RepotrectinibAugtyro
Treatment of adult patients with locally advanced (Stage IIIB) or metastatic (Stage IV) c-ROS proto-oncogene 1 (ROS1)-positive non-small cell lung cancer (NSCLC).
Decision on record
- Meeting May 2025 Recommended ROS1-positive NSCLC (Stage IIIB or IV)
Access path
- TGA registered · Augtyro
TGA label narrower than the PBS population
- May 2025Recommended · restricted
vs entrectinib
- PBS listing · Authority Required
From the public summary
7.1 The PBAC recommended the General Schedule, Authority Required (written/online PBS authorities system) PBS listing of repotrectinib for the treatment of adult patients with locally advanced (Stage IIIB) or metastatic (Stage IV) c-ROS proto-oncogene 1 (ROS1) positive non-small cell lung cancer (NSCLC).PSD · May 2025
7.2 The PBAC considered the equi-effective doses for the standard dosing regimen were:PSD · May 2025
6.42 The submission applied a cost minimisation approach (CMA) based on the clinical claim of non-inferior effectiveness and safety of repotrectinib and entrectinib. The ESC considered a CMA may be reasonable, given its view on the clinical claims.PSD · May 2025
6.43 The submission stated that the equi-effective doses was based on steady-state dosing, with 600 mg (once daily) for entrectinib and 320 mg (160 mg twice daily) for repotrectinib. The calculation was based on manufacturer recommended dosage, instead of trial-based dosage, due to data limitations. These doses are only correct for patients taking standard doses.PSD · May 2025
6.38 The submission described repotrectinib as non-inferior in terms of effectiveness compared to entrectinib and crizotinib. The evaluation considered the claim was uncertain and may not be adequately supported.PSD · May 2025
6.39 The ESC also considered the claim of non-inferior comparative effectiveness to entrectinib was uncertain due to the inherent limitations of the available data, however considered on balance that the claim may be reasonable.PSD · May 2025
6.3 The Lung Foundation Australia noted lung cancer is the leading cause of cancer deaths, the 4th leading cause of all death and the lowest survival rate compared to the most common cancers in Australia. It further noted the negative impact of lung cancer diagnosis on individuals’ mental health and wellbeing and the importance of new treatment options to improve prognosis.PSD · May 2025
6.5 The Thoracic Oncology Group of Australasia (TOGA) noted acquired resistance mutations develop in at least 50% of patients treated with entrectinib and crizotinib, limiting the durability of response. TOGA noted repotrectinib has clinical activity against ROS1–positive NSCLC, including the most common resistance mutations, detected in approximately a third of ROS1 cases.PSD · May 2025
Cost-effectiveness
Cost-minimisation analysis performed; no ICER calculated by design.
The submission applied a cost minimisation approach (CMA) based on the clinical claim of non-inferior effectiveness and safety of repotrectinib and entrectinib. PSD · 2025
Decision context
PopulationAdult patients with locally advanced (Stage IIIB) or metastatic (Stage IV) ROS1-positive non-squamous NSCLC, with WHO performance status ≤2, with evidence of ROS1 gene rearrangement, either TKI-naïve or intolerant to prior ROS1 receptor tyrosine kinase inhibitors.
Submission history
| Decided | Outcome | Comparator | ICER | Evidence |
|---|---|---|---|---|
| May 2025 | Recommended · restricted | entrectinib | — | Single-arm · ORR |
Clinical evidence
| Trial | Phase | N | Primary outcome | Status |
|---|---|---|---|---|
| TRIDENT-1 | Ph 1, PHASE2 | 500 | Dose limiting toxicities (DLTs) (Phase 1) | recruiting |
Consumer voice
Eight contributors (2 individuals, 1 family member/carer, 1 healthcare professional, and 4 organisations) submitted supportive comments about PBS listing for repotrectinib. They highlighted the need for additional therapeutic options as disease eventually progresses, the potency and brain penetration benefits of repotrectinib, and the significant burden of lung cancer on mental health and wellbein
while their current treatment is effective, the disease eventually progresses, and an alternate option is required for the opportunity to prolong life Consumer comments · PSD
Similar precedents
Regulatory · TGA
Label narrower than PBS population — PBAC restricts to first-line, non-squamous histology, performance status ≤2, and documented ROS1 rearrangement; TGA label permits all ROS1-positive NSCLC as monotherapy.