RisdiplamEvrysdi
Treatment of spinal muscular atrophy (SMA) in adults and children aged 2 years or older weighing 20 kg or more. The tablet formulation is being listed as an alternative to the currently available oral liquid formulation under the same restrictions.
Decisions on record
- Meeting Nov 2025 Recommended Spinal muscular atrophy
- Meeting Jul 2024 Recommended Spinal muscular atrophy with SMN2 gene copy number 3
- Meeting Mar 2023 Recommended Spinal muscular atrophy (SMA); 5q SMA
- Meeting Mar 2021 Recommended Spinal muscular atrophy types 1, 2 or 3a; aged 18 years or under
Access path
- TGA registered · Evrysdi
TGA label narrower than the PBS population
- Mar 2021Recommended · restricted
vs Nusinersen (Population 1); Best supportive care…
- Mar 2023Recommended · restricted
Comparator changed: Nusinersen (Population 1); Best supportive care (Populations 2 and 3)…
- Jul 2024Recommended · restricted
Comparator changed: nusinersen (populations 1 and 2); onasemnogene abeparvovec…
- Nov 2025Recommended · restricted
Comparator changed: nusinersen → risdiplam 0.75 mg/mL powder for oral liquid
- PBS listing · Authority Required
From the public summary
6.1 The PBAC recommended the listing of risdiplam 5 mg tablets (Evrysdi) for the treatment of spinal muscular atrophy (SMA), on the basis that it should be available only under special arrangements as a complex authority required (CAR) medicine. The PBAC noted the sponsor’s request for a Section 85 General Schedule listing, however, 21PSD · Nov 2025
6.2 The PBAC is satisfied that risdiplam tablets, for some patients, offer a more convenient alternative to the oral solution, due to their transportability and dispersibility properties.PSD · Nov 2025
5.9 As a Category 4 submission, the economic analysis has not been independently evaluated.PSD · Nov 2025
5.10 A cost-minimisation approach has been requested for the listing of RIS tablet compared with RIS oral liquid.PSD · Nov 2025
5.6 The submission claimed RIS tablet is non-inferior to RIS oral liquid in terms of effectiveness and safety. The Therapeutic Goods Administration’s Delegate’s Overview supported that the RIS tablet is bioequivalent to the RIS oral liquid.PSD · Nov 2025
5.7 The PBAC considered that the claim of non-inferior effectiveness was reasonable.PSD · Nov 2025
5.2 The PBAC noted and welcomed input from an individual (1), parent (1) and the National Paediatric Medicines Forum. Their comments described how a tablet formulation of risdiplam would provide some patients independence by simplifying dosing, and how travelling with tablets would be more practical than transporting a refrigerated glass bottle.PSD · Nov 2025
5.18 There are existing risk sharing arrangements (RSAs) in place for the current PBS listing of RIS oral liquid.PSD · Nov 2025
Cost-effectiveness
Cost-minimisation analysis; no ICER calculated by design
The submission proposed the equi-effective doses to be RIS 5 mg tablet once daily = RIS 5 mg oral liquid once daily. PSD · 2025
Decision context
PopulationPatients aged 2 years or older weighing 20 kg or more with genetically confirmed spinal muscular atrophy (5q homozygous SMN1 deletion or heterozygous deletion with pathogenic variant), including paediatric symptomatic Type I, II or IIIa SMA, and adults with childhood-onset SMA not receiving invasive permanent assisted ventilation.
Submission history
| Decided | Outcome | Comparator | ICER | Evidence |
|---|---|---|---|---|
| Nov 2025 | Recommended · restricted | risdiplam 0.75 mg/mL powder for oral liquid | — | Single-arm · Bioequivalence |
| Jul 2024 | Recommended · restricted | nusinersen | — | Single-arm · Event free survival |
| Mar 2023 | Recommended · restricted | nusinersen (populations 1 and 2); onasemnogene abeparvovec (population 3, effectively withdrawn) | — | Cost-minimisation · Cost-minimisation |
| Mar 2021 | Recommended · restricted | Nusinersen (Population 1); Best supportive care (Populations 2 and 3) | — | RCT · OS, EFS, motor function (BSID-III, CHOP-INTEND, HINE-2, MFM32, RULM, HFMSE, SMAIS) |
Clinical evidence
| Trial | Phase | N | Primary outcome | Status |
|---|---|---|---|---|
| RAINBOWFISH | Ph 2 | 26 | Percentage of Participants With Two Copies of the Survival Motor Neuron (SMN) 2 Gene (Excl… | active not recruiting |
| FIREFISH | Ph 2 | 62 | Part 1: Selected Part 2 Dose of Risdiplam | completed |
| SUNFISH | Ph 2 | 231 | Part 1: Selected Part 2 Dose of Risdiplam for Participants With a Body Weight (BW) of >/=2… | completed |
| BP42066 (NCT04718181) | Ph 1 | 131 | Part 1: Plasma Concentration of Risdiplam in Cohorts A and B | completed |
Consumer voice
Consumers highlighted that a tablet formulation of risdiplam would provide independence through simplified dosing and practical benefits for travel compared to refrigerated solutions. They emphasized that tablet size would be critical for swallowability and that both formulations should be available for equitable access.
a tablet formulation of risdiplam would provide some patients independence by simplifying dosing Consumer comments · PSD
Similar precedents
Regulatory · TGA
Label narrower than PBS population — PBAC restricts to age ≥2 years, weight ≥20 kg, genetically confirmed SMA, and excludes patients on invasive permanent ventilation.