spinal muscular atrophy
Rare disease · 2017–2025
Every decision
| Medicine | Meeting | Outcome | Consumer input |
|---|---|---|---|
| risdiplam Treatment of spinal muscular atrophy (SMA) in adults and paediatric patients (symptomatic Type I, II or IIIa SMA) aged 2 years or older weighing 20 kg or more. | Nov 2025 | Recommended with restriction | 1 individuals |
| risdiplam Treatment of pre-symptomatic spinal muscular atrophy (SMA) in patients aged under 36 months with confirmed genetic diagnosis (SMN1 deletion or mutation) and 3… | Jul 2024 | Recommended with restriction | 1 individuals |
| onasemnogene abeparvovec Pre-symptomatic treatment of babies with spinal muscular atrophy (SMA) and 3 copies of the Survival Motor Neuron 2 (SMN2) gene. | Jul 2023 | Recommended with restriction | input received Spinal Muscular Atrophy Australia · Sydney Children Hospital Network |
| risdiplam Treatment of 5q spinal muscular atrophy (SMA) in adults with symptom onset prior to 19 years of age and no prior disease-modifying treatment during childhood,… | Mar 2023 | Recommended with restriction | 22 individuals |
| nusinersen Treatment of adult patients (aged 19 years or older) diagnosed with spinal muscular atrophy (SMA) with symptom onset before 19 years of age (primarily SMA… | Mar 2022 | Recommended with restriction | input received |
| onasemnogene abeparvovec Treatment of pre-symptomatic paediatric patients less than 9 months of age with spinal muscular atrophy (SMA) who are genetically diagnosed with bi-allelic… | Nov 2022 | Not recommended | 22 individuals Spinal Muscular Atrophy Australia · National Network · Paediatric Neuromuscular Clinical Research Network |
| nusinersen Treatment of spinal muscular atrophy (SMA) in adult patients (>18 years) with 5q SMA who experienced signs and symptoms prior to 19 years of age. | Jul 2021 | Not recommended | 72 individuals Muscular Dystrophy Association of NSW · Duchenne Australia · SMA Australia |
| risdiplam Treatment of spinal muscular atrophy (SMA) Types 1, 2, and 3a in patients aged 18 years or under at treatment initiation (Population 1); SMA Type 3b in… | Mar 2021 | Recommended with restriction | 219 individuals SMA Australia · Muscular Dystrophy Association of NSW |
| nusinersen Extension of listing for pre-symptomatic initiation of treatment in patients with genetically confirmed spinal muscular atrophy (SMA) with SMN1 deletion or… | Jul 2020 | Recommended with restriction | 11 individuals Spinal Muscular Atrophy Australia · Rare Voices Australia |
| nusinersen Treatment of spinal muscular atrophy (SMA) in patients with symptom onset prior to 19 years of age, with removal of the age limit of 18 years for initiation of… | Nov 2020 | Not recommended | 121 individuals SMA Australia |
| nusinersen Pre-symptomatic initiation of treatment of patients with Spinal Muscular Atrophy (SMA) who have SMN1 deletion or mutation with up to 3 copies of the SMN2 gene… | Jul 2019 | Deferred | input received Spinal Muscular Atrophy Australia · Rare Voices Australia |
| nusinersen Pre-symptomatic initiation of treatment of patients with Spinal Muscular Atrophy (SMA) who have up to 3 copies of the survival-of-motor-neuron 2 (SMN2) gene. | Jul 2019 | Deferred | input received Spinal Muscular Atrophy Australia · Rare Voices Australia |
| nusinersen Treatment of paediatric patients with infantile-onset or childhood-onset Spinal Muscular Atrophy (SMA) with onset of symptoms prior to 3 years of age. | Jul 2018 | Recommended with restriction | no section in document |
| nusinersen Treatment of infantile-onset (Type I) and childhood-onset (Types II and III) spinal muscular atrophy (SMA). | Nov 2017 | Not recommended | 1,087 individuals SMA Australia · Muscular Dystrophy Association of NSW |
Who spoke
Spinal Muscular Atrophy Australia · SMA Australia · Rare Voices Australia · Muscular Dystrophy Association of NSW · Duchenne Australia · Sydney Children Hospital Network · National Network · Paediatric Neuromuscular Clinical Research Network
What the PBS pays
$106M in government benefit over 2024–25, across 2,111 services.
This is spend on the medicines considered for this condition, not spend on the disease. A medicine used for several conditions is counted in full under each, so figures across conditions add to more than national PBS expenditure.
| Medicine | Government benefit |
|---|---|
| nusinersen | $55.2M |
| risdiplam | $49.3M |
| onasemnogene abeparvovec | $1.5M |
Appraised elsewhere
NICE, in England and Wales, has appraised 1 technology for this condition, recommending 1 . A different committee, a different population and a different price: this is what has been looked at there, not a verdict on what happened here.
| Technology | Appraisal | Outcome | Date |
|---|---|---|---|
| nusinersen and risdiplamNusinersen and risdiplam for treating spinal muscular atrophy | TA1162 | recommended restricted | 2026-06 |
Conditions are classified by a language model from the indication text of each submission, so an individual label here can be wrong. Consumer input is read from the document's own consumer comments section: 13 of 14 decisions since 2014 carried some. Silence means no comment reached the committee through that facility, not that nobody was affected. More on how this was built.