Patisiran has been recommended by the Pharmaceutical Benefits Advisory Committee for those with hereditary transthyretin amyloidosis with polyneuropathy.
Patisiran is an intravenous a gene silencing therapy that is given every 3 weeks. In the landmark APOLLO trial Patisiran was proven to reduce the production of the disease-causing transthryretin protein resulting in slowed disease progression and improved quality of life. 56% of patients showed improvement in neuropathy symptoms after 18 months of treatment.
This important recommendation is the first step in the process for Patisiran to be listed on the PBS for those with hereditary ATTR with polyneuropathy. The AAN looks forward to this important treatment becoming available on the PBS in 2024 after further negotiations between the pharmaceutical company and Australian government. Liaise with your nearest AAN centre for program updates.
