PBAC's latest decision on Palovarotene: Recommended with restriction (2024). Considered for Chronic treatment of fibrodysplasia ossificans progressiva (FOP) to reduce the formation of heterotopic ossification, and flare-up treatment of FOP in patients aged ≥8 years (females) or ≥10 years (males).
PBAC outcome
Recommended with restriction
Authority Required
ICER (AUD/QALY)
Redacted
commercial-in-confidence
Submissions
1
first 2024
Submissions
1
2024 → 2024
Eligible population
Patients with FOP aged ≥8 years (females) or ≥10 years (males), confirmed by genetic testing for pathogenic ACVR1 gene mutation.
Therapy area
Rare disease
Line of therapy
First-line
Evidence base
RCT
Primary endpoint
Annualised change in new HO volume
Pivotal trial size
99 patients
Key trials
MOVE
Comparator
standard of care (SoC) comprising symptomatic treatment of flare-ups
Economic model
CUA
ICER note
The document does not provide an explicit numeric ICER value. The economic model section appears to be redacted or omitted from the provided text.