

Developments in genetic testing have shown that a proportion of ALS is caused by changes (mutations) in particular genes (genetic subtypes), even in people with no known family history.⁹ One such genetic subtype is caused by mutations in the SOD1 gene (SOD1-ALS) and is estimated to affect around 70 Australians.^4,5 In SOD1-ALS, these mutations can result in the accumulation of abnormal SOD1 protein that are toxic to motor neurons.^4,10

Commenting on the reimbursement, Professor Dominic Rowe, Head of Neurology and Consultant Neurologist at Macquarie University Hospital, said: “MND is one of the most devastating neurological diseases we encounter, affecting not only patients but entire families. Despite advances in care, the impact of MND in Australia continues to grow. For people living with SOD1-ALS, today’s announcement is particularly significant because we now have access to a therapy specifically designed to target the underlying genetic cause of their disease. This represents the culmination of more than three decades of research since the SOD1 gene was first identified as a cause of inherited MND.

“Importantly, this reinforces the value of early diagnosis and appropriate genetic testing. Understanding the underlying cause of a person's disease can help connect patients and families with specialist care, research opportunities and, where appropriate, treatments designed for their specific form of MND,” Prof. Rowe added.

Despite increasing awareness of MND and ALS, diagnosis is often delayed to up to 12 months, reflecting the variability of early symptoms.^11,12 And while the course and rate of disease progression can vary considerably between individuals,⁸ the median life expectancy for ALS is 27 months from diagnosis, highlighting the seriousness and urgency of the disease.¹³

Clare Sullivan, Chief Executive Officer of MND Australia, said the PBS listing of QALSODY has been highly anticipated by the MND community.

“QALSODY is making a difference for people living with MND associated with a mutation in the SOD1 gene. MND is a devastating disease robbing people of their ability to move their bodies, to chew and swallow and eventually to breathe, sometimes this decline is over only a few months. I’m hearing stories of hope for QALSODY positively impacting people’s disease progression, which is important for a disease that usually takes away time from families and diminishes one's dignity. While SOD1-ALS affects a relatively small number of Australians, the significance of this development should not be underestimated both for people living with this form of MND today, and for what advances in targeted treatments may mean for other forms of the disease in the future.

“The listing of this new therapy also gives genetic testing even greater relevance for people diagnosed with MND. We encourage people to speak with their neurologist about whether genetic testing is appropriate for them and what the results may mean for their care,” she added.

Alice Tien, Managing Director of Biogen Australia and New Zealand, said today’s announcement from the Hon Mark Butler, Minister for Health and Ageing, Minister for Disability and the NDIS, at the National MND Conference 2026 in Adelaide marked a significant milestone for the MND community.

“We welcome the decision to make QALSODY available through the PBS for eligible Australians living with SOD1-ALS and thank the Government for making this important therapy publicly accessible. Its listing reflects years of scientific research and collaboration across the MND community. Most importantly, we thank the clinicians, advocacy organisations, people living with MND and their families who have contributed to helping make this treatment available in Australia.”

QALSODY is a targeted therapy for people whose ALS is caused by a SOD1 mutation.^10,14,15, The medicine is designed to reduce the amount of harmful SOD1 protein that accumulates in nerve cells, to slow the disease.^9,10,15 It is administered directly into the fluid surrounding the spinal cord via a lumbar puncture, with three initial loading doses given every 14 days, followed by a one-dose maintenance treatment every 28 days.^10,15

QALSODY was provisionally registered by the Therapeutic Goods Administration (TGA) in April 2026 for the treatment of adults with ALS associated with a mutation in SOD1 gene.⁶

As with any medicine, QALSODY can cause side effects. Common side effects reported in QALSODY-treated participants included back pain, pain in arms or legs, fatigue, muscle and joint pain, fever, muscle stiffness or nerve pain and an increase in protein and/or white blood cell count occurring in the fluid that surrounds the brain and spinal cord.^10,15

People living with SOD1-ALS or their families seeking further information are encouraged to consult their treating healthcare professional.

ENDS

The QALSODY Consumer Medicine Information can be accessed here.

TGA indication¹⁰

QALSODY has provisional approval in Australia for the treatment of adults with amyotrophic lateral sclerosis (ALS) associated with a mutation in the superoxide dismutase 1 (SOD1) gene. The decision to approve this indication has been made on the totality of evidence including impacts on clinical function, respiratory function, muscle strength, survival, target engagement and reductions in NfL (a marker of axonal injury and neurodegeneration) in SOD1-ALS patients treated with QALSODY. Continued approval of this indication depends on additional data.

About QALSODY (tofersen) ^10,15

Contraindications:

• Hypersensitivity to QALSODY or any of the ingredients.

Interactions:

• The co-administration of other intrathecal medicinal products with QALSODY has not been evaluated and the safety of these combinations is not known.

Precautions:

• You may have a urine test (to check your kidneys) and a blood test (to check that your blood clots properly) before you start treatment. This is because other medicines in the same group as QALSODY can affect the kidneys and the cells in the blood which help clotting. These tests may not be needed every time you are given QALSODY.
• If you are pregnant or breastfeeding, think you may be pregnant or are planning to have a baby, ask your doctor for advice before you are given this medicine. QALSODY is not recommended to be used during pregnancy and in women of childbearing potential not using contraception.

Safety:

• Common side effects may include back pain, pain in arms or legs, increase in protein and/or white blood cell count occurring in the fluid that surrounds the brain and spinal cord, feeling tired, muscle and joint pain, fever, muscle stiffness or nerve pain..
• Serious side effects may include inflammation of the spinal cord (myelitis), irritation or inflammation of nerve roots (radiculitis), swelling of the optic nerve in the eye (papilloedema), increased pressure around the brain (increased intracranial pressure) or inflammation of the lining around the brain and spinal cord (aseptic meningitis).

Motor neurone disease / amyotrophic lateral sclerosis

Motor neurone disease (MND) is a progressive and life-limiting neurodegenerative disease that affects both upper and lower motor neurons. It has a median survival of two to four years from symptom onset and less than 10 percent of patients with MND survive beyond 10 years.^13,16

Amyotrophic lateral sclerosis (ALS) is the most common form of MND, although most countries use both terms interchangeably.³ It has gained global public recognition through the Ice Bucket Challenge and, in Australia, the Big Freeze.^17,18

ALS typically presents between the ages of 40 and 70 years.¹⁹ In most cases, symptoms begin in the limbs, while around one quarter of people first experience difficulties with speech or swallowing (bulbar onset), and a smaller proportion present initially with respiratory symptoms.^20,12

SOD1-associated amyotrophic lateral sclerosis (SOD-1 ALS)

SOD1-associated amyotrophic lateral sclerosis (SOD-1 ALS) is a genetic subtype of ALS. Mutations in the SOD1 gene causes SOD1 proteins to build up inside nerve cells that control muscle movement, which damages nerves and leads to the loss of muscle function.⁴ SOD1‑ALS can present at a younger age and may progress more rapidly than ALS overall.⁴ Not all SOD1 mutations are inherited - a proportion of people with SOD1- ALS have no known family history of the disease.⁹ Genetic testing is therefore relevant for people diagnosed with ALS and not only those with a strong family history²¹ and is available through reimbursable testing pathways in appropriate clinical settings.

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, two globally co-developed treatments to address a defining pathology of Alzheimer’s disease, the first treatment to target a genetic form of ALS, the first oral treatment approved for postpartum depression, and the first approved treatment for Friedreich’s 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.

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