PalovaroteneSohonos
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).
Decision on record
- Meeting Nov 2024 Recommended Fibrodysplasia ossificans progressiva
Access path
- TGA registered · Sohonos
TGA label narrower than the PBS population
- Nov 2024Recommended
vs standard of care (SoC) comprising symptomatic treatment of…
- PBS listing · Authority Required
From the public summary
7.1 The PBAC recommended the listing of palovarotene for the treatment of fibrodysplasia ossificans progressiva (FOP). The PBAC recognised the high and urgent clinical need for treatments for FOP, which is an ultra-rare disease with very substantial impacts on quality of life for patients and their carers.PSD · Nov 2024
The PBAC also advised the clinical trial data and uncertain economic modelling meant the rule of rescue claim that palovarotene provided a worthwhile clinical 34PSD · Nov 2024
6.57 The submission presented a stepped economic evaluation based on the MOVE study.PSD · Nov 2024
The type of economic evaluation presented was a cost-utility analysis.PSD · Nov 2024
6.52 The submission described palovarotene (in combination with SoC) as having superior efficacy and inferior safety compared to SoC.PSD · Nov 2024
6.53 The therapeutic conclusion of efficacy is supported by lower annualised HO in the PBS relevant subgroup. This was consistent with the view of the TGA ACM, which concluded that the results of the post-hoc comparison of annualised new HO demonstrated meaningful benefit. The ESC agreed with the commentary that the magnitude of this benefit was highly uncertain as:PSD · Nov 2024
6.2 The PBAC noted and welcomed the input from 2 organisations (FOP Australia and International FOP Association) via the Consumer Comments facility on the PBS website. The PBAC noted that the comments from FOP Australia reflected input from 12 individuals including people living with FOP, their friends and family members. The comments described the experience of living with FOP, and the challenges and priorities for people with FOP and their families.PSD · Nov 2024
The bone growth of FOP leads to a wide range of complications, including death. 2. Anything that can reduce or delay any amount of bone growth is of huge value to patients, including those at advanced stages, as even tiny amounts of bone can be extremely functionally significant. 8PSD · Nov 2024
Cost-effectiveness
The document does not provide an explicit numeric ICER value. The economic model section appears to be redacted or omitted from the provided text.
Decision context
PopulationPatients with FOP aged ≥8 years (females) or ≥10 years (males), confirmed by genetic testing for pathogenic ACVR1 gene mutation.
Submission history
| Decided | Outcome | Comparator | ICER | Evidence |
|---|---|---|---|---|
| Nov 2024 | Recommended | standard of care (SoC) comprising symptomatic treatment of flare-ups | $1.05m | RCT · Annualised change in new HO volume |
Clinical evidence
| Trial | Phase | N | Primary outcome | Status |
|---|---|---|---|---|
| MOVE | Ph 3 | 107 | Annualized New Heterotopic Ossification (HO) | completed |
Consumer voice
Two organisations (FOP Australia and International FOP Association) provided input reflecting experiences of 12 individuals with FOP and their families. The comments emphasised living with unpredictable bone growth causing progressive loss of independence, and the value of any intervention that reduces or delays bone growth, even minimally.
Living with FOP means living with the constant threat of unpredictable bone growth, causing progressive and painful loss of independence and mobility. Consumer comments · PSD
Similar precedents
Regulatory · TGA
Label narrower than PBS population — PBAC restricts to first-line use and requires genetic confirmation of ACVR1 mutation; TGA label lacks these requirements.
Listed in law
PBS listings commencing 1 Sep 2025 were made by these 3 determinations under the National Health Act 1953. A determination covers every listing that commenced that day, so this is the legal instrument in force for the date — not a document naming this medicine. Open it and check.
| Determination | Made | Status | |
|---|---|---|---|
| PB 100 of 2025 | 29 Aug 2025 | Repealed | PDF ↗ |
| PB 94 of 2025 | 29 Aug 2025 | Repealed | PDF ↗ |
| PB 95 of 2025 | 29 Aug 2025 | Repealed | PDF ↗ |