PBAC's latest decision on Zilucoplan: Not recommended (2025). Considered for Treatment of generalised myasthenia gravis (gMG) in adult patients with anti-acetylcholine receptor (AChR) antibody positive status who are refractory to conventional immunotherapies.
PBAC outcome
Not recommended
Authority Required
ICER (AUD/QALY)
Redacted
commercial-in-confidence
Submissions
2
first 2024
Submissions
2
2024 → 2025
Eligible population
Adult patients (≥18 years) with symptomatic generalised myasthenia gravis who are AChR-antibody positive and refractory to conventional immunotherapies (defined as treatment for ≥1 year with ≥2 of specified therapies, or history of ≥1 year of specified therapy plus requirement for chronic PLEX/IVIg/subcutaneous immunoglobulin ≥every 3 months), receiving concomitant standard therapy (anticholinesterases, corticosteroids, and/or immunosuppressive agents).
Therapy area
Neurology
Line of therapy
Later-line
Evidence base
RCT
Primary endpoint
Other
Key trials
RAISE
Comparator
placebo
Economic model
CUA
ICER note
ICER values are redacted/commercially sensitive. The document indicates an estimated ICER of $ 1 per QALY gained (within ranges $355,000 to <$455,000 or $255,000 to <$355,000) but exact values are not published.
Risk sharing
Risk-sharing arrangement in place — The sponsor proposed an RSA (risk sharing arrangement) based on the financial estimates of the resubmission, with no further details provided.
Why PBAC said no
Reasons cited in the latest PSD: limitations of the economic evaluation, high price (driver of ICER), inadequate restriction criteria (not requiring optimisation of corticosteroid/immunosuppressive therapy or timeframe for assessing response), continuation criterion based on 3-point MG-ADL reduction (46.1% of placebo group achieved this by placebo effect alone), absence of stopping rules, inadequate safety data despite meningococcal infection risk requiring vaccination, overestimated assumptions regarding IVIg/PLEX cost offsets and clinical event rates
Submission history
Jul 2024: Recommended with restriction · Authority Required