Life changing gene therapy for children with spinal muscular astrophy on the PBS

The Albanese Government is giving more young children access to a life-changing gene therapy for spinal muscular atrophy, through an expanded listing on the PBS.
 
From 1 November 2026, access to Zolgensma® (onasemnogene abeparvovec) has been expanded from babies up to 9 months, to eligible children up to 21 kilograms in weight.
 
Spinal muscular atrophy is a rare inherited genetic condition that affects the nerves that control muscle movement (motor neurons). It causes progressive muscle weakness and wasting, and in its most severe forms leads to paralysis and death.
 
One in 35 Australians unknowingly carry the spinal muscular atrophy gene and one in 10,000 babies born in the country is affected by the disease.

Left untreated, spinal muscular atrophy is the number one genetic cause of death of children under two years of age.
 
Zolgensma is a one-time gene therapy designed to address the underlying genetic cause of SMA by delivering a functional copy of the SMN1 gene, helping the body produce an essential protein needed for muscle function and survival.
 
Without PBS subsidy, this one-off treatment could cost patients more than $2.5 million.
 
It’s expected up to five children will benefit from this life-changing listing each year.
 
The listing of these and other medicines means the Albanese Government has now funded over 500 new and amended PBS listings since July 2022.
 
Quotes attributable to Minister Butler:
 
“Zolgensma is a one-off, transformative treatment that addresses the underlying cause of spinal muscular atrophy.

“Timing is critical to treating spinal muscular atrophy with Zolgensma, but at a cost of over $2.5 million it is out of reach for families.
 
“The Albanese Government is making this life changing treatment available and affordable for more families and their young children.”