TeprotumumabTepezza
Treatment of active, moderate-to-severe thyroid eye disease (TED).
Decisions on record
- Meeting May 2025 Recommended Active, moderate-to-severe thyroid eye disease no PSD
- Meeting Mar 2025 Not recommended Thyroid eye disease (TED), active moderate-to-severe
Access path
- TGA registered · Tepezza
TGA label narrower than the PBS population
- May 2025Recommended · restricted
vs IV methylprednisolone (IVMP) with or without mycophenolate…
- PBS listing · Authority Required
From the public summary
7.1 The PBAC did not recommend teprotumumab for the treatment of active, moderate- to-severe (MS) thyroid eye disease (TED). The PBAC considered there was a high clinical need in the requested patient population, and the evidence presented demonstrated that teprotumumab was more effective in reducing proptosis and diplopia compared to the current standard of care (SoC), which may lead to a reduced utilisation of eye surgery.PSD · Mar 2025
The PBAC considered that revision to model inputs and a price reduction would be required to address residual uncertainty and achieve a cost-effective listing, together with a risk sharing arrangement.PSD · Mar 2025
6.62 Three comparisons were presented in the submission’s economic analysis: • Teprotumumab vs IVMP in the 1L setting • Teprotumumab vs IVMP + MMF in the 1L setting • Teprotumumab vs tocilizumab in the 2L setting 30 OFFICIALPSD · Mar 2025
OFFICIAL Public Summary Document – March 2025 PBAC Meeting with May 2025 Addendum Neither comparison in the 1L setting reflects current practice, where MMF would be used in patients with minimal response after three infusions of IVMP, and orbital radiotherapy at 12 weeks if there is ongoing severe disease.11 The evaluation considered that due to the lack of clinical evidence presented in the submission to support the comparison of teprotumumab to IVMP + …PSD · Mar 2025
6.52 The submission described teprotumumab as superior in terms of effectiveness compared to SoC comprising of IVMP with or without MMF in the 1L setting and tocilizumab in the 2L setting.PSD · Mar 2025
6.53 The ESC considered the claim of superior efficacy for teprotumumab over IVMP with or without MMF was uncertain. The presented MAIC had a number of issues that resulted in considerable uncertainty in the estimate of effectiveness of teprotumumab vs IVMP.PSD · Mar 2025
6.3 The health care professionals (HCPs) noted there was an unmet clinical need for effective and disease specific therapies for patients with TED in Australia. The HCPs commented on the clinical benefits associated with teprotumumab over current SOC, noting that teprotumumab targeted the underlying pathophysiology of TED, and that based on the clinical trial evidence, teprotumumab was associated with significant improvements in CAS, proptosis, and …PSD · Mar 2025
The HCPs also considered treatment with teprotumumab would be associated with a number of quality-of-life benefits and enable patients to better engage in their routine daily activities. The HCPs noted that teprotumumab was generally well-tolerated and considered that side effects could be managed through careful screening and monitoring.PSD · Mar 2025
Cost-effectiveness
ICER values appear to be redacted from the public summary document.
Decision context
PopulationAdults aged 18 years or older with active, moderate-to-severe thyroid eye disease, defined as clinical activity score (CAS) ≥3 for the most severely affected eye and two or more of: proptosis ≥3 mm above normal, lid retraction ≥2 mm, moderate-to-severe soft-tissue involvement, or inconstant/constant diplopia.
Submission history
| Decided | Outcome | Comparator | ICER | Evidence |
|---|---|---|---|---|
| May 2025 | Recommended · restricted | IV methylprednisolone (IVMP) with or without mycophenolate mofetil (MMF) in first-line setting; tocilizumab in second-li | — | RCT · Proptosis and diplopia |
Clinical evidence
| Trial | Phase | N | Primary outcome | Status |
|---|---|---|---|---|
| OPTIC-X | Ph 3 | 51 | Percentage of Participants With a ≥ 2 mm Reduction From Baseline in the Study Eye Without … | completed |
Consumer voice
Healthcare professionals highlighted unmet clinical need for teprotumumab in TED, noting superior efficacy over current standard of care with quality-of-life benefits. TED patients emphasized the high disease burden and significant impact on daily functioning, including work capacity and ocular symptoms.
teprotumumab targeted the underlying pathophysiology of TED, and that based on the clinical trial evidence, teprotumumab was associated with significant improvements in CAS, proptosis, and diplopia versus SOC Consumer comments · PSD
Similar precedents
Regulatory · TGA
Label narrower than PBS population — PBAC specifies active disease with defined CAS ≥3 and clinical criteria; TGA label permits moderate-to-severe without activity requirement.