IptacopanFabhalta
Treatment of adults with complement 3 glomerulopathy (C3G) with either native kidneys or disease recurrence following a kidney transplant.
Decisions on record
- Meeting Nov 2025 Not recommended Complement 3 glomerulopathy with native kidneys or disease recurrence following kidney transplant
- Meeting Jul 2024 Recommended Paroxysmal nocturnal hemoglobinuria with inadequate response to C5 inhibitor
Access path
- TGA registered · Fabhalta
TGA label narrower than the PBS population
- Jul 2024Recommended
vs pegcetacoplan
- Nov 2025Not recommended
Comparator changed: pegcetacoplan → Standard of care
From the public summary
7.1 The PBAC did not recommend iptacopan for treatment of complement 3 glomerulopathy (C3G). The PBAC considered that there is a high clinical need for treatments for C3G, which is a condition that impacts relatively young patients and causes decline in kidney function. The PBAC considered it is possible that iptacopan provides a clinical benefit for some patients in terms of slowing progression to end- stage kidney disease.PSD · Nov 2025
The PBAC considered that the modelled outcomes relating to avoidance of long-term dialysis and transplant were improbable and highly uncertain due to the limited clinical data, reliance on surrogate outcomes, and unsupported assumptions in the economic model. The PBAC considered that the cost-effectiveness of iptacopan had not been established as the ICER was extremely high at the requested price despite optimistic inputs to the economic model.PSD · Nov 2025
6.66 The submission presented a stepped economic evaluation of iptacopan compared to placebo for the treatment of C3G in adult patients with native kidneys. The economic evaluation was based on the APPEAR-C3G trial, X2202 phase 2 study and the B12001B extension study, with additional modelled data. The economic evaluation was presented as a cost-effectiveness/cost-utility analysis.PSD · Nov 2025
6.67 The submission also presented a scenario analysis assessing the cost effectiveness of iptacopan compared to placebo in post-transplant patients who experience disease recurrence.PSD · Nov 2025
6.60 The submission described iptacopan as superior in terms of efficacy compared to standard of care in patients with C3G who have native kidneys. The evaluation considered this claim may be reasonable in terms of short-term reductions in proteinuria but was inadequately supported in terms of changes in eGFR and longer- term reductions in the risk of progression to end-stage kidney disease.PSD · Nov 2025
6.61 The submission described iptacopan as inferior in terms of safety compared to standard of care in patients with C3G who have native kidneys. The ESC agreed with the evaluation that this claim was reasonable.PSD · Nov 2025
6.5 The PBAC noted and welcomed the input from individuals (2), health care professionals (14) and organisations (4) via the Office of Health Technology Assessment Consultation Hub. The inputs from health professionals described C3G as rare and complex to diagnose, with most individuals (50%) experiencing progression to ESKF within 10 years of diagnosis, and high rates of recurrence after kidney transplant (60-90%).PSD · Nov 2025
The input from individuals with C3G noted the impact of chronic kidney disease, relapses and treatment burden on their ability to participate in daily activities and their mental health.PSD · Nov 2025
Cost-effectiveness
ICER value not stated in the public text; economic analysis was performed but specific ICER figure is not published in this PSD.
Decision context
PopulationAdults aged ≥18 years with C3G (complement 3 glomerulopathy) with either native kidneys or disease recurrence following a kidney transplant. For native kidney patients: eGFR ≥30 mL/min/1.73 m², UPCR ≥1.0 g/g (113 mg/mmol), stabilised on ACE inhibitor or ARB unless contraindicated/intolerant. For post-transplant patients: recurrent C3G confirmed by kidney biopsy with eGFR ≥30 mL/min/1.73 m² and stable immunosuppression.
Submission history
| Decided | Outcome | Comparator | ICER | Evidence |
|---|---|---|---|---|
| Nov 2025 | Not recommended | Standard of care | — | RCT · Surrogate |
| Jul 2024 | Recommended | pegcetacoplan | — | Meta-analysis · Other |
Clinical evidence
| Trial | Phase | N | Primary outcome | Status |
|---|---|---|---|---|
| APPLY-PNH | Ph 3 | 97 | Marginal Proportion (Expressed as Percentages) of Participants With Sustained Increase in … | completed |
| APPOINT-PNH | Ph 3 | 40 | Marginal Proportion (Expressed as Percentage) of Participants With Sustained Increase in H… | completed |
| APPEAR-C3G | Ph 3 | 98 | Adult cohort: Log-transformed ratio to baseline in UPCR (sampled from a 24-hour urine coll… | recruiting |
Consumer voice
Health professionals (14), individuals with C3G (2), and organisations (4) provided input highlighting C3G as rare and complex with high progression to end-stage kidney disease and transplant recurrence rates. Consumer and specialist groups emphasised the significant symptom burden, treatment challenges, unmet therapeutic need, and substantial impact on patients, families, and healthcare systems.
Input highlighted the unmet need for effective therapies to treat the underlying pathophysiology of C3G and described iptacopan's benefits as: potential increased effectiveness compared to current treatments; reduced immunosuppressive side-effects; reduced morbidity and excess mortality associated with dialysis; reduced healthcare costs associated with dialysis; reduced demand for donor organs for transplant; and extending transplant survival in patients with recurrent C3G. Consumer comments · PSD
Similar precedents
Regulatory · TGA
Label narrower than PBS population — PBAC restricts to eGFR ≥30, UPCR ≥1.0, and RAS inhibitor use/contraindication status; TGA label specifies no such thresholds.