PRINCETON, N.J., June 1, 2026
Bristol Myers Squibb has achieved a significant regulatory milestone after the U.S. Food and Drug Administration (FDA) accepted for Priority Review its supplemental New Drug Application (sNDA) for Camzyos® (mavacamten) as a treatment for adolescents aged 12 to under 18 years with symptomatic obstructive hypertrophic cardiomyopathy (oHCM). If approved, Camzyos could become the first pharmacological therapy specifically indicated for adolescents with oHCM, addressing a substantial unmet medical need in a patient population that currently relies on therapies with significant limitations. The FDA has assigned a Prescription Drug User Fee Act (PDUFA) target date of September 30, 2026, positioning the therapy for a potentially important expansion of its clinical use beyond adults. The application is supported by positive results from the pivotal Phase 3 SCOUT-HCM clinical trial, which demonstrated meaningful improvements in cardiac function while maintaining a safety profile consistent with that previously observed in adults.
Phase 3 SCOUT-HCM Trial Demonstrates Significant Clinical Benefit
The FDA submission is based on data from the Phase 3 SCOUT-HCM trial, a randomized, double-blind, placebo-controlled international study evaluating Camzyos in adolescent patients with symptomatic obstructive hypertrophic cardiomyopathy. The study successfully met its primary endpoint, showing a clinically meaningful and statistically significant reduction in Valsalva left ventricular outflow tract (LVOT) gradient at Week 28 compared with placebo. The reduction in LVOT obstruction is considered a critical marker of improved cardiac performance and symptom control in patients with oHCM.
Importantly, the trial demonstrated a favorable safety profile, with no participants experiencing left ventricular ejection fraction below 50%, a key measure of cardiac function. The positive findings were presented at the American College of Cardiology (ACC) Annual Scientific Session & Expo 2026 and simultaneously published in The New England Journal of Medicine, highlighting the scientific significance of the results.
Potential First-in-Class Treatment for Adolescents with oHCM
Advertisement
Hypertrophic cardiomyopathy is a serious inherited cardiac condition characterized by abnormal thickening of the heart muscle, which can obstruct blood flow and significantly impair exercise tolerance and quality of life. Adolescents diagnosed with obstructive HCM often face limitations in physical activity and may experience symptoms such as fatigue, chest pain, shortness of breath, and dizziness.
Existing treatment options frequently involve beta-blockers, calcium channel blockers, or invasive procedures, all of which carry notable limitations and risks. Camzyos, a selective cardiac myosin inhibitor, directly targets the underlying hypercontractility responsible for oHCM by reducing excessive cardiac muscle contraction and improving blood flow through the heart. By addressing the root cause of the disease rather than merely managing symptoms, Camzyos represents a potential paradigm shift in the treatment of adolescent oHCM patients. Bristol Myers Squibb believes the therapy could transform care for younger patients facing a lifelong cardiovascular condition with limited therapeutic alternatives.
Expanding Leadership in Cardiovascular Innovation
Camzyos has already established itself as a leading therapy in adult obstructive hypertrophic cardiomyopathy and is currently approved in more than 60 countries and regions worldwide. In the United States alone, nearly 25,000 patients have been prescribed the therapy through more than 4,500 healthcare providers. The Priority Review designation underscores both the urgency of addressing unmet needs in adolescent oHCM and the potential clinical impact of expanding access to this innovative treatment.
As Bristol Myers Squibb continues to advance its cardiovascular portfolio, the Camzyos adolescent program highlights the company’s commitment to delivering transformative therapies that improve patient outcomes and redefine standards of care. With regulatory review now underway, healthcare providers, patients, and families will closely monitor the FDA’s decision as the treatment moves one step closer to becoming the first approved pharmacological option for adolescents living with symptomatic obstructive hypertrophic cardiomyopathy.
Source: Bristol Myers Squibb press release
