PBAC's latest decision on Mavacamten: Recommended with restriction (2023). Considered for Treatment of adults with symptomatic obstructive hypertrophic cardiomyopathy (HCM) with peak left ventricular outflow tract (LVOT) gradient of at least 50 mm Hg, who have had prior treatment with both a beta-blocker and a non-dihydropyridine calcium channel blocker (unless intolerant or contraindicated).
PBAC outcome
Recommended with restriction
Authority Required
ICER (AUD/QALY)
Redacted
commercial-in-confidence
Submissions
3
first 2022
Submissions
3
2022 → 2023
Eligible population
Adult patients (aged ≥18 years) with symptomatic obstructive hypertrophic cardiomyopathy (NYHA class II or III), confirmed peak LVOT gradient ≥50 mm Hg (at rest or with provocation), LVEF ≥55%, and prior treatment with both a beta-blocker and a non-dihydropyridine calcium channel blocker (unless intolerant or contraindicated), treated by a cardiologist or consultant physician with HCM experience.
Therapy area
Cardiovascular
Line of therapy
Later-line
Evidence base
RCT
Primary endpoint
Composite (improvement of ≥1.5 mL/kg/min in peak mixed venous oxygen tension and ≥1 class reduction in NYHA Functional Class, OR improvement of ≥3.0 mL/kg/min in peak oxygen consumption with no worsening in NYHA Functional Class)
Key trials
EXPLORER-HCM, VALOR-HCM
Comparator
beta-blocker and/or calcium channel blocker (standard of care)
Economic model
CUA
ICER note
ICER value is redacted and falls within the stated range of $35,000 to <$45,000 per QALY gained following a price reduction offered by the sponsor.
ICER (historical)
$35k/QALY–$45k/QALY in an earlier submission (2023) — the latest submission carried no numeric base case. No single PSD states this combined range; see source PSDs.
Risk sharing
Risk-sharing arrangement in place — Revised Risk Sharing Arrangement (RSA) inclusive of a percentage rebate above the subsidisation cap for expenditure over the subsidisation threshold.
Submission history
Nov 2022: Recommended with restriction · Authority Required
Jul 2023: Recommended with restriction · Authority Required
Nov 2023: Recommended with restriction · Authority Required — ICER $45k/QALY