FDA Approves Expanded Indication for CAMZYOS (mavacamten) for the Treatment of Symptomatic Obstructive Hypertrophic Cardiomyopathy (oHCM) in Adults and Pediatric Patients – BMS
Bristol Myers Squibb announced that the 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.
“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 <18 years with symptomatic NYHA (New York Heart Association) Class II-III obstructive hypertrophic cardiomyopathy. Enrolled patients had LVEF ≥60%, Valsalva LVOT peak gradient ≥30 mmHg and a maximal gradient ≥50 mmHg at rest or with provocation, and background oHCM therapy with a beta blocker, calcium channel blocker, disopyramide, or combinations was permitted.
CAMZYOS met the trial’s primary endpoint, demonstrating a statistically significant reduction in Valsalva left ventricular outflow tract (LVOT) gradient at Week 28 compared with placebo.
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.
“The FDA approval of CAMZYOS for pediatric patients with symptomatic obstructive hypertrophic cardiomyopathy represents a landmark moment for pediatric cardiology. For the first time, children with this serious condition have a therapy that is FDA-approved to reduce left ventricular outflow tract obstruction,” said Joseph Rossano, MD, Principal Investigator of SCOUT-HCM and Chief of the Division of Cardiology at Children’s Hospital of Philadelphia. “This historic milestone was made possible by the outstanding efforts of the international SCOUT-HCM investigators and participating families whose commitment has transformed clinical research into a therapy with potential to meaningfully improve the lives of pediatric patients with symptomatic obstructive hypertrophic cardiomyopathy.”
“I cannot help but think back to my 12-year-old self receiving my diagnosis and being told there were no treatment options approved specifically for my disease,” said Lisa Salberg, CEO and founder of the Hypertrophic Cardiomyopathy Association. “This is truly a milestone moment for patients and families. Having a targeted therapy for cardiac myosin is a significant breakthrough, and it’s a welcome change to bring this approval to a younger population. Thank you to all of the researchers, scientists, and the entire HCM ecosystem that has helped bring this new therapy to more patients.”
About the Phase 3 SCOUT-HCM Trial
SCOUT-HCM (NCT06253221) is a Phase 3 randomized, double-blind, placebo-controlled, international trial that enrolled 44 adolescent patients (12 years to <18 years old) with symptomatic oHCM. The trial includes three treatment periods totaling up to 200 weeks: a 28-week placebo-controlled period, followed by a 28-week active-treatment period (when patients randomized to placebo cross over to CAMZYOS), and an open-label long-term extension period for up to 144 weeks. 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. Patients were randomized in a 1:1 ratio and administered either CAMZYOS, starting dose of 2.5 mg CAMZYOS for body weight 35 to <45 kg, 5 mg CAMZYOS for body weight ≥45 kg, or placebo, once daily for 28 weeks. Dose adjustment was based on clinical echocardiogram parameters. The primary endpoint is change from baseline to Week 28 in Valsalva LVOT gradient (mmHg). Secondary endpoints include change from baseline to Week 28 in efficacy parameters of resting and postexercise LVOT gradients, peak oxygen consumption (proportion of patients achieving an increase from baseline in pVO2), symptoms (proportion of patients with at least 1 NYHA Class improvement) and HCM Symptom Questionnaire Shortness of Breath domain (HCMSQ SoB), plus safety and pharmacokinetic parameters.
