Sydney, Australia – July 31, 2022.
Starting August 1, 2022, adults (at least 19 years of age or older) with spinal muscular atrophy (SMA) 5q and symptom onset before their 19e birthday will be able to access SPINRAZA® (nusinersen) on the Pharmaceutical Benefits Scheme (PBS),1 the first and only reimbursed treatment for this patient population in Australia.2
SMA is a rare genetic disease caused by a shortage of a protein called survival motor neuron (SMN). This results in the loss of nerve cells in the spine, leading to progressive weakness in the muscles of the shoulders, hips, thighs, and upper back. For adults living with SMA, the level of muscle weakness can be significant, including paralysis and difficulty with the most basic functions of life, such as breathing and swallowing.3.4
SPINRAZA is a type of medicine called an antisense oligonucleotide that works by tricking the body into producing more SMN protein in a type of SMA known as SMA 5q. 3
SMA occurs in about one in every 6,000 to 11,000 births.5.6 Currently, around 650 to 800 Australians are living with SMA.seven
Professor Matthew Kiernan, Bushell Professor of Neurology at the Royal Prince Alfred Hospital and co-director of the Brain and Mind Center at the University of Sydney, said SMA has a significant impact on the daily lives of adults with the condition. . “For adults with SMA, this leads to a loss of functional ability over time, with an increased risk of respiratory infections and increased hospital admissions. For many, it becomes a gradual battle against time to maintain quality of life and independence, which can be distressing for themselves, their caregivers and family members,” Prof Kiernan said.
SPINRAZA has been available on PBS since June 2018 for children with SMA who show symptoms before the age of three, and since 2020 for infants with pre-symptomatic SMA.8.9 “Until now there have been no treatment options available for adults with SMA. This PBS listing is welcome news and means that Australians living with SMA now have access to treatment regardless of age,” said said Julie Cini, Spokesperson for Spinal Muscular Atrophy Australia “This was a highly anticipated decision that the SMA community has been waiting for.”
David Henderson, Chief Executive of Biogen ANZ, said: “At Biogen ANZ, we are proud of our continued commitment to adults and children living with SMA. We understand the impact of SMA not only on the individual, but also on their family and those who care for them. Today’s announcement illustrates why the connection between science and humanity is so important, as we continue to invest in research and support for the SMA community.
Issued by the SenateSHJ on behalf of Biogen
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