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- Meeting Nov 2021 Withdrawn Cryopyrin associated periodic syndromes (CAPS) no PSD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 listing of canakinumab for the treatment of moderate to severe cryopyrin-associated periodic syndromes (CAPS), on the basis that it should be available only under special arrangements under Section 100.PSD · Nov 2017
7.2 The PBAC accepted that anakinra is the appropriate comparator. The PBAC did not accept the equi-effective doses proposed in the submission (anakinra 100 mg once daily for eight weeks and canakinumab 150 mg once every 8 weeks) noting that dose escalations for neither anakinra nor canakinumab were considered. The PBAC also considered it appropriate for the price of canakinumab to be adjusted to account for the likely administration costs for canakinumab.PSD · Nov 2017
6.26 The equi-effective doses were estimated in the submission as:PSD · Nov 2017
- canakinumab 150 mg once every 8 weeks = anakinra 100 mg once daily for eight weeks (5600 mg).PSD · Nov 2017
6.15 The submission did not make a direct clinical claim and instead provided a summary of results across the comparisons presented. The submission cited no statistically significant differences between canakinumab and tocilizumab for the ACR 30 Pedi response, while canakinumab was found to be better than adalimumab for this outcome.PSD · Mar 2015
While the results of indirect comparisons indicated that there are no statistically significant differences between canakinumab and these comparators for efficacy outcomes, there was no identified non-inferiority margin and importantly, there was heterogeneity in the populations as well as differences in designs and durations of the trials that limited the reliability of the analyses.PSD · Mar 2015
6.40 The submission noted that the anakinra Public Summary Document (November 2014) referred to the implementation of a capping arrangement to ensure PBS expenditure does not exceed agreed limits. The sponsor expects to be asked to sign up to the Deed of Agreement governing the CAPS market in the event that the canakinumab submission is successful. For more detail on PBAC’s view, see section 7 PBAC outcome.PSD · Nov 2017
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.