FutibatinibLytgobi
Locally advanced or metastatic cholangiocarcinoma in patients with fibroblast growth factor receptor 2 (FGFR2) fusion or rearrangement that have progressed after at least one prior line of systemic therapy.
Decisions on record
- Meeting Nov 2025 Deferred Locally advanced or metastatic cholangiocarcinoma with FGFR2 genetic variant, previously treated no PSD
- Meeting Jul 2025 Not recommended Cholangiocarcinoma with FGFR2 fusion/rearrangement after prior systemic therapy
- Meeting Mar 2025 Not recommended Locally advanced or metastatic cholangiocarcinoma with FGFR2 fusion/rearrangement, after prior systemic therapy
Access path
- TGA registered · Lytgobi
TGA label equal than the PBS population
- Mar 2025Not recommended
vs FOLFOX chemotherapy (modified FOLFOX6)
- ↻ resubmittedNov 2025Recommended
Comparator changed: FOLFOX chemotherapy (modified FOLFOX6) → FOLFOX (modified FOLFOX 6…
- PBS listing · Authority Required
From the public summary
5.1 The PBAC did not recommended futibatinib for the treatment of patients with locally advanced or metastatic cholangiocarcinoma who have previously progressed on systemic therapy and have a fibroblast growth factor receptor 2 fusion or rearrangement. The PBAC considered the early re-entry resubmission did not adequately address the outstanding issues requested as part of its March 2025 PBAC consideration.PSD · Jul 2025
5.2 The PBAC recalled that it previously considered there is a high clinical need for more effective therapies for CAA and noted that consumer input received for the March 2025 and July 2025 meetings supported the availability of futibatinib.PSD · Jul 2025
model presented was likely not reflective of the The OS HR in the economic evaluation has been true cost effectiveness of futibatinib. The PBAC amended to 0.32, to align with the upper OS HR CI noted the economic model resulted in an limit from the March 2025 submission, claiming undiscounted life year gain of 2.48 over the 10 this value represents the highest plausible value year time horizon of the model and considered N for the HR given the observed …PSD · Jul 2025
The PBAC noted the economic model assumed a time horizon of 10 years with no convergence of The time horizon of the economic evaluation has OS modelled. The PBAC agreed with the been reduced from 10 years to 7 years.PSD · Jul 2025
4.7 The PBAC previously considered the clinical claim of superior comparative effectiveness versus the nominated comparator (FOLFOX) was reasonable; however the magnitude of effect is highly uncertain and likely overestimated due to differences 3Note that the results presented in this section are derived from analyses conducted by the applicant for the purposes of informing the PBAC and MSAC consideration.PSD · Jul 2025
OFFICIAL Public Summary Document – July 2025 PBAC Meeting with November 2025 Addendum and Out of Session Addendum in baseline characteristics (tumour site, FGFR2 status), less favourable HR in other indirect comparisons, and the unanchored MAIC (paragraph 6.65, futibatinib PSD, March 2025 PBAC meeting).PSD · Jul 2025
4.3 The Medical Oncology Group of Australia (MOGA) also expressed its support for the futibatinib submission. The PBAC noted that the MOGA presented a European Society for Medical Oncology Magnitude of Clinical Benefit Scale (ESMO-MCBS) for futibatinib of 2 (out of a maximum of 5, where 5 and 4 represent the grades with substantial improvement)1, on the basis of objective response rate in the FOENIX-CCA2 trial.PSD · Jul 2025
6.125 The submission considered that due to the small and identifiable population (through testing for FGFR2 fusions or rearrangements) and the minor cost to PBS budgets, no For more detail on PBAC’s view, see section 7 PBAC outcome.PSD · Mar 2025
Cost-effectiveness
Resubmission ICER presented in redacted range; previous submission base case was $95,000 to < $115,000 per QALY. Current resubmission targeted ICER of less than $75,000 per QALY.
Decision context
PopulationAdult patients with locally advanced or metastatic cholangiocarcinoma with FGFR2 fusion or rearrangement that have progressed after at least one prior line of systemic therapy.
Why it was knocked back
- Uncertain clinical benefit likely overestimated in MAIC, unanchored MAIC confers high risk of bias, conservative indirect comparisons (Paine 2022, Borad 2022) show less favourable HRs, economic model uncertainties persist despite revisions, ICER threshold expectations not met
Submission history
| Decided | Outcome | Comparator | ICER | Evidence |
|---|---|---|---|---|
| Nov 2025 | Recommended | FOLFOX (modified FOLFOX 6 chemotherapy) | $75k–95k | Single-arm · OS |
| Mar 2025 | Not recommended | FOLFOX chemotherapy (modified FOLFOX6) | — | Single-arm · OS |
Codependent tests
| Service | Biomarker | MSAC outcome | Year |
|---|---|---|---|
| Testing of tumour tissue to detect FGFR2 fusions or rearrangements in people with cholangiocarcinoma | FGFR2 fusions or rearrangements | deferred | 2025 |
Consumer voice
Rare Cancers Australia, Pancare Foundation, and the Liver Foundation highlighted the severity of cholangiocarcinoma, quality-of-life impacts from current chemotherapy, and benefits of futibatinib's oral administration for rural/remote patients. Cost and access barriers were identified as major concerns. The Medical Oncology Group of Australia supported the submission.
Rare Cancers Australia described the severity of CCA, trade-offs associated with side-effects of current chemotherapy options and quality of life, and the benefits associated with futibatinib's oral administration, particularly for rural and remote patients. Consumer comments · PSD
Similar precedents
Regulatory · TGA
Label equal than PBS population — Both specify adult patients with locally advanced/metastatic intrahepatic cholangiocarcinoma, FGFR2 fusion/rearrangement, progressed after ≥1 prior line.