PBAC's latest decision on Risdiplam: Recommended (2025). Considered for 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.
PBAC outcome
Recommended
Authority Required
ICER (AUD/QALY)
Cost-min
cost-minimisation analysis
Submissions
4
first 2021
Submissions
4
2021 → 2025
Eligible population
Patients 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.
Therapy area
Rare disease
Line of therapy
Any
Evidence base
Single-arm
Primary endpoint
Bioequivalence
Key trials
Study BP42066 (NCT04718181)
Comparator
risdiplam 0.75 mg/mL powder for oral liquid
Economic model
Cost-minimisation
ICER note
Cost-minimisation analysis; no ICER calculated by design
Submission history
Mar 2021: Recommended with restriction · Authority Required
Mar 2023: Recommended with restriction · Authority Required
Jul 2024: Recommended with restriction · Authority Required