Life changing gene therapy for babies with spinal muscular atrophy

Department of Health

The Albanese Government is giving more families access to life changing gene therapy for spinal muscular atrophy through the Pharmaceutical Benefits Scheme (PBS).

Zolgensma® (onasemnogene abeparvovec) has been expanded to treat more babies with genetically diagnosed spinal muscular atrophy who are yet to show any symptoms.

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 babies aged under the age of 2.

Zolgensma is a one-time-only dose of gene therapy that works by supplying a fully functioning copy of the gene needed to make an essential protein called ‘Survival Motor Neuron’ (SMN) and rescuing viable motor neurons. This can stop the progression of spinal muscular atrophy and sustain the child’s muscle function.

The PBS listing is now expanded to include babies up to nine months of age who have a SMN2 gene copy number of 3. About 15 infants a year will be impacted by this life changing listing.

This life changing therapy is now available on the PBS.

Quotes attributable to Minister Butler:

“Zolgensma is a one-off, transformative treatment that addresses the underlying cause of spinal muscular atrophy.

“Time is of the essence to treat the disease with Zolgensma, but at a cost of

$2.5 million it is out of reach for families.

“The Albanese Government has worked hard to make this life changing treatment available and affordable for families and their babies as soon as possible.

“The PBS is a vital lifeline to medicines for millions and we’ll continue to ensure it meets the needs of all Australians.”

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