Cabozantinib
Treatment of adult patients with unresectable locally advanced or metastatic well- or moderately-differentiated pancreatic neuroendocrine tumour (pNET) or extra-pancreatic neuroendocrine tumour (epNET) after prior systemic therapy.
Decisions on record
- Meeting Mar 2026 Noted Clear cell variant renal cell carcinoma no PSD
- Meeting Jul 2025 Recommended Unresectable/metastatic well-differentiated pancreatic or extra-pancreatic neuroendocrine tumours after prior systemic therapy
- Meeting Mar 2024 Recommended Renal cell carcinoma (RCC)
- Meeting Mar 2024 Recommended Renal cell carcinoma (RCC) - non-clear cell variant
- Meeting Mar 2024 Recommended Differentiated thyroid cancer (DTC)
- Meeting Nov 2023 Not recommended Locally advanced or metastatic differentiated thyroid cancer (radioactive iodine refractory/ineligible)
- Meeting Nov 2020 Recommended Renal cell carcinoma (RCC)
- Meeting Mar 2020 Not recommended Cabozantinib is indicated for the treatment of advanced renal cell carcinoma (RCC): in treatment-naive adults with
3 earlier decisions
- Jul 2019 Not recommended Hepatocellular carcinoma
- Mar 2019 Not recommended Renal cell carcinoma (RCC)
- Dec 2017 Recommended Clear cell variant renal cell carcinoma (RCC)
Access path
- Dec 2017Recommended
vs everolimus
- Mar 2019Not recommended
Comparator changed: everolimus → sunitinib
- Jul 2019Not recommended
Comparator changed: sunitinib → best supportive care (BSC)
- Mar 2020Not recommended
Comparator changed: best supportive care (BSC) → sunitinib
- ↻ resubmittedNov 2020Recommended · restricted
- Nov 2023Not recommended
Comparator changed: sunitinib → best supportive care (BSC)
- ↻ resubmittedMar 2024Recommended · restricted
Comparator changed: best supportive care (BSC) → Best supportive care (placebo)
- Mar 2024Recommended
Comparator changed: best supportive care (BSC) → Best supportive care (placebo)
- Mar 2024Recommended
Comparator changed: best supportive care (BSC) → Best supportive care (placebo)
- Jul 2025Recommended
Comparator changed: pembrolizumab plus lenvatinib (PEM+LEN) → Sunitinib (pNET…
- PBS listing · Authority Required
From the public summary
7.1 The PBAC recommended cabozantinib for the treatment of patients with unresectable or metastatic, well-differentiated extra-pancreatic (epNET) or pancreatic (pNET) neuroendocrine tumours who have progressed on at least one prior systemic therapy other than a somatostatin analogue (SSA).PSD · Jul 2025
7.2 The PBAC welcomed advice from consumers, clinicians and organisations (via the consumer comments and the sponsor hearing) that there is a clinical need for additional treatments for patients with NETs. The PBAC noted that NETs are relatively rare, indolent at onset and progress slowly, with a variable prognosis depending on location, stage and grade.PSD · Jul 2025
6.62 The submission conducted two types of economic evaluations:PSD · Jul 2025
• Cost utility analyses (CUAs), comprising modelled evaluations based on direct evidence from the pivotal CABINET trial, in the fourth line setting, where the submission made a claim of superior efficacy versus BSC for both the pNET and epNET populations; andPSD · Jul 2025
- Compared to chemotherapy, comparable effectiveness, different but manageable safety profile.PSD · Jul 2025
- Compared to BSC, superior effectiveness and inferior but manageable safety. Source: Table 1-1, p18 of the submission.PSD · Jul 2025
6.2 The PBAC noted and welcomed the input from individuals (2), health care professionals (2) and organisations (Rare Cancers Australia, NeuroEndocrine Cancer Australia, and the Medical Oncology Group of Australia (MOGA)) via the Consumer Comments facility on the PBS website. The comments described a range of benefits of treatment with cabozantinib including improved progression free survival (PFS), 11PSD · Jul 2025
One individual indicated that despite trying eight therapies and participating in trials, they experienced disease progression or serious side effects, however since starting cabozantinib their symptoms had reduced significantly in terms of both frequency and intensity. The PBAC noted that this advice was supportive of the evidence provided in the submission.PSD · Jul 2025
Cost-effectiveness
No numeric ICER values stated in the public summary document. Cost-minimisation analysis requested for third-line pNET vs sunitinib and third-line epNET vs chemotherapy; cost-effectiveness analysis vs placebo/BSC requested for fourth-line settings, but specific ICER values are not disclosed in this PSD.
The PBAC considered that CBZ+NIVO had a different safety profile compared to NIVO+IPI. PBAC · 2024
Decision context
PopulationAdult patients (aged ≥18 years) with unresectable locally advanced or metastatic well- or moderately-differentiated pNET or epNET who have progressive disease on prior systemic therapy or developed intolerance necessitating treatment withdrawal, with WHO grade 0, 1 or 2 disease.
Why it was knocked back
- Unclear place in therapy across requested populations; inconsistent restrictions compared to nominated comparators (sunitinib, everolimus); inclusion of locally advanced and moderately-differentiated disease with inadequate efficacy data in these subgroups; absence of ECOG performance status criterion; lack of requirement for prior progression on specific systemic therapies other than SSA; proposed solo therapy requirement inconsistent with CABINET trial protocol which permitted concomitant SSA use; WHO grade 0, 1, or 2 criterion does not align with WHO classification system; lack of one-course-per-lifetime restriction; potential need for flow-on changes to existing SSA listings not adequately addressed
Submission history
| Decided | Outcome | Comparator | ICER | Evidence |
|---|---|---|---|---|
| Jul 2025 | Recommended | Sunitinib (pNET third-line), chemotherapy (epNET third-line), best supportive care (fourth-line both populations) | — | RCT · PFS |
| Mar 2024 | Recommended · restricted | Best supportive care (placebo) | $55k–75k | RCT · PFS |
| Mar 2024 | Recommended | sunitinib | — | RCT · PFS, ORR, OS |
| Mar 2024 | Recommended | pembrolizumab plus lenvatinib (PEM+LEN) | — | RCT | Single-arm | Registry | Cost-minimisation · PFS, ORR, OS and safety |
| Nov 2023 | Not recommended | best supportive care (BSC) | — | RCT · PFS |
| Nov 2020 | Recommended · restricted | sunitinib | — | RCT · PFS |
| Mar 2020 | Not recommended | sunitinib | — | RCT · PFS |
| Jul 2019 | Not recommended | best supportive care (BSC) | — | RCT · OS |
| Mar 2019 | Not recommended | sunitinib | — | RCT · PFS |
| Dec 2017 | Recommended | everolimus | — | RCT · PFS |
Clinical evidence
Consumer voice
Consumer input from 2 individuals, 2 healthcare professionals, and 3 organisations described benefits of cabozantinib including improved progression-free survival, quality of life, and reduced cancer-related symptoms. Comments highlighted limited treatment options for neuroendocrine cancers and advocated for broader indication coverage and first-line availability.
One individual indicated that despite trying eight therapies and participating in trials, they experienced disease progression or serious side effects, however since starting cabozantinib their symptoms had reduced significantly in both frequency and intensity. Consumer comments · PSD
Similar precedents
Regulatory · TGA
Label narrower than PBS population — PBAC restricts to moderately-differentiated tumours and WHO grade 0-1-2 disease; TGA label includes all well-differentiated without these constraints.