EXPLORER-HCM:
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CAMZYOS (n=123) | Placebo (n=128) | |
Dizziness, % | 27 | 18 |
Syncope, % | 6 | 2 |
For patients with NYHA class II–III obstructive HCM,
CAMZYOS® Select Safety Profile in EXPLORER-HCM and EXPLORER-LTE1
Explore this page: | EXPLORER-HCM | EXPLORER-LTE
EXPLORER-HCM: Safety
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Discontinuations1
- Overall, 244 (97%) patients completed treatment. 7 patients discontinued treatment prematurely2; 3 due to adverse events (2 on CAMZYOS [atrial fibrillation and syncope], 1 on placebo [sudden death]); 2 patients withdrew (1 on CAMZYOS, 1 on placebo); and 2 patients discontinued for other reasons (1 on CAMZYOS, 1 on placebo)3
- Syncope (0.8%) was the only adverse drug reaction leading to discontinuation in patients receiving CAMZYOS1
Effects on systolic function1
- Mean (SD) resting LVEF was 74% (6) at baseline in both treatment groups
- Mean (SD) absolute change from baseline in LVEF was -4% (8) in the CAMZYOS group and 0% (7) in the placebo group over the 30-week treatment period
- At Week 38, following an 8-week interruption of trial drug, mean LVEF was similar to baseline for both treatment groups
- 7 (6%) patients in the CAMZYOS group and 2 (2%) patients in the placebo group experienced reversible reductions in LVEF <50% (median 48%: range 35%-49%) while on treatment
- In 3 of the 7 patients on CAMZYOS and 1 of the 2 patients on placebo, these reductions were asymptomatic
- In all 7 patients treated with CAMZYOS, LVEF recovered following interruption of CAMZYOS
Adverse reactions are defined as responses to the drug that are unintended, occurring at doses typically used for treatment of the disease.4
HCM=hypertrophic cardiomyopathy; LTE-long-term extension; LVEF=left ventricular ejection fraction; NYHA=New York Heat Association; SD=standard deviation.
EXPLORER-LTE: Safety
Interim analysis from EXPLORER-LTE
At ~3.5 Years, CAMZYOS Showed a Consistent Safety Profile5
The safety profile of the MAVA-LTE trial was consistent with that of the established pivotal trial, EXPLORER-HCM. In EXPLORER-LTE, serious TEAEs were reported in 63 (27%) patients (total of 117 events recorded). Of these, 28 patients (12%) experienced CV-related serious TEAEs* (total of 40 events recorded), and 10 patients† experienced drug-related serious TEAEs (total of 10 events recorded).5
LVEF <50% was observed in 20 patients (9%), with 22 events reported. LVEF <40% was seen in 6 patients (2.6%), accounting for 6 events. There were no occurrences of LVEF below 30%. Five deaths were recorded, all unrelated to CAMZYOS.‡
* | CV serious TEAEs of clinical interest are defined as serious TEAEs related to major adverse CV events, atrial fibrillation, ventricular arrhythmias, syncope/presyncope, cardiac failure, hypotension, and QTcF prolongation.5 |
† | Includes cardiac failure (n=3), decreased ejection fraction (n=5), atrial fibrillation (n=1), and atrial flutter (n=1).5 |
‡ | By Week 180, 5 patients had died due to bacterial endocarditis, cardiac arrest, acute myocardial infarction, intracerebral hemorrhage, or liver metastasis (n=1 each), all unrelated to treatment.5 |
CV=cardiovascular; QTcF=QT interval corrected using Fridericia’s formula; TEAE=treatment-emergent adverse event.
With Prolonged Follow-up, Mean LVEF Remained >60% At All Trial Visits5
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20 patients (8.7%) experienced reductions in LVEF <50% in a total of 22 events5
All 20 patients recovered LVEF to ≥50% after treatment interruption5
14 resumed treatment and 6 discontinued treatment (1 later re-enrolled)5
LVEF reductions were reversible after treatment interruption, affected a limited number of patients, and were generally consistent with EXPLORER-HCM.5
§ | Baseline is defined as last non-missing measurement before the first dose of CAMZYOS in MAVA-LTE.5 |
BL=baseline.
References:
- CAMZYOS [package insert]. Princeton, NJ: Bristol-Myers Squibb Company; 2025.
- Olivotto I, Oręziak A, Barriales-Villa R, et al; EXPLORER-HCM study investigators. Mavacamten for treatment of symptomatic obstructive hypertrophic cardiomyopathy (EXPLORER-HCM): a randomised, double-blind, placebo-controlled, phase 3 trial. Lancet. 2020;396(10253):759-769 [supplementary appendix]. doi:10.1016/S0140-6736(20)31792-X
- Olivotto I, Oręziak A, Barriales-Villa R, et al; EXPLORER-HCM study investigators. Mavacamten for treatment of symptomatic obstructive hypertrophic cardiomyopathy (EXPLORER-HCM): a randomised, double-blind, placebo-controlled, phase 3 trial. Lancet. 2020;396(10253):759-769. doi:10.1016/S0140-6736(20)31792-X
- Coleman JJ, Pontefract SK. Adverse drug reactions. Clin Med (Lond). 2016;16(5):481-485. doi:10.7861/clinmedicine.16-5-481
- Garcia-Pavia P, Oręziak A, Masri A, et al. Long-term effect of mavacamten in obstructive hypertrophic cardiomyopathy. Eur Heart J. 2024;45(47):5071-5083. doi:10.1093/eurheartj/ehae579
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