New treatments for SMA continue to be researched, developed, and evaluated through clinical trials. SMA Australia advocates for the latest overseas-approved treatment options to be made available to Australians living with SMA and will keep the community updated as access expands.

Clinical trials – research studies that assess the safety and effectiveness of new medical treatments, tests or procedures in people – may be available for you or your child.  Your neurologist can advise on relevant trials, or you can explore current worldwide clinical trials at ClinicalTrials.gov and discuss potential options with your neurologist.

For a broader view of SMA drug development, the Cure SMA (US) website provides a detailed pipeline of medicines at various stages, from pre-clinical discovery to FDA approval and availability in the United States. You can explore the SMA Drug Pipeline here.

In Australia, the Therapeutic Goods Administration (TGA) oversees medicine approvals, ensuring treatments meet rigorous standards for safety, efficacy, and quality before becoming available to the public. While some medicines gain TGA approval soon after receiving FDA approval, others may face a longer process due to factors such as local clinical trials and additional data requirements – or may not receive TGA approval at all.

Once approved by the TGA, a treatment can be submitted to the Pharmaceutical Benefits Advisory Committee (PBAC) for evaluation. Medicines that receive a positive PBAC recommendation and are subsequently listed on the Pharmaceutical Benefits Scheme (PBS) become accessible for eligible patients in Australia.