PegcetacoplanEmpaveli
Treatment of patients aged 12 years and older with complement 3 glomerulopathy (C3G) or primary immune complex membranoproliferative glomerulonephritis (IC-MPGN) to reduce proteinuria and stabilise or slow decline in estimated glomerular filtration rate.
Decisions on record
- Meeting Mar 2026 Not recommended Complement 3 glomerulopathy (C3G) or primary immune complex membranoproliferative glomerulonephritis (IC-MPGN)
- Meeting Nov 2025 Recommended Bilateral geographic atrophy or dry AMD secondary to age-related macular degeneration with intact fovea in treated eye
- Meeting May 2025 Recommended Paroxysmal nocturnal haemoglobinuria (treatment-naïve or C5 inhibitor treated)
- Meeting Mar 2024 Withdrawn Geographic atrophy secondary to age-related macular degeneration no PSD
- Meeting Jul 2022 Recommended Paroxysmal nocturnal haemoglobinuria with inadequate response or intolerance to C5 inhibitor treatment
- Meeting Mar 2022 Not recommended Paroxysmal nocturnal haemoglobinuria
Access path
- TGA registered · Empaveli
TGA label narrower than the PBS population
- Mar 2022Recommended · restricted
vs eculizumab and ravulizumab
- Jul 2022Recommended · restricted
Comparator changed: eculizumab and ravulizumab → ravulizumab
- May 2025Recommended · restricted
- Nov 2025Recommended · restricted
Comparator changed: ravulizumab → best supportive care
- Mar 2026Not recommended
Comparator changed: best supportive care → standard of care consisting of RAAS blockade…
From the public summary
7.1 The PBAC recommended the listing of pegcetacoplan, on the basis that it should be available as a General Schedule listing for the treatment of geographic atrophy (GA) secondary to age-related macular degeneration (AMD). The Committee acknowledged the high clinical need for treatments for GA, which is a leading cause of irreversible vision loss in older Australians.PSD · Nov 2025
OFFICIAL Public Summary Document – November 2025 PBAC Meeting transforming microperimetry outcomes to best-corrected visual acuity (BCVA). The PBAC considered the cost-effectiveness analysis would be more reliable with these alternative parameters and an incremental cost-effectiveness ratio (ICER) in the order of $55,000 to < $75,000 per QALY gained would be acceptable.PSD · Nov 2025
6.42 The submission presented a modelled economic evaluation based on direct randomised trials, DERBY and OAKS. The economic model assessed pegcetacoplan compared to BSC in patients with GA secondary to AMD for patients with non-subfoveal lesions in their best seeing eye. The evaluation considered that the clinical claim of superior effectiveness for pegcetacoplan compared to BSC, in patients with non-subfoveal lesions (i.e.PSD · Nov 2025
This argument was also supported by the sponsor hearing (see paragraph 6.1).PSD · Nov 2025
6.38 The submission described pegcetacoplan as superior in terms of effectiveness compared to sham injection. The ESC agreed with the evaluation that this claim was adequately supported but the magnitude of the benefit was uncertain.PSD · Nov 2025
6.39 The submission described pegcetacoplan as manageable in terms of safety compared to sham. This claim was not an appropriate safety claim as the submission did not make a comparative safety claim for pegcetacoplan compared to sham. Based on the presented evidence, pegcetacoplan has inferior safety compared to sham injection with a higher rate of adverse events related to treatment (12.1% vs. 3.9% in DERBY) and a higher rate of nAMD (7.5% vs.PSD · Nov 2025
6.2 The PBAC noted and welcomed the input from health care professionals (6), health -care related organisations (3), consumer groups (1) and individuals (69) via the Office of Health Technology Assessment Consultation Hub.PSD · Nov 2025
6.3 The PBAC noted feedback from prospective consumers who would like to access the medicine to treat their own health condition which described the significant cost barriers associated with access to pegcetacoplan especially for those at advanced age.PSD · Nov 2025
Cost-effectiveness
ICER value not stated in the public text; economic model details are not disclosed in this PSD.
The ESC agreed with the evaluation that, while pegcetacoplan may slow the progression of GA lesion growth, it was highly uncertain how this will translate into a delay in loss of visual acuity for patients. PBAC · 2025
Decision context
PopulationPatients aged 12 years and older with C3G or primary IC-MPGN with native kidneys or disease recurrence following a kidney transplant, with eGFR ≥30 mL/min/1.73 m² and not undergoing dialysis, weighing at least 30 kg.
Submission history
| Decided | Outcome | Comparator | ICER | Evidence |
|---|---|---|---|---|
| Mar 2026 | Not recommended | standard of care consisting of RAAS blockade (ACEI/ARB), SGLT2i and lifestyle measures, with or without immunosuppressiv | — | RCT |
| Nov 2025 | Recommended · restricted | best supportive care | — | RCT · Surrogate |
| May 2025 | Recommended · restricted | ravulizumab | — | Cost-minimisation · Cost-minimisation |
| Jul 2022 | Recommended · restricted | ravulizumab | — | Cost-minimisation · Cost-minimisation |
| Mar 2022 | Recommended · restricted | eculizumab and ravulizumab | — | RCT · Other |
Clinical evidence
| Trial | Phase | N | Primary outcome | Status |
|---|---|---|---|---|
| GALE | Ph 3 | 792 | Incidence and severity of ocular and systemic adverse events | completed |
Consumer voice
Consumers and healthcare professionals highlighted the significant quality-of-life benefits of pegcetacoplan in slowing geographic atrophy progression and preserving eyesight, while emphasizing substantial financial barriers to private prescription access and concerns about PBS subsidisation limiting treatment to one eye only.
The PBAC acknowledged the lived experience of individual consumers using pegcetacoplan under private prescription, and the benefits associated with slowing progression of GA and preservation of existing eyesight, as these outcomes most directly contributed to maintenance of quality of life and independence. Consumer comments · PSD
Similar precedents
Regulatory · TGA
Label narrower than PBS population — PBAC restricts to non-subfoveal GA with fellow eye subfoveal involvement; TGA label lacks fellow eye requirement and specificity.