CanakinumabIlaris
Treatment of paediatric colchicine-resistant or intolerant familial Mediterranean fever (FMF) patients who continue canakinumab treatment into adulthood (provided they initiated canakinumab treatment before turning 18 years of age).
Decisions on record
- Meeting Mar 2026 Recommended Colchicine-resistant Familial Mediterranean Fever (crFMF) in adults and children aged 2 years and older
- Meeting Nov 2021 Withdrawn Cryopyrin associated periodic syndromes (CAPS) no PSD
- Meeting Nov 2017 Recommended Cryopyrin associated periodic syndromes (CAPS)
- Meeting Mar 2015 Recommended Systemic juvenile idiopathic arthritis
Access path
- TGA registered · Ilaris
TGA label narrower than the PBS population
- Mar 2015Recommended · restricted
vs tocilizumab
- Nov 2017Recommended · restricted
Comparator changed: tocilizumab → anakinra
- Mar 2026Recommended · restricted
Comparator changed: anakinra → best supportive care (colchicine at maximum tolerated…
- PBS listing · Authority Required
From the public summary
7.1 The PBAC recommended the Section 100 (Highly Specialised Drugs Program) listing of canakinumab for the treatment of treatment of colchicine-resistant or intolerant familial Mediterranean fever (FMF).PSD · Mar 2026
The PBAC considered that a risk sharing arrangement would be required to manage the uncertainty associated with the long-term clinical benefit for canakinumab and the uncertain dosing and duration of therapy.PSD · Mar 2026
6.42 The submission presented a modelled economic evaluation of canakinumab plus BSC versus BSC alone for the treatment of paediatric patients with FMF who are intolerant or resistant to colchicine. The economic evaluation was based on the results of the CLUSTER trial with additional modelled data. The economic evaluation was presented as a stepped cost-effectiveness/cost-utility analysis, based on a Monte Carlo microsimulation model.PSD · Mar 2026
Table 7: Summary of model structure, key inputs and rationale Component Summary Treatments Canakinumab plus BSC versus BSC Time horizon 70 years in the base case versus up to 112 weeks of follow-up in the CLUSTER trial.PSD · Mar 2026
effective than BSC alone, with inferior but tolerable safety. Source: Table 1.1, p29 of the submission.PSD · Mar 2026
Abbreviations: BSC, best supportive care; FMF, familial Mediterranean feverPSD · Mar 2026
6.76 The ESC noted that the PBAC may wish to consider the need for a Risk Sharing Arrangement to mitigate the risk of 1) increasing numbers of colchicine intolerant patients given the availability of a PBS-listed alternative, 2) use of higher and more frequent dosing of canakinumab than estimated, and 3) the cost to the Government given the uncertainty around the incidence and prevalence of FMF. For more detail on PBAC’s view, see section 7 PBAC outcome.PSD · Mar 2026
Cost-effectiveness
ICER redacted as commercial-in-confidence; only published and effective prices are redacted in the document
Decision context
PopulationPaediatric patients (aged 2 to <18 years at treatment initiation) with colchicine-resistant or intolerant FMF, confirmed by Tel-Hashomer criteria and at least one known MEFV gene exon 10 pathogenic variant, with evidence of active disease (at least one febrile attack per month for ≥3 months despite maximal colchicine, or persistently elevated CRP/serum amyloid A, or documented colchicine contraindication/intolerance).
Submission history
| Decided | Outcome | Comparator | ICER | Evidence |
|---|---|---|---|---|
| Mar 2026 | Recommended · restricted | best supportive care (colchicine at maximum tolerated dose, NSAIDs, and oral corticosteroids) | — | RCT |
| Nov 2017 | Recommended · restricted | anakinra | — | Meta-analysis · Disease flare |
| Mar 2015 | Recommended · restricted | tocilizumab | — | RCT · ACR Pedi 30 response |
Similar precedents
Regulatory · TGA
Label narrower than PBS population — PBAC restricted to moderate to severe CAPS; TGA label does not specify severity threshold.