The life-saving gene therapy Zolgensma, priced at $2.5 million per infusion, is being expanded for Australian children with spinal muscular atrophy, with eligibility expanding from babies up to nine months to children weighing up to 21 kilograms.
The therapy delivers a working copy of the faulty gene that causes spinal muscular atrophy (SMA), a condition that damages nerves controlling muscles and is described by paediatric neurologist Professor Michelle Farrar as "the biggest genetic cause of infant death that we have." Around one in 10,000 babies is affected.
Eligibility was previously limited to babies up to nine months old. The expansion allows older children to receive the one-off treatment, which Health Minister Mark Butler confirmed was the most expensive drug on the Pharmaceutical Benefits Scheme. "It's a one-off course of treatment that has extraordinary results in improving conditions," Butler said.
Adriana Sharpe, whose daughter was the first baby in Australia identified with SMA through newborn screening, said she was told her child might never reach age two. "We were told there was no cure and that left untreated the muscles would waste away," Sharpe said. Her daughter received emergency gene therapy at four weeks old and is now eight years old. "It's been life changing and lifesaving," Sharpe said. "She's doing incredibly well. She's achieved all of her milestones which has been amazing."
Professor Farrar said the expansion "gives families the opportunity to take their time to decide whether this is the right treatment for their child" and that early and rapid diagnosis and urgent treatment are important.
Analysis
Why This Matters
- The expansion gives older children with SMA access to a treatment that can prevent muscle wasting and death, where previously they had none.
- It offers families more time to make a decision about the costly, one-off therapy.
- The move could reduce Australia's infant mortality rate from SMA, a leading genetic cause of infant death.
Background
Spinal muscular atrophy is a genetic disorder that destroys the nerves controlling movement, swallowing and breathing. Without treatment, most affected children die by age two. Zolgensma delivers a functional copy of the missing SMN1 gene in a single intravenous infusion. It has been listed on the PBS since 2021, initially for babies under nine months. The new weight-based eligibility criterion expands access to children who may have been diagnosed later or whose disease progressed more slowly.
Key Perspectives
What to Watch
- How many additional children will receive the therapy in the first year of the expanded criteria.
- Whether other countries follow Australia's lead in widening eligibility.
- Long-term outcomes data for children treated at older ages and heavier weights.
Checked
12 claims in this story checked against 1 source: 12 supported.
Show each claim
- SupportedZolgensma eligibility is expanding in Australia from babies up to nine months old to children weighing up to 21 kg.7NEWS: “Eligibility is now expanding from babies up to nine months to children weighing up to 21 kilograms, allowing more children to access this life-changing treatment each year.”
- SupportedZolgensma is the most expensive drug on the PBS.7NEWS: “Health Minister Mark Butler confirmed the treatment was the most expensive on the pharmaceutical benefits scheme.”
- SupportedZolgensma costs $2.5 million per infusion.7NEWS: “The only problem is a single infusion costs $2.5 million, making it the most expensive drug on the pharmaceutical benefits scheme.”
- SupportedProfessor Michelle Farrar described SMA as “the biggest genetic cause of infant death that we have.”7NEWS: “Professor Michelle Farrar, a paediatric neurologist, said it was “the biggest genetic cause of infant death that we have.””
- SupportedAround one in 10,000 babies is affected by SMA.7NEWS: “Around one in 10,000 babies is affected.”
- SupportedButler said: “It’s a one-off course of treatment that has extraordinary results in improving conditions.”7NEWS: ““It’s a one-off course of treatment that has extraordinary results in improving conditions,” Butler said.”
- SupportedAdriana Sharpe’s daughter was the first baby in Australia identified with SMA through newborn screening.7NEWS: “Adriana Sharpe was told her baby girl might never reach her second birthday after newborn screening picked up spinal muscular atrophy, making her the first in Australia to be identified through the screening process.”
- SupportedSharpe said: “We were told there was no cure and that left untreated the muscles would waste away.”7NEWS: ““We were told there was no cure and that left untreated the muscles would waste away.””
- SupportedSharpe’s daughter received emergency gene therapy at four weeks old and is now eight.7NEWS: “Sharpe’s daughter received emergency gene therapy at just four weeks old. She’s now eight years old.”
- SupportedSharpe said: “It’s been life changing and lifesaving.”7NEWS: ““It’s been life changing and lifesaving,” Sharpe said.”
- SupportedSharpe said her daughter was doing incredibly well and had achieved all her milestones.7NEWS: ““She’s doing incredibly well. She’s achieved all of her milestones which has been amazing.””
- SupportedFarrar said the expansion gives families time to decide whether the treatment is right for their child.7NEWS: ““It gives families the opportunity to take their time to decide whether this is the right treatment for their child,” Professor Farrar said.”
