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Australia Offers Subsidized Coverage of Spinraza for Pre-symptomatic Children With SMA

Australia Offers Subsidized Coverage of Spinraza for Pre-symptomatic Children With SMA
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Australia is expanding access to Spinraza (nusinersen) to children and infants with severe spinal muscular atrophy (SMA) who have not yet developed symptoms, a period when the medicine is likely to benefit patients the most.

Effective Dec. 1, Biogen’s Spinraza will be added to the Pharmaceutical Benefits Scheme (PBS) for the treatment of asymptomatic children and infants diagnosed with severe types of SMA, following a recommendation from the independent Pharmaceutical Benefits Advisory Committee (PBAC), Australian Health Minister Greg Hunt announced in a press release.

The decision means that children and infants diagnosed with SMA will be able to start treatment with Spinraza from birth. Treatment given during the pre-symptomatic stages is thought to be the most effective, since motor neurons — the nerve cells responsible for controlling voluntary muscles and whose loss characterizes SMA — are still fairly preserved at this stage.

This decision is expected to benefit approximately 18 children and infants, and their families per year.

Without the subsidy, families would need to pay AU$367,000 (approximately $269,000) a year for Spinraza. The addition of the therapy to the Australian PBS means Australians will pay about AU$41 (approximately $30) or AU$6.60 (approximately $5) per script with a concession card.

Spinraza, the first disease-modifying therapy for all types of SMA, was first made available through the PBS in May 2018 for patients with SMA age 18 or younger.

The PBAC is considering an application to expand Spinraza access to adults. A decision is expected by Dec. 18 and will be announced on the PBS website.

Patricia holds a Ph.D. in Cell Biology from University Nova de Lisboa, and has served as an author on several research projects and fellowships, as well as major grant applications for European Agencies. She has also served as a PhD student research assistant at the Department of Microbiology & Immunology, Columbia University, New York.
Total Posts: 85

Ana holds a PhD in Immunology from the University of Lisbon and worked as a postdoctoral researcher at Instituto de Medicina Molecular (iMM) in Lisbon, Portugal. She graduated with a BSc in Genetics from the University of Newcastle and received a Masters in Biomolecular Archaeology from the University of Manchester, England. After leaving the lab to pursue a career in Science Communication, she served as the Director of Science Communication at iMM.

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Patricia holds a Ph.D. in Cell Biology from University Nova de Lisboa, and has served as an author on several research projects and fellowships, as well as major grant applications for European Agencies. She has also served as a PhD student research assistant at the Department of Microbiology & Immunology, Columbia University, New York.
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