FDA approves expanded CAMZYOS use for symptomatic oHCM in adults and kids
The U.S. Food and Drug Administration (FDA) has broadened the use of CAMZYOS (mavacamten), a drug from Bristol Myers Squibb, to treat symptomatic obstructive hypertrophic cardiomyopathy (oHCM) in adults and in pediatric patients weighing 30 kg (about 66 pounds) or more. The expansion follows positive results from a Phase 3 trial and adds a treatment option for people with significant breathing and exercise difficulties caused by oHCM.
What changed and what it means
The FDA approved CAMZYOS for a broader group: both adults and children who weigh at least 30 kg now have an FDA-approved option to improve functional capacity and symptoms from oHCM. This makes CAMZYOS the most broadly indicated cardiac myosin inhibitor among its class. The change is based on Phase 3 SCOUT-HCM trial data showing benefit in reducing symptoms and improving how far patients can walk or exercise before symptoms limit them.
CAMZYOS was already approved for adults with oHCM; the new approval covers pediatric patients as well, expanding access to a medicine that directly targets the heart muscle’s abnormal contraction. The FDA notes this population previously had fewer approved options for treatment beyond lifestyle changes or procedures.
Who is affected
Adults with symptomatic obstructive hypertrophic cardiomyopathy (oHCM) and pediatric patients weighing at least 30 kg who have symptoms such as shortness of breath, chest pain, or exercise intolerance may be eligible for CAMZYOS under the expanded indication. oHCM is a condition where thickened heart muscle can hinder normal heart function, causing symptoms during activity or at rest. The new label does not imply all patients with oHCM should start CAMZYOS; eligibility is determined by doctors based on individual health status and risk factors.
Practical next steps for patients and clinicians
Clinicians should review the patient’s weight (minimum 30 kg) and symptom profile to assess suitability for CAMZYOS under the expanded indication. Patients currently taking CAMZYOS should consult their prescribing physician about the new pediatric eligibility and any changes to monitoring. Common monitoring for CAMZYOS includes regular follow-up visits to assess symptoms, exercise tolerance, and heart function, as well as safety checks for potential drug interactions and adverse effects. Patients should not stop CAMZYOS without medical advice.
For those not yet treated, doctors may consider CAMZYOS as part of a shared decision-making process that weighs potential benefits against other treatment options, such as lifestyle management or procedures, depending on the patient’s condition and preferences.
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