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Now Approved in Pediatric Patients Weighing ≥30 kg With Symptomatic oHCM1

Explore this page:    |    Study Design    |    LVOT Gradient    |    NYHA Class    |   Pediatric Safety

CAMZYOS Pediatric Dosing: To find information on Dosing & Administration, please see US Full Prescribing Information, including Boxed Warning. The CAMZYOS 1-mg dose strength is anticipated to be available in mid-December 2026.

Study Design: SCOUT-HCM2,3

The efficacy and safety of CAMZYOS were evaluated in SCOUT-HCM, a Phase III, randomized, double-blind, placebo-controlled trial in patients aged 12 to 17 years with symptomatic NYHA Class II–III obstructive HCM. Patients were randomized 1:1 to receive a starting dose of CAMZYOS (n=23) based on weight (2.5 mg in patients 35 to <45 kg; 5 mg in patients ≥45 kg) with potential titrations to once-daily doses of 1, 2.5, 5, 10, or 15 mg; or placebo (n=21) for 28 weeks. Enrolled patients had LVEF ≥60%, Valsalva LVOT peak gradient ≥30 mmHg and a maximal gradient ≥50 mmHg at rest or with provocation, and background obstructive HCM therapy with a beta blocker, calcium channel blocker, disopyramide, or combinations was permitted. Dose adjustment was allowed at Weeks 5, 9, 12, and 24 based on echocardiographic assessments of Valsalva LVOT gradient and LVEF.

Patients were excluded for HCM phenocopies (Noonan's Syndrome, Fabry's Disease), recent (last 6 months) LVEF <50%, planned escalation of HCM therapy or cardiac intervention, or treatment with moderate to strong CYP2C19 inhibitors or strong CYP3A4 inhibitors (within 14 days before screening).

The primary endpoint was the change from baseline in Valsalva LVOT gradient at Week 28. Secondary endpoints included change from baseline to Week 28 in resting LVOT gradient, post-exercise peak LVOT gradient, maximal LV wall thickness, E/e′, and HCMSQ-SoB domain score. Additional secondary endpoints included the proportion of patients achieving an increase in pVO₂, resting or Valsalva LVOT gradient <30 mmHg at Week 28, ≥1 NYHA class improvement, or ≥1 mitral regurgitation grade improvement. Secondary and exploratory endpoints were summarized descriptively with estimates of between-group differences and 95% confidence intervals, which were not adjusted for multiplicity and should not be interpreted as formal hypothesis testing. Safety endpoints included incidence of adverse events, serious adverse events, and a decrease in LVEF to <50%.

At baseline, 83% and 86% of patients were receiving beta blockers, 13% and 10% were receiving calcium channel blockers, and 17% and 24% were receiving disopyramide, in the CAMZYOS and placebo groups, respectively. Mean LVEF was 69.0% in the CAMZYOS group and 67.4% in the placebo group; mean Valsalva LVOT gradient was 78 mmHg in the CAMZYOS group and 81 mmHg in the placebo group.

CYP=cytochrome P450; E/e′=ratio between early mitral inflow velocity and mitral annular early diastolic velocity; HCM=hypertrophic cardiomyopathy; HCMSQ-SoB=Hypertrophic Cardiomyopathy Symptom Questionnaire–Shortness-of-Breath domain score; LV=left ventricle; LVEF=left ventricular ejection fraction; LVOT=left ventricular outflow tract; NYHA=New York Heart Association; pVO2=peak oxygen consumption.

Reducing LVOT Obstruction Objectively Marks Therapeutic Benefit in Obstructive HCM in Adolescents4

Primary Endpoint: CAMZYOS is the first FDA-approved treatment in pediatric patients (12–17 years) proven to reduce obstruction as measured by Valsalva LVOT gradient vs placebo at Week 281,2

SCOUT-HCM primary endpoint: Mean change in Valsalva LVOT (SD) gradient from baseline to Week 281,2

CAMZYOS achieved: -49.4 (42.6) mmHg at Week 28 in Valsalva LVOT gradient vs -1.8 (37.8) mmHg for placebo; least squares mean difference:
-48.0 mmHg (95% CI: -67.7, -28.3); P<0.0001

The results for secondary and exploratory efficacy endpoints were summarized descriptively with estimates of between-group differences and 95% 
confidence intervals, which were not adjusted for multiplicity and should not be interpreted as formal hypothesis testing.

Secondary efficacy endpoints: Mean change in resting and postexercise LVOT gradients from baseline to Week 281,2

  • Reductions [SD] in mean resting LVOT gradient were observed for CAMZYOS (-41 [35] mmHg) and placebo (+5.3 [23] mmHg) patients
  • Patients achieved reductions of -31.2 mmHg (95% CI: -46.3, -16.1) in postexercise LVOT gradient with CAMZYOS vs +10.5 mmHg (95% CI: -5.5, 26.6) with placebo

Secondary endpoint: 65% of patients (n=15/23) receiving CAMZYOS and 5% of patients (n=1/21) receiving placebo were no longer obstructed (resting or post-Valsalva)* at Week 285

*Unobstructed defined as maximal LVOT gradient (resting or Valsalva) of <30 mmHg.5
CI=confidence interval; FDA=US Food and Drug Administration; HCM=hypertrophic cardiomyopathy; LVOT=left ventricular outflow tract; SD=standard deviation.

NYHA Class Is a Subjective Measure of a Patient's Functional Capacity (Symptoms)6

Secondary Endpoint: Reductions of ≥1 NYHA class from baseline to Week 28 were evaluated in pediatric patients (12–17 years) taking CAMZYOS5

The results for secondary and exploratory efficacy endpoints were summarized descriptively with estimates of between-group differences and 95% confidence intervals, which were not adjusted for multiplicity and should not be interpreted as formal hypothesis testing.

Missing NYHA class at Week 28 was imputed using available data from Week 23. If multiple NYHA class records existed within the Week 23 analysis window, then the latest record was used. After imputation, participants whose response status at Week 28 was still missing were classified as nonresponders.5 
NYHA=New York Heart Association.

No New Adverse Reactions Were Seen in Adolescents Receiving CAMZYOS1,2

CAMZYOS demonstrated a safety profile in SCOUT-HCM consistent with prior studies in adults1,2

LVEF remained ≥50% in all patients in both groups

*In one patient, an inappropriate shock was delivered by an implantable cardioverter-defibrillator that led to an interruption in treatment during the patient’s hospitalization.2
LVEF=left ventricular ejection fraction.

References:

  1. CAMZYOS [package insert] Princeton, NJ: Bristol-Myers Squibb Company; 2026.
  2. Rossano JW, Canter C, Wolf CM, et al. Mavacamten in adolescents with obstructive hypertrophic cardiomyopathy. N Engl J Med. 2026;395(4):362-373.
  3. Rossano J, Canter C, Wolf C, et al. Mavacamten in symptomatic adolescent patients with obstructive hypertrophic cardiomyopathy: design of the phase 3 SCOUT-HCM trial. Am Heart J. 2026;292:107283.
  4. Ommen SR, Ho CY, Asif IM, et al. AHA/ACC/AMSSM/HRS/PACES/SCMR Guideline for the management of hypertrophic cardiomyopathy: a report of the American Heart Association/American College of Cardiology Joint Committee on Clinical Practice Guidelines. Circulation. 2024;149:e1239–e1311. doi: 10.1161/CIR.0000000000001250
  5. Rossano JW, Canter C, Wolf CM, et al. Mavacamten in adolescents with obstructive hypertrophic cardiomyopathy. N Engl J Med. 2026;395(4):362-373 [supplementary appendix].
  6. Ho CY, Olivotto I, Jacoby D, et al. Study design and rationale of EXPLORER-HCM: evaluation of mavacamten in adults with symptomatic obstructive hypertrophic cardiomyopathy. Circ Heart Fail. 2020;13(6):e006853.
References

IMPORTANT SAFETY INFORMATION

WARNING: RISK OF HEART FAILURE

CAMZYOS reduces left ventricular ejection fraction (LVEF) and can cause heart failure due to systolic dysfunction.

Echocardiogram assessments of LVEF are required prior to and during treatment with CAMZYOS. Initiation of CAMZYOS in patients with LVEF <55% is not recommended. Interrupt CAMZYOS if LVEF is <50% at any visit or if the patient experiences heart failure symptoms or worsening clinical status.

Concomitant use of CAMZYOS with certain cytochrome P450 inhibitors or discontinuation of certain cytochrome P450 inducers may increase the risk of heart failure due to systolic dysfunction; therefore, the use of CAMZYOS is contraindicated with the following:

 
  • Strong CYP2C19 inhibitors
  • Moderate to strong CYP2C19 inducers or moderate to strong CYP3A4 inducers

Because of the risk of heart failure due to systolic dysfunction, CAMZYOS is available only through a restricted program under a Risk Evaluation and Mitigation Strategy (REMS) called the CAMZYOS REMS Program.

CONTRAINDICATIONS

CAMZYOS is contraindicated with concomitant use of:

 
  • Strong CYP2C19 inhibitors
  • Moderate to strong CYP2C19 inducers or moderate to strong CYP3A4 inducers

WARNINGS AND PRECAUTIONS

Heart Failure
CAMZYOS reduces systolic contraction and can cause heart failure or significantly reduce ventricular function. Patients who experience a serious intercurrent illness (eg, serious infection) or arrhythmia (eg, atrial fibrillation or other uncontrolled tachyarrhythmia) are at greater risk of developing systolic dysfunction and heart failure.

Assess the patient’s clinical status and LVEF prior to and regularly during treatment and adjust the CAMZYOS dose accordingly. New or worsening arrhythmia, dyspnea, chest pain, fatigue, palpitations, leg edema, or elevations in N-terminal pro-B-type natriuretic peptide (NT-proBNP) may be signs and symptoms of heart failure and should also prompt an evaluation of cardiac function.

Asymptomatic LVEF reduction, intercurrent illnesses, and arrhythmias require additional dosing considerations.

Initiation of CAMZYOS in patients with LVEF <55% is not recommended. Avoid concomitant use of CAMZYOS in patients on disopyramide, ranolazine, verapamil with a beta blocker, or diltiazem with a beta blocker as these medications and combinations increase the risk of left ventricular systolic dysfunction and heart failure symptoms and clinical experience is limited.

CYP450 Drug Interactions Leading to Heart Failure or Loss of Effectiveness

CAMZYOS is primarily metabolized by CYP2C19 and CYP3A4 enzymes. Concomitant use of CAMZYOS and drugs that interact with these enzymes may lead to life-threatening drug interactions such as heart failure or loss of effectiveness.

Advise patients of the potential for drug interactions, including with over-the-counter medications (such as omeprazole, esomeprazole, or cimetidine). Advise patients to inform their healthcare provider of all concomitant products prior to and during CAMZYOS treatment.

CAMZYOS Risk Evaluation and Mitigation Strategy (REMS) Program

CAMZYOS is only available through a restricted program called the CAMZYOS REMS Program because of the risk of heart failure due to systolic dysfunction. Notable requirements of the CAMZYOS REMS Program include the following:

 
  • Prescribers must be certified by enrolling in the REMS Program
  • Patients must enroll in the REMS Program and comply with ongoing monitoring requirements
  • Pharmacies must be certified by enrolling in the REMS Program and must only dispense to patients who are authorized to receive CAMZYOS
  • Wholesalers and distributors must only distribute to certified pharmacies

Further information is available at  www.CAMZYOSREMS.com  or by telephone at  1-833-628-7367.

Embryo-Fetal Toxicity

CAMZYOS may cause fetal toxicity when administered to a pregnant female, based on animal studies. Confirm absence of pregnancy in females of reproductive potential prior to treatment and advise patients to use effective contraception during treatment with CAMZYOS and for 4 months after the last dose. Combined hormonal contraceptives (CHCs) containing a combination of ethinyl estradiol and norethindrone may be used with CAMZYOS. However, CAMZYOS may reduce the effectiveness of certain other CHCs. If these CHCs are used, advise patients to add nonhormonal contraception (such as condoms) during concomitant use and for 4 months after the last dose of CAMZYOS.

ADVERSE REACTIONS

In the EXPLORER-HCM trial, adverse reactions occurring in >5% of patients and more commonly in the CAMZYOS group than in the placebo group were dizziness (27% vs 18%) and syncope (6% vs 2%). There were no new adverse reactions identified in VALOR-HCM. The safety of CAMZYOS in pediatric patients (≥35 kg) was evaluated in SCOUT-HCM. There were no new adverse reactions compared to those identified in adult studies.

Effects on Systolic Function

In the EXPLORER-HCM trial, 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. In the EXPLORER-HCM trial, 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 all 7 patients treated with CAMZYOS, LVEF recovered following interruption of CAMZYOS.

DRUG INTERACTIONS

Potential for Other Drugs to Affect Plasma Concentrations of CAMZYOS

CAMZYOS is primarily metabolized by CYP2C19 and to a lesser extent by CYP3A4 and CYP2C9. Inducers and inhibitors of CYP2C19 and moderate to strong inhibitors or inducers of CYP3A4 may affect the exposures of CAMZYOS.

Impact of Other Drugs on CAMZYOS:

 
  • Strong CYP2C19 Inhibitors: Concomitant use increases CAMZYOS exposure, which may increase the risk of heart failure due to systolic dysfunction. Concomitant use is contraindicated
  • Moderate to Strong CYP2C19 Inducers or Moderate to Strong CYP3A4 Inducers:  Concomitant use decreases CAMZYOS exposure, which may reduce CAMZYOS’ efficacy. The risk of heart failure due to systolic dysfunction may increase with discontinuation of these inducers as the levels of induced enzyme normalizes. Concomitant use is contraindicated
  • Weak CYP2C19 Inhibitors or Moderate CYP3A4 Inhibitors: Concomitant use with a weak CYP2C19 inhibitor or a moderate CYP3A4 inhibitor increases CAMZYOS exposure, which may increase the risk of adverse drug reactions. Initiate CAMZYOS at the recommended starting dosage in patients who are on stable therapy with a weak CYP2C19 inhibitor or a moderate CYP3A4 inhibitor. Reduce dose of CAMZYOS by one level (ie, 15 mg to 10 mg, 10 mg to 5 mg, 5 mg to 2.5 mg, or 2.5 mg to 1 mg [pediatric dosing]) in patients who are on CAMZYOS treatment and intend to initiate a weak CYP2C19 inhibitor or a moderate CYP3A4 inhibitor. Avoid initiation of concomitant weak CYP2C19 and moderate CYP3A4 inhibitors in patients who are on the lowest recommended dose of CAMZYOS. For short-term use (eg, 1 week), interrupt CAMZYOS for the duration of treatment with a weak inhibitor of CYP2C19 or a moderate inhibitor of CYP3A4. CAMZYOS may be reinitiated at the previous dose immediately on discontinuation of concomitant therapy.
  • Moderate CYP2C19 Inhibitors or Strong CYP3A4 Inhibitors: Concomitant use with a moderate CYP2C19 inhibitor or strong CYP3A4 inhibitor increases CAMZYOS exposure, which may increase the risk of adverse drug reactions. Discontinuing use of a moderate CYP2C19 inhibitor or strong CYP3A4 inhibitor after long-term concomitant use may decrease CAMZYOS exposure, which may reduce CAMZYOS’ efficacy. Initiate CAMZYOS at one dose level below the recommended starting dose in patients who are on stable therapy with a moderate CYP2C19 inhibitor or a strong CYP3A4 inhibitor. Reduce dose of CAMZYOS by one level (ie, 15 mg to 10 mg; 10 mg to 5 mg; 5 mg to 2.5 mg; or 2.5 mg to 1 mg [pediatric dosing]) in patients who are on CAMZYOS and intend to initiate a moderate CYP2C19 inhibitor or a strong CYP3A4 inhibitor. Avoid initiation of concomitant moderate CYP2C19 and strong CYP3A4 inhibitors in patients who are on the lowest recommended dose of CAMZYOS. An increase in dose of CAMZYOS may be needed if the moderate inhibitor of CYP2C19 or strong inhibitor of CYP3A4 is discontinued after long-term concomitant use. Monitor for new or worsening symptoms. For short-term use (eg, 1 week), interrupt CAMZYOS for the duration of treatment with a moderate inhibitor of CYP2C19 or a strong inhibitor of CYP3A4. CAMZYOS may be reinitiated at the previous dose immediately on discontinuation of concomitant therapy.

Potential for CAMZYOS to Affect Plasma Concentrations of Other Drugs

CAMZYOS is an inducer of CYP3A4, CYP2C9, and CYP2C19. Concomitant use with CYP3A4, CYP2C9, or CYP2C19 substrates may reduce plasma concentration of these drugs. Closely monitor when CAMZYOS is used with concomitant CYP3A4, CYP2C9, or CYP2C19 substrates unless otherwise recommended in the Prescribing Information.

Certain Combined Hormonal Contraceptives (CHCs): Progestin and ethinyl estradiol are CYP3A4 substrates. Concomitant use of CAMZYOS may decrease exposures of certain progestins, which may lead to contraceptive failure. CHCs containing a combination of ethinyl estradiol and norethindrone may be used with CAMZYOS, but if other CHCs are used, advise patients to add nonhormonal contraception (such as condoms) or use an alternative contraceptive method that is not affected by CYP450 enzyme induction (eg, intrauterine system) during concomitant use and for 4 months after the last dose of CAMZYOS.

Drugs That Reduce Cardiac Contractility

Expect additive negative inotropic effects of CAMZYOS and other drugs that reduce cardiac contractility. Avoid concomitant use of CAMZYOS in patients on disopyramide, ranolazine, verapamil with a beta blocker, or diltiazem with a beta blocker as these medications and combinations increase the risk of left ventricular systolic dysfunction and heart failure symptoms and clinical experience is limited.

If concomitant therapy with a negative inotrope is initiated, or if the dose of a negative inotrope is increased, monitor LVEF closely until stable doses and clinical response have been achieved.

SPECIFIC POPULATIONS

Pregnancy

CAMZYOS may cause fetal harm when administered to a pregnant female. Advise pregnant females about the potential risk to the fetus with maternal exposure to CAMZYOS during pregnancy. There is a pregnancy safety study for CAMZYOS. If CAMZYOS is administered during pregnancy, or if a patient becomes pregnant while receiving CAMZYOS or within 4 months after the last dose of CAMZYOS, healthcare providers should report CAMZYOS exposure by contacting Bristol Myers Squibb at 1-800-721-5072 or www.bms.com.

Lactation

The presence of CAMZYOS in human or animal milk, the drug’s effects on the breastfed infant, or the effects on milk production are unknown. The developmental and health benefits of breastfeeding should be considered along with the mother’s clinical need for CAMZYOS and any potential adverse effects on the breastfed child from CAMZYOS or from the underlying maternal condition.

Females and Males of Reproductive Potential

Confirm absence of pregnancy in females of reproductive potential prior to initiation of CAMZYOS. Advise females of reproductive potential to use effective contraception during treatment with CAMZYOS and for 4 months after the last dose. CHCs containing a combination of ethinyl estradiol and norethindrone may be used with CAMZYOS. However, CAMZYOS may reduce the effectiveness of certain other CHCs. If these CHCs are used, advise patients to add nonhormonal contraception (such as condoms) or use an alternative contraceptive method during concomitant use and for 4 months after the last dose of CAMZYOS.

INDICATION

CAMZYOS® (mavacamten) is indicated for the treatment of adults and pediatric patients weighing 30 kg or more with symptomatic obstructive hypertrophic cardiomyopathy (HCM) to improve functional capacity and symptoms.

Please see U.S. Full Prescribing Information, including  Boxed WARNING.

3500-US-2600496   09/26

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