Clinicians welcome first reimbursed therapy in Spinal Muscular Atrophy

Pharma News: From 1 August 2022, adults with 5q Spinal Muscular Atrophy (SMA) and symptom onset prior to their 19th birthday will be able to access Biogen’s SPINRAZA (nusinersen) on the Pharmaceutical Benefits Scheme (PBS), the first and only reimbursed treatment for this patient population in Australia.

Professor Matthew Kiernan, Bushell Chair of Neurology at Royal Prince Alfred Hospital and Co-Director of the Brain and Mind Centre, University of Sydney said SMA has a significant impact on the day to day lives of adults with the disease.

“For adults with SMA this results in a loss of functional ability over time, with an increased risk of respiratory infections and increased hospital admissions. For many, it becomes a progressive battle against time to preserve quality of life and independence which can be distressing for themselves, their carers, and family members,” Professor Kiernan said.

SPINRAZA has been available on the PBS since June 2018 for children with SMA who present with symptoms prior to three years of age, and since 2020 for infants with pre-symptomatic SMA.8,9 “Until now there have been no treatment options available for adults living with SMA. This PBS listing is welcome news and means that Australians living with SMA now have access to treatment regardless of age,” Julie Cini, Spinal Muscular Atrophy Australia spokesperson said. “This was a highly anticipated decision the SMA community has been waiting for.

David Henderson, Managing Director, Biogen ANZ said, “At Biogen ANZ, we are proud of our continued commitment to both adults and children living with SMA. We understand the impact SMA has not only on the individual but on their families and those who care for them. This announcement today exemplifies why the connection between science and humanity is so important, as we continue to invest in research and support for the SMA community.”

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 of the muscles in 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, like breathing and swallowing.