MavacamtenCamzyos
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).
Decisions on record
- Meeting Nov 2023 Recommended Symptomatic obstructive hypertrophic cardiomyopathy
- Meeting Jul 2023 Not recommended Hypertrophic cardiomyopathy (HCM)
- Meeting Nov 2022 Not recommended Symptomatic obstructive hypertrophic cardiomyopathy
Access path
- TGA registered · Camzyos
TGA label narrower than the PBS population
- Nov 2022Not recommended
vs Standard of care (beta-blocker or non-dihydropyridine…
- Jul 2023Not recommended
Comparator changed: Standard of care (beta-blocker or non-dihydropyridine calcium channel…
- ↻ resubmittedNov 2023Recommended
Comparator changed: Standard of care (beta-blocker or calcium channel blocker) →…
- PBS listing · Authority Required
From the public summary
5.1 The PBAC recommended the listing of mavacamten for the treatment of adults with symptomatic obstructive hypertrophic cardiomyopathy (HCM). The PBAC is satisfied that mavacamten provides, for some patients, a significant improvement in efficacy over standard of care, in terms of symptomatic improvement, and it would be cost effective at the reduced price proposed in the resubmission.PSD · Nov 2023
5.2 The PBAC considered that the resubmission had addressed the substantive outstanding issues identified at the July 2023 meeting via a revised restriction, revised economic modelling, a price reduction, revised financial estimates, and an RSA proposal with a % rebate above the subsidisation cap.PSD · Nov 2023
4.3 The resubmission provided a revised economic model in line with the PBAC advice from July 2023, as detailed in Table 1. The only disputed change related to hospitalisations rates for HCM, which have been modelled to increase by NYHA class.PSD · Nov 2023
The proposed CV hospitalisation rates were based on an Advisory Board survey of 9 cardiologists and remained unchanged from the November 2022 and July 2023 submissions. The PBAC had previously considered a more conservative approach would be appropriate and suggested halving the CV hospitalisations rates: NYHA II (i.e.PSD · Nov 2023
The resubmission described mavacamten ± BB/CCB as superior in terms of effectiveness and inferior in terms of safety compared to SOC alone (BB/CCB).PSD · Jul 2023
The therapeutic conclusion for comparative effectiveness presented in the resubmission was reasonable for the management of symptomatic outcomes pertaining to obstructive HCM. The PBAC previously noted that EXPLORER-HCM was a small trial (N=251) with a short duration of comparative follow up (30 weeks); and symptomatic/functional endpoints were only reported.PSD · Jul 2023
The comment described the expected benefits of treatment with mavacamten in 7PSD · Nov 2023
4.14 In July 2023, the PBAC considered that a resubmission should include a revised RSA inclusive of a 100% rebate to Government for expenditure over the subsidisation cap 11PSD · Nov 2023
Cost-effectiveness
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.
Decision context
PopulationAdult 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.
Risk sharingRevised Risk Sharing Arrangement (RSA) inclusive of a percentage rebate above the subsidisation cap for expenditure over the subsidisation threshold.
Submission history
| Decided | Outcome | Comparator | ICER | Evidence |
|---|---|---|---|---|
| Nov 2023 | Recommended | beta-blocker and/or calcium channel blocker (standard of care) | $35k–45k | RCT · 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) |
| Jul 2023 | Not recommended | Standard of care (beta-blocker or calcium channel blocker) | — | RCT · Surrogate |
| Nov 2022 | Not recommended | Standard of care (beta-blocker or non-dihydropyridine calcium channel blocker) | — | RCT |
Clinical evidence
| Trial | Phase | N | Primary outcome | Status |
|---|---|---|---|---|
| EXPLORER-HCM | Ph 3 | 251 | Percentage of Participants Achieving A Clinical Response | completed |
| VALOR-HCM | Ph 3 | 112 | Composite of Decision to Proceed With Septal Reduction Therapy (SRT) and SRT Guideline Eli… | completed |
Consumer voice
One individual provided consumer input via the PBS website describing expected benefits of treatment with mavacamten.
Similar precedents
Regulatory · TGA
Label narrower than PBS population — PBAC restricts to peak LVOT gradient ≥50 mm Hg, LVEF ≥55%, and prior dual therapy failure; TGA label has no biomarker or treatment history requirements.