Access to a life-saving and life-changing medicine will soon be expanded for Australian children and infants with Spinal Muscular Atrophy (SMA), thanks to a breakthrough medicines listing on the Pharmaceutical Benefits Scheme (PBS).

From 1 December, subsidised access to the life-changing medicine Spinraza® (nusinersen) will be expanded to include children and infants genetically diagnosed with the most severe types of SMA before the onset of symptoms (pre-symptomatic).

SMA is a rare inherited genetic muscle wasting disease characterised by a loss of motor neurons. It causes progressive muscle weakness and wasting, and its most severe forms can cause paralysis and death.

Without the subsidy, families would pay more than $367,000 a year for treatment. From 1 December families and their children will be able to access this life changing medicine for $41 or $6.60 per script with a concession card.

Approximately 18 children and infants, and their families, are expected to benefit from this expanded PBS listing per year.

The expanded listing of Spinraza will allow infants and children, diagnosed with SMA through a genetic test, to start treatment prior to the onset of symptoms.

This means children and infants diagnosed with SMA will be able to commence treatment with this medicine from birth and up to three years earlier, which is so critical to address the impact of this debilitating condition.

The least amount of damage to a child’s motor neurons occurs when they are pre-symptomatic.

The medicine works by slowing progression of the disease. In many cases, it can halt the progression of the disease or significantly improve motor function.

Starting treatment early is critical to halting the impact of this significantly debilitating and life-threatening condition, and preventing irreversible impacts.

Left unaddressed, infants with SMA struggle to meet the simplest of development milestones such as holding their head up, sitting up by themselves, walking and standing.

One in 10,000 live births in Australia are affected by SMA and the disease is the number one genetic cause of death of babies under two in Australia. There is no known cure for SMA.

One in 35 people in Australia unknowingly carry the faulty SMA gene. Being a carrier does not mean you are affected by the condition.

Spinraza is the first and only treatment of its kind to be listed on the PBS for SMA. It was first listed on the PBS in June 2018, and so far over 160 Australians per year are being treated with this medicine.

This PBS listing was recommended by the independent Pharmaceutical Benefits Advisory Committee (PBAC).

Since 2013, the Government has approved more than 2,500 new or amended listings on the PBS. This represents an average of around 30 listings or amendments per month – or one each day – at an overall investment by the Government of $11.8 billion.

The Government’s commitment to ensuring Australians can access affordable medicines when they need them, remains rock solid.

The independent medical experts on the PBAC have been considering an application to expand the listing of Spinraza for the treatment of adults. An update on these deliberations will be provided on 18 December on the PBS website at: https://www.pbs.gov.au/pbs/home.

Further information on SMA can be found at: https://smaaustralia.org.au/wp-content/uploads/SMA_2020_A4_Information_Guide-compressed.pdf