Mavacamten

Camzyos was initially approved by the FDA in 2022 for adult patients with symptomatic NYHA class II-III oHCM. Credit: NMK-Studio / Shutterstock.com. The US Food and Drug Administration (FDA) has expanded the approved indication for Bristol Myers Squibb’s (BMS) Camzyos (mavacamten) to treat symptomatic obstructive hypertrophic cardiomyopathy (oHCM) in adults and paediatric patients weighing at least 30kg (66lb). The therapy is indicated to improve functional capacity and symptoms. The regulatory decision was supported by findings from the double-blind, randomised Phase III SCOUT-HCM trial, which assessed the cardiac myosin inhibitor (CMI) against a placebo in 44 adolescent patients aged 12 to under 18 years. Camzyos achieved the primary endpoint by producing a statistically significant reduction in the Valsalva left ventricular outflow tract (LVOT) gradient at week 28 compared with placebo. During the study, no patients recorded a left ventricular ejection fraction (LVEF) below 50%, and no adverse events led to treatment discontinuation. Serious adverse events occurred in two patients in the Camzyos cohort (9%) and two in the placebo group (10%), with no new safety concerns identified beyond the known adult profile. BMS immunology and cardiovascular commercialisation senior vice-president Al Reba said: “Today’s approval of Camzyos for paediatric patients with symptomatic obstructive hypertrophic cardiomyopathy marks an important milestone for young patients and families who are facing a serious cardiovascular disease with significant unmet medical need. “For young patients living with this disease, the burden on daily life can be substantial during a critical stage of development, and we are proud to help bring an innovative new treatment option to this community. “This approval also strengthens the growing body of clinical evidence supporting Camzyos, which is the most extensively studied cardiac myosin inhibitor with up to five years of follow-up in adults, and its role in the treatment of oHCM.” Camzyos was initially approved by the FDA in 2022 for adult patients with symptomatic NYHA class II-III oHCM, with more than 5,000 healthcare providers prescribing it to over 25,000 US patients to date. The therapy has gained regulatory clearances across more than 60 countries, supported by Phase III trials conducted in China, Japan, and other international regions, including EXPLORER-CN, HORIZON-HCM, and SCOUT-HCM. The company is in discussions with other international regulatory authorities regarding potential paediatric approvals. In August 2026, the FDA granted accelerated approval to BMS’ Zenbexus (iberdomide), along with daratumumab and hyaluronidase-fihj and dexamethasone (ZDd), to treat adult multiple myeloma patients.

CAMZYOS is the only FDA-approved therapy for the treatment of oHCM in a pediatric population Approval based on positive results of Phase 3 SCOUT-HCM trial, building on the extensive clinical evidence for CAMZYOS in adults with oHCM PRINCETON, N.J.--(BUSINESS WIRE)-- $BMY #CAMZYOS -- Bristol Myers Squibb (NYSE: BMY) today announced that the U.S. Food and Drug Administration (FDA) approved CAMZYOS ® (mavacamten) for the treatment of symptomatic obstructive hypertrophic cardiomyopathy (oHCM) to improve functional capacity and symptoms in adults and pediatric patients weighing 30 kg (66 lbs) or more. CAMZYOS now has the broadest indication of any cardiac myosin inhibitor (CMI). This expanded indication offers a new treatment option for patients with significant unmet need. 1 “Today’s approval of CAMZYOS for pediatric patients with symptomatic obstructive hypertrophic cardiomyopathy marks an important milestone for young patients and families who are facing a serious cardiovascular disease with significant unmet medical need,” said Al Reba, senior vice president, Immunology & Cardiovascular Commercialization, Bristol Myers Squibb. “For young patients living with this disease, the burden on daily life can be substantial during a critical stage of development, and we are proud to help bring an innovative new treatment option to this community. This approval also strengthens the growing body of clinical evidence supporting CAMZYOS, which is the most extensively studied cardiac myosin inhibitor with up to five years of follow up in adults, and its role in the treatment of oHCM.” This FDA approval is based on positive results from the Phase 3 SCOUT-HCM trial, which evaluated the efficacy and safety of CAMZYOS (n=23) versus placebo (n=21) in patients aged 12 to 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 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. Contacts Bristol Myers Squibb Media Inquiries: media@bms.com Investors: investor.relations@bms.com Read full story here