New Treatment Option for Adults and Pediatric Patients Weighing 30 kg (66 lbs) or More With Symptomatic Obstructive Hypertrophic Cardiomyopathy

Sponsored by Bristol Myers Squibb

Until now, pediatric patients with symptomatic obstructive hypertrophic cardiomyopathy (oHCM) have not had an FDA-approved treatment option specifically for their disease. On September 30, 2026, the U.S. Food and Drug Administration (FDA) approved CAMZYOS^® (mavacamten) for symptomatic obstructive hypertrophic cardiomyopathy (oHCM) to improve functional capacity and symptoms in adults and pediatric patients weighing 30 kg (66 lbs) or more, making CAMZYOS the only FDA-approved therapy approved for the treatment of oHCM in a pediatric population.¹

CAMZYOS, the most extensively studied cardiac myosin inhibitor (CMI), is supported by the largest body of worldwide evidence in the CMI treatment class, with approvals resulting from several phase III clinical trials (EXPLORER-HCM, VALOR-HCM, SCOUT-HCM), and up to five years of follow up in multiple pivotal trial long-term extensions (EXPLORER-LTE through week 252, VALOR-HCM LTE through week 128), providing additional data on the consistent and sustained results for CAMZYOS to improve symptoms and impact cardiac structure and function.^2,3,4 This landmark moment now makes it available to younger patients.

A First-of-Its-Kind Option for this Patient Population

Obstructive HCM is a chronic, progressive disease characterized by hypercontractility, driven by excess myosin-actin cross-bridges.^2-5 For pediatric patients, symptomatic oHCM can also be associated with substantial morbidity, such as risks for ventricular arrhythmias, heart failure and atrial fibrillation.^1,6 Management of oHCM in pediatric patients has historically included therapies such as beta blockers, calcium channel blockers, disopyramide, and, in some cases, invasive septal reduction procedures, underscoring the need for additional treatment options in this population.^7,8

CAMZYOS is a selective, reversible, allosteric inhibitor of cardiac myosin that targets hypercontractility, the source of oHCM. Reduction in cardiac contractility with CAMZYOS treatment leads to reduced left ventricular outflow tract (LVOT) obstruction, improved energy consumption, and lower cardiac filling pressures – these effects have translated to demonstrated symptom improvements in clinical studies for adults with symptomatic oHCM, enabling them to be more active in their daily lives.^1,2,5

With CAMZYOS now indicated for the treatment of adults and pediatric patients weighing 30 kg (66 lbs) or more with symptomatic oHCM to improve functional capacity and symptoms, health care providers (HCPs) now have an FDA-approved, disease-specific option to offer patients across additional age groups for the first time.

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.

IMPORTANT SAFETY INFORMATION

SCOUT-HCM Establishes Efficacy and Safety in Pediatric Patients

The pediatric approval is based on positive results from SCOUT-HCM (NCT06253221), a phase 3 randomized, double-blind, placebo-controlled international trial enrolling 44 patients (CAMZYOS, n=23; placebo, n=21) aged 12 to <18 years with symptomatic New York Heart Association (NYHA) Class II-III oHCM. The trial includes a 28-week placebo-controlled period, a subsequent 28-week active-treatment period in which placebo patients cross over to CAMZYOS, and an open-label long-term extension of up to 144 weeks.^1,7 Enrolled patients had LVEF ≥60%, Valsalva LVOT peak gradient ≥30 mmHg and a maximal gradient ≥50 mmHg at rest or with provocation. Background oHCM therapy, with a beta blocker, calcium channel blocker, disopyramide, or combinations, was permitted.^1,7

CAMZYOS met the primary endpoint, demonstrating a statistically significant reduction in Valsalva LVOT gradient at week 28 vs placebo (-49 mmHg vs. -1.8 mmHg; least-squares mean difference: −47.98 mmHg; 95% CI: −67.66 to −28.30; P < 0.0001).¹ From baseline to week 28, reductions were observed with CAMZYOS and placebo across select secondary endpoints, including resting LVOT gradient (-41 mmHg for CAMZYOS, +5.3 mmHg for placebo), post-exercise LVOT (-31.2 mmHg for CAMZYOS, +10.5 mmHg for placebo), and maximal LV wall thickness (-2.5 mm for CAMZYOS, -0.7 mm for placebo).⁷ Secondary endpoints were not adjusted for multiplicity and are presented descriptively; these endpoints were not powered for significance.⁷

In SCOUT-HCM, no patients experienced left ventricular ejection fraction (LVEF) below 50% and no adverse events led to treatment discontinuation. No new adverse reactions were identified beyond the safety profile observed in adults. Serious adverse events occurred in 2 patients each in the CAMZYOS (9%) and placebo (10%) groups.^1,7

Building on the Pivotal Trial in Adults

The SCOUT-HCM findings build on the pivotal EXPLORER-HCM trial that supported CAMZYOS’ initial 2022 FDA approval in adults. In that phase 3, randomized, double-blind, placebo-controlled 30-week trial of 251 adults with symptomatic NYHA Class II-III oHCM, 37% of patients taking CAMZYOS (n=123) achieved the primary composite functional endpoint at week 30, compared with 17% on placebo, n=128 (treatment difference, 19%; 95% CI, 9%, 30%; P = .0005).¹

With CAMZYOS, 65% of patients achieved NYHA class improvement, vs. 31% with placebo; treatment difference, 34% (95% CI: 22, 45), P<0.0001. The mean (SD) changes in Valsalva LVOT gradient were -49 (34) mmHg for the CAMZYOS group and -12 (31) mmHg for the placebo group. Eligible patients had LVEF ≥55%, and LVOT peak gradient ≥50 mmHg at rest or with provocation, randomized to a starting dose of 5 mg of CAMZYOS or placebo once daily. Background therapy with a beta blocker or calcium channel blocker was used by 92% of patients in the pooled CAMZYOS (n=119/123) and placebo (n=112/128) groups.^1,2

A Growing Body of Evidence for CAMZYOS

CAMZYOS is supported in adults by EXPLORER long-term extension (EXPLORER-LTE), a single-arm, open-label 5-year extension study (n=231) of the phase 3 EXPLORER-HCM study to evaluate the long-term safety and efficacy of CAMZYOS, starting at 5 mg.² The majority of patients (92%) were on background therapy with a beta blocker or calcium channel blocker at baseline.² Through approximately 5 years of follow-up (week 252), the safety and tolerability profile of CAMZYOS remained consistent with EXPLORER-HCM.⁴

Analyses of exploratory endpoints showed sustained reductions in LVOT obstruction [change from baseline to week 252 (95% CI), Valsalva LVOT gradient, n=184: -55.6 mmHg (-60.8, -50.4)] and improvements in symptoms in patients who remained on treatment at Week 252.⁴ Nearly all patients (97.4%, n=225/231) achieved a Valsalva LVOT gradient ≤ 30 mmHg at any point during the trial, which is the threshold for obstruction in patients with oHCM.⁴

Most patients (69.6%, n=128/184) improved by ≥1 NYHA class; 109 patients (59%) were NYHA Class I (asymptomatic), 39% were NYHA Class II, and 2% (n=3) of patients were NYHA Class III at week 252.⁴ As a single-arm, open-label extension, EXPLORER-LTE data are descriptive, were not powered for statistical significance, and do not include a placebo comparison.⁴ Through Week 252, LVEF <50% had occurred in 11.7% of patients (n=27/231) during the study.⁴ These LTE data are not included in the CAMZYOS U.S. Full Prescribing Information and caution should be used in interpreting the data. Baseline characteristics for EXPLORER-LTE changed due to time and the consolidation of both arms to one cohort.

In the real-world setting, CAMZYOS has been prescribed by more than 5,000 HCPs to over 25,000 adults in the U.S. alone, reflecting growing clinical adoption across the U.S.⁹

Advancing Symptomatic oHCM Care for Younger Patients

For a generation of young patients (weighing 30 kg [66 lbs] or more) with symptomatic oHCM, this milestone opens a new chapter of care, grounded in rigorous clinical evidence, a consistent safety profile, and a growing foundation of real-world experience in adults with NYHA Class II-III oHCM. As scientific research continues to change the course of cardiovascular disease, this approval serves as a powerful reminder that innovation in cardiac care has the potential to reach patients in need earlier in life.

IMPORTANT SAFETY INFORMATION

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.

Please see US Full Prescribing Information, including Boxed WARNING.

References

1. CAMZYOS Prescribing information. Princeton, NJ: Bristol-Myers Squibb Company; 2026.
2. Olivotto I, Oreziak 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
3. Desai MY, Wolski K, Owens A, et al; VALOR-HCM Investigators. Mavacamten in patients with hypertrophic cardiomyopathy referred for septal reduction: week 128 results from VALOR-HCM. Circulation. 2025;151(19):1378-1390. doi:10.1161/CIRCULATIONAHA.124.072445
4. Owens AT et al. Treatment With Mavacamten for Up to 5 Years in Patients With Obstructive Hypertrophic Cardiomyopathy: Final Results From MAVA-LTE. Oral presentation at ESC 2026; August 28-31, 2026; Munich, Germany
5. Anderson RL, Trivedi DV, Sarkar SS, et al. Deciphering the super relaxed state of human β-cardiac myosin and the mode of action of mavacamten from myosin molecules to muscle fibers. Proc Natl Acad Sci U S A. 2018;115(35):E8143-E8152. doi:10.1073/pnas.1809540115
6. 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. doi:10.1016/j.ahj.2025.107283
7. Rossano JW, Canter C, Wolf CM, et al; SCOUT-HCM Investigators. Mavacamten in adolescents with obstructive hypertrophic cardiomyopathy. N Engl J Med. Published online March 29, 2026. doi:10.1056/NEJMoa2601103
8. Ommen SR, Ho CY, Asif IM, et al. 2024 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(23):e1239-e1311.
9. Data on file. Total unique patients started on CAMZYOS. Valid as of July 2026. Princeton, NJ: Bristol-Myers Squibb Company; 2026.

© 2026 MyoKardia, Inc., a Bristol Myers Squibb company.
 CAMZYOS^® and the CAMZYOS Logo are trademarks of MyoKardia, Inc.

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