News & Trends - Pharmaceuticals
Biogen’s life-saving Spinal Muscular Atrophy drug PBS listed

Pharma News: Babies and infants with the most severe form of Spinal Muscular Atrophy (SMA) will now be able to access Biogen’s SPINRAZA (nusinersen) before symptoms of occur, meaning the least amount of damage to their motor neurons as possible.
From 1 December 2020, SPINRAZA will be listed on the Pharmaceutical Benefits Scheme (PBS) for the pre-symptomatic treatment of SMA – a rare, debilitating, and often fatal, genetic neuromuscular condition, which results in progressive muscle wasting for which there is no known cure.
Without the subsidy, families would pay more than $367,000 a year for treatment. 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.
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Previously, parents and clinicians would have to wait for symptoms to initiate treatment with SPINRAZA which meant that irreversible motor loss had already occurred.
“Biogen has been, and continues to be, committed to working with all stakeholders to ensure SPINRAZA is available as an option for all people with SMA to ensure the best quality of life and outcomes for people with this devastating disease” commented Kylie Bromley, Managing Director of Biogen Australia and New Zealand.
“Today’s announcement now provides an option for more children with pre-symptomatic, genetically diagnosed SMA to access treatment, creating more hope for the future for them and their families.”
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.
Left unaddressed, infants with Spinal Muscular Atrophy (SMA) struggle to meet the simplest of development milestones such as holding their head up, sitting up by themselves, walking and standing. In its most severe forms, SMA can cause paralysis and death.
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