Pegunigalsidase AlfaELFABRIO
Treatment of Fabry disease in adult patients with end-organ damage (renal, cardiac, cerebrovascular disease, or uncontrolled chronic pain).
Decision on record
- Meeting Jul 2025 Recommended Fabry disease
Access path
- TGA registered · ELFABRIO
TGA label narrower than the PBS population
- Jul 2025Recommended · restricted
vs migalastat
- PBS listing · Authority Required
From the public summary
7.1 The PBAC recommended the listing of pegunigalsidase alfa for the treatment of Fabry Disease (FD) as an Authority Required (Written) listing for initial treatment and an 45PSD · Jul 2025
7.2 The PBAC noted that two other enzyme replacement therapies (ERTs) – agalsidase alfa and agalsidase beta – are currently available through the Life Saving Drugs Program (LSDP), and migalastat is the only medicine listed on the PBS for FD. The PBAC considered pegunigalsidase alfa provides an alternative therapy for FD.PSD · Jul 2025
6.68 The submission did not present a cost-minimisation approach to the proposed main comparators (ERT, agalsidase alfa and agalsidase beta) claiming that both these medications are currently listed on the LSDP and have not demonstrated cost- effectiveness for PBAC consideration. The prices of agalsidase alfa and agalsidase beta on the LSDP were not known to the sponsor.PSD · Jul 2025
6.69 The submission instead presented a cost-minimisation approach against migalastat with a proposal seeking a % price premium over migalastat. The submission claimed this was based on the PBAC’s consideration that the migalastat efficacy claim of non- inferior efficacy against ERT was not well supported, and this was reflected in the history of FD pricing, with migalastat initially being listed on the LSDP at a lower (but unknown) price than agalsidase …PSD · Jul 2025
6.60 The submission described pegunigalsidase alfa as non-inferior to both agalsidase alfa and agalsidase beta in terms of efficacy and safety, providing an alternative option for both ERT-naïve and ERT-experienced patients.PSD · Jul 2025
• Overall, the results from BALANCE at 24 months met the pre-specified non- inferiority margin for annualised change in eGFR slope. While there are some concerns about the validity of the non-inferiority margin, the primary and secondary efficacy outcomes indicate similar findings between pegunigalsidase alfa and agalsidase beta, except for urine lyso-Gb3 and FCE results, which favour agalsidase beta, and MSSI results which favour pegunigalsidase alfa.PSD · Jul 2025
6.2 The PBAC noted and welcomed the input from individuals (4), health care professionals (1) and organisations (2) via the Consumer Comments facility on the PBS website. The comments described the challenges with living with FD, including disruption to daily life and reduced ability to participate in physical activity, work, social activities and education due to symptoms, as well as the emotional impact of managing a chronic condition.PSD · Jul 2025
Comments also stated that current LSDP criteria prevent access to effective treatment in early stages of disease which could potentially prevent permanent impairment, and broader eligibility criteria would improve access to individuals, particularly for those without the migalastat-amenable mutation. Health professional input stated that pegunigalsidase alfa was well tolerated, however there is a risk of hypersensitivity reactions in some individuals.PSD · Jul 2025
Cost-effectiveness
Cost-minimisation analysis versus migalastat; ICER not calculated. Submission based on cost-minimisation approach.
Decision context
PopulationAdults aged 18 years and older with confirmed Fabry disease and end-organ damage (renal disease, cardiac disease, cerebrovascular disease, ischaemic disease, uncontrolled chronic pain, or significant gastrointestinal symptoms).
Submission history
| Decided | Outcome | Comparator | ICER | Evidence |
|---|---|---|---|---|
| Jul 2025 | Recommended · restricted | migalastat | — | RCT · Other |
Clinical evidence
| Trial | Phase | N | Primary outcome | Status |
|---|---|---|---|---|
| BALANCE | Ph 3 | 78 | Annualized Change (Slope) in Estimated Glomerular Filtration Rate (eGFR) | completed |
Consumer voice
Consumer input described significant challenges with living with Fabry disease including disruption to daily life and reduced participation in activities, challenges with current ERT options due to limited efficacy and treatment burden, and support for pegunigalsidase alfa as offering improved quality of life, better treatment tolerability, and access to treatment for those ineligible under curren
The comments described the challenges with living with FD, including disruption to daily life and reduced ability to participate in physical activity, work, social activities and education due to symptoms, as well as the emotional impact of managing a chronic condition. Consumer comments · PSD
Similar precedents
Regulatory · TGA
Label narrower than PBS population — PBAC restricts to patients with documented end-organ damage; TGA label permits all confirmed Fabry disease adults.