RavulizumabUltomiris 100 mg/ml
Treatment of newly diagnosed and treatment-refractory patients with generalised myasthenia gravis who are anti-acetylcholine receptor (AChR) antibody positive and remain symptomatic despite standard therapy.
Decisions on record
- Meeting Mar 2025 Recommended Generalised myasthenia gravis (gMG), anti-acetylcholine receptor antibody positive
- Meeting Nov 2024 Recommended Neuromyelitis optica spectrum disorder
- Meeting Jul 2024 Not recommended Generalised myasthenia gravis anti-acetylcholine receptor antibody positive
- Meeting Mar 2024 Noted Generalised myasthenia gravis (gMG) no PSD
- Meeting Jul 2023 Recommended Atypical haemolytic uraemic syndrome (aHUS)
- Meeting Jul 2023 Recommended Paroxysmal nocturnal haemoglobinuria (PNH)
- Meeting Mar 2023 Deferred Atypical haemolytic uraemic syndrome (aHUS)
- Meeting Jul 2021 Recommended Paroxysmal nocturnal haemoglobinuria
1 earlier decision
- Jul 2020 Not recommended Paroxysmal nocturnal haemoglobinuria (PNH)
Access path
- TGA registered · Ultomiris 100 mg/ml
TGA label narrower than the PBS population
- Jul 2020Not recommended
vs eculizumab (primary), best supportive care (secondary)
- ↻ resubmittedJul 2021Recommended · restricted
Comparator changed: eculizumab (primary), best supportive care (secondary) → eculizumab…
- Jul 2023Recommended · restricted
Comparator changed: eculizumab and best supportive care → eculizumab
- Jul 2023Recommended · restricted
Comparator changed: eculizumab and best supportive care → eculizumab
- Jul 2024Not recommended
Comparator changed: eculizumab → placebo
- ↻ resubmittedNov 2024Recommended · restricted
Comparator changed: placebo → best supportive care (placebo)
- Mar 2025Recommended · restricted
Comparator changed: best supportive care (placebo) → placebo
- PBS listing · Authority Required
From the public summary
8.1 The PBAC recommended the listing of ravulizumab 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. The PBAC recognised the high clinical need for new therapies to treat this condition, which has substantial impacts on patient quality of life.PSD · Mar 2025
OFFICIAL Public Summary Document – March 2025 PBAC Meeting IVIg or PLEX. Further, the PBAC considered that there was no reliable evidence to suggest that ravulizumab was superior in terms of efficacy or safety compared with the other three therapies considered at the March 2025 meeting for the treatment of gMG (zilucoplan, efgartigimod and rozanolixizumab).PSD · Mar 2025
6.43 The economic evaluation was extensively revised from the March 2024 submission which presented an economic analysis of ravulizumab when used as a treatment option immediately prior to the classification of patients as having treatment- refractory disease (i.e. before IVIg/PLEX).PSD · Mar 2025
6.44 The resubmission presented a stepped economic evaluation of ravulizumab in combination with standard therapy compared to placebo in combination with standard therapy for the treatment of AChR positive generalised myasthenia gravis in newly diagnosed and treatment refractory patients. The economic evaluation was based on a direct randomised trial (CHAMPION-MG) with additional modelled data.PSD · Mar 2025
6.38 The resubmission described ravulizumab in combination with standard therapy as superior in terms of efficacy and inferior in terms of safety compared to placebo in combination with standard therapy in generalised myasthenia gravis patients with newly diagnosed disease or treatment refractory disease. The evaluation and the ESC considered that this claim was reasonable.PSD · Mar 2025
6.39 The evaluation and the ESC considered that the following issues should be noted for the comparison versus standard care: 29 OFFICIALPSD · Mar 2025
6.3 The PBAC noted and welcomed the input from individuals (16), health care professionals (1 individual clinician, 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 ravulizumab and the rapid onset of action. The input outlined the risk of infection associated with ravulizumab including the risk of meningococcal infection. However, the majority of contributors expressed an enthusiasm to try ravulizumab regardless of potential adverse events due to its reported efficacy.PSD · Mar 2025
Cost-effectiveness
ICER values not stated in public text. The PSD notes that the July 2024 PBAC considered the ICER presented was very high and likely underestimated, but specific numerical values are not disclosed in this resubmission document.
A claim of non-inferiority was made in the submission based on an unanchored (naïve) indirect comparison between two single-arm studies. PSD · 2023
Decision context
PopulationAdult patients with generalised myasthenia gravis (not pure ocular) who are AChR antibody positive: (1) newly diagnosed (within 2 years) with MG-ADL ≥6 despite stable corticosteroids and/or non-steroidal immunosuppressants, who have failed IVIg/PLEX; or (2) treatment-refractory with ≥24 months prior immunosuppressive therapy and failed IVIg/PLEX. Patients must not be in myasthenic crisis.
Submission history
| Decided | Outcome | Comparator | ICER | Evidence |
|---|---|---|---|---|
| Mar 2025 | Recommended · restricted | placebo | — | RCT · Other |
| Nov 2024 | Recommended · restricted | best supportive care (placebo) | $455k–555k | RCT · PFS |
| Jul 2024 | Not recommended | placebo | — | RCT · Other |
| Jul 2023 | Recommended · restricted | eculizumab | — | Cost-minimisation · Surrogate |
| Jul 2023 | Recommended · restricted | eculizumab | — | Single-arm · Surrogate |
| Jul 2021 | Recommended · restricted | eculizumab and best supportive care | — | RCT · Other |
| Jul 2020 | Not recommended | eculizumab (primary), best supportive care (secondary) | — | RCT · Transfusion avoidance, haemolysis (LDH change), breakthrough haemolysis, quality of life, stabilised haemoglobin |
Consumer voice
Consumers and healthcare professionals emphasized the high unmet need for new gMG therapies, highlighting significant quality-of-life impacts including inability to perform daily tasks and work, as well as the burden on family and finances. Contributors expressed enthusiasm for new treatments despite infection risks, with particular interest in more manageable modes of administration compared to c
The comments outlined a hope that the new therapies will reduce gMG symptoms, reduce the need for other medications and associated side-effects, and reduce hospital visits, contributing to an overall improved quality of life. Consumer comments · PSD
Similar precedents
Regulatory · TGA
Label narrower than PBS population — PBAC restricts to newly diagnosed or treatment-refractory patients with specific prior therapy failures and symptom thresholds; TGA label permits all gMG AChR+ patients on standard therapy.