RozanolixizumabRystiggo
Treatment of adult patients with generalised myasthenia gravis (gMG) who are anti-acetylcholine receptor (AChR) antibody positive, as an alternative bridging therapy to intravenous immunoglobulin (IVIg) and plasma exchange (PLEX) while remission induction occurs with non-steroidal immunosuppressants.
Decision on record
- Meeting Mar 2025 Recommended Generalised myasthenia gravis (gMG), anti-acetylcholine receptor antibody positive
Access path
- TGA registered · Rystiggo
TGA label narrower than the PBS population
- Mar 2025Recommended · restricted
vs intravenous immunoglobulin (IVIg)
- Nov 2025Recommended · restricted
- PBS listing · Authority Required
From the public summary
8.1 The PBAC recommended the listing of rozanolixizumab for the treatment of generalised myasthenia gravis (gMG), on the basis that it should be available only under special arrangements under the Section 100 Highly Specialised Drugs Program.PSD · Mar 2025
The PBAC recognised the high clinical need for new therapies to treat this condition, which has substantial impacts on patient quality of life. The recommendation was made on the basis of a cost-comparison versus intravenous immunoglobulin (IVIg), supported by a cost-per-responder analysis versus placebo. The PBAC acknowledged the limitations of the available evidence for chronic IVIg, however the PBAC 52 OFFICIALPSD · Mar 2025
6.69 The submission claimed that one six-week treatment cycle of rozanolixizumab (6 infusions with tiered weight-based dosing) was non-inferior to six months of treatment with IVIg (2 g/kg induction dose split over 2 infusions and 7 × 1 g/kg maintenance infusions). This claim was not adequately supported by the available clinical data but was not the basis of the equi-effective doses used in the cost- minimisation approach.PSD · Mar 2025
6.70 The submission estimated equi-effective doses based on separate annualised estimates of use for both rozanolixizumab (17.80 infusions per year) and IVIg (19.26 infusions per year). This corresponded to equi-effective doses of rozanolixizumab 10,508 mg and IVIg 927 g per year.PSD · Mar 2025
• Rozanolixizumab is likely to be at least non-inferior in terms of efficacy and safety to PLEX, based on MSAC’s previous conclusion that PLEX is non-inferior in terms of efficacy and safety compared with IVIg.PSD · Mar 2025
• Rozanolixizumab is non-inferior to efgartigimod for efficacy, based on the proportion of MG-ADL responders, and safety, based on serious adverse events and discontinuation due to adverse events. Source: Table 1.1-1, pp9-10 of the submission.PSD · Mar 2025
6.3 The PBAC noted and welcomed the input from individuals (17), health care professionals (9 individual health care professionals plus a group of 11 neurologists) and an organisation (Myasthenia Alliance Australia (MAA)) via the Consumer Comments facility on the PBS website. The comments described the high unmet need for new therapies to treat gMG.PSD · Mar 2025
6.4 The comments noted the efficacy associated with rozanolixizumab and its rapid onset of action. One clinician outlined that FcRn blockers provide an alternative to IVIg and PLEX in that they can provide rapid improvement in patients who have significant weakness, which is important as some patients are unresponsive or intolerant to IVIg, and PLEX is not available at all centres.PSD · Mar 2025
Cost-effectiveness
Cost-minimisation analysis; no ICER calculated as the submission was based on equi-effective dosing with IVIg.
The submission claimed that one six-week treatment cycle of rozanolixizumab (6 infusions with tiered weight-based dosing) was non-inferior to six months of treatment with IVIg (2 g/kg induction dose split over 2 infusions and 7 × 1 g/kg maintenance infusions). PSD · 2025
Decision context
PopulationAdult patients with generalised myasthenia gravis (MGFA Disease Class II to IVa) who are AChR antibody positive, with a Myasthenia Gravis Composite (MGC) score of at least 4 points, receiving concomitant treatment with at least one non-steroidal immunosuppressant (azathioprine, methotrexate, cyclophosphamide, ciclosporin, or mycophenolate), and not receiving concurrent IVIg or plasma exchange.
Submission history
| Decided | Outcome | Comparator | ICER | Evidence |
|---|---|---|---|---|
| Nov 2025 | Recommended · restricted | intravenous immunoglobulin (IVIg) | — | RCT · Surrogate |
| Mar 2025 | Recommended · restricted | intravenous immunoglobulin (IVIg) | — | RCT |
Consumer voice
Seventeen individuals, nine individual healthcare professionals, eleven neurologists as a group, and Myasthenia Alliance Australia provided input describing high unmet need for new gMG therapies, significant quality-of-life impacts, treatment limitations, and support for access to new treatments with manageable administration modes and improved symptom control.
The comments described the high unmet need for new therapies to treat gMG. Consumer comments · PSD
Similar precedents
Regulatory · TGA
Label narrower than PBS population — PBAC restricts to AChR-positive only, excludes MuSK-positive patients, and adds specific clinical/treatment criteria.