LumasiranOXLUMO
Primary hyperoxaluria type 1 (PH1) in patients with elevated urinary oxalate levels or clinical symptoms indicative of hyperoxaluria, as an addition to best supportive care.
Decision on record
- Meeting Mar 2025 Recommended Primary hyperoxaluria type 1 (PH1)
Access path
- TGA registered · OXLUMO
TGA label narrower than the PBS population
- Mar 2025Recommended · restricted
vs best supportive care (BSC)
- PBS listing · Authority Required
From the public summary
7.1 The PBAC recommended the listing of lumasiran for the treatment of primary hyperoxaluria type 1 (PH1). In making this recommendation, the PBAC accepted there is a high unmet clinical need for treatment options for patients with PH1, and that lumasiran is effective in reducing urinary oxalate and plasma oxalate and may also lead to a reduction in the utilisation of dialysis and liver-kidney transplantation.PSD · Mar 2025
7.2 The PBAC recognised the high unmet clinical need for treatment for PH1. The PBAC noted that PH1 is an ultra-rare disease with substantial impacts on the quality of life of patients and their families and caregivers.PSD · Mar 2025
6.55 The submission presented a modelled economic evaluation of lumasiran with BSC compared to BSC alone in patients with PH1. The economic evaluation was based on data from the three ILLUMINATE trials and included additional literature data. The economic analysis was presented as a cost-utility analysis (CUA) over a lifetime time horizon.PSD · Mar 2025
6.56 Table 10 presents a summary of the model structure and key inputs and rationale of the economic evaluation.PSD · Mar 2025
6.49 The ESC considered that the clinical claim of superior effectiveness compared with BSC alone was adequately supported by the clinical data presented in the submission.PSD · Mar 2025
However, the magnitude of benefit was uncertain due to the rarity of primary hyperoxaluria type 1 and the associated small number of patients included in thePSD · Mar 2025
6.2 The PBAC noted and welcomed the input from individuals (6), health care professionals (3) and organisations (4) via the Consumer Comments facility on the PBS website. Clinicians, individuals and their caregivers and supporters described PH1 as a devastating condition that severely impacts the physical and psychological wellbeing of individuals with PH1 and their supporting caregivers and family.PSD · Mar 2025
Comments emphasised that patients who develop ESKD experience an increased risk of cardiovascular issues and have a reduced life expectancy. Patients with PH1 also face significant emotional, psychological and cognitive challenges, which impact on their ability to succeed in school and hinder their ability to navigate adult life independently.PSD · Mar 2025
Cost-effectiveness
ICER not stated in the public summary document; appears to be commercially sensitive or redacted.
Decision context
PopulationPatients with primary hyperoxaluria type 1 confirmed by genetic testing, with either urinary oxalate ≥0.70 mmol/24 h/1.73 m² or urinary oxalate:creatinine ratio greater than upper limit of normal, or clinical symptoms such as nephrocalcinosis, renal stones, renal impairment, or systemic oxalosis; excluding those with prior liver transplant.
Submission history
| Decided | Outcome | Comparator | ICER | Evidence |
|---|---|---|---|---|
| Mar 2025 | Recommended · restricted | best supportive care (BSC) | — | RCT · Surrogate |
Clinical evidence
| Trial | Phase | N | Primary outcome | Status |
|---|---|---|---|---|
| ILLUMINATE-A | Ph 3 | 39 | Percent Change in 24-hour Urinary Oxalate Excretion Corrected for Body Surface Area (BSA) … | completed |
| ILLUMINATE-B | Ph 3 | 18 | Percentage Change in Spot Urinary Oxalate:Creatinine Ratio From Baseline to Month 6 | completed |
Consumer voice
Individuals, healthcare professionals, and organisations described PH1 as a devastating condition with severe physical and psychological impacts. They highlighted significant treatment burdens from current therapies and expressed strong support for lumasiran due to its potential to reduce oxalate levels, prevent kidney complications, and improve quality of life.
Clinicians, individuals and their caregivers and supporters described PH1 as a devastating condition that severely impacts the physical and psychological wellbeing of individuals with PH1 and their supporting caregivers and family. Consumer comments · PSD
Similar precedents
Regulatory · TGA
Label narrower than PBS population — PBAC restricts to patients with confirmed genetic testing and elevated urinary oxalate or clinical symptoms; TGA label covers all PH1 patients regardless.