The hypertrophic cardiomyopathy treatment drug 'Camzyos (generic name mavacamten)' is expected to be prescribed for adolescent patients as well. Bristol Myers Squibb (BMS) announced plans to discuss expanding indications after meeting primary endpoints in a Phase 3 clinical trial for patients aged 12-17 with obstructive hypertrophic cardiomyopathy (oHCM).
BMS announced on the 11th (local time) that it achieved the primary endpoint in the 'SCOUT-HCM' Phase 3 clinical trial involving 44 adolescent oHCM patients (aged 12 to under 18). It is noted that it is rare for a myosin inhibitor class drug to succeed in Phase 3 trials for adolescent patient groups.
In this clinical trial, Camzyos significantly reduced the degree of obstruction in the pathway for blood to exit the heart (LVOT gradient) after 28 weeks of treatment compared to before treatment. BMS explained that statistically significant improvements were also confirmed in key secondary endpoints such as LVOT gradient at rest and after exercise, peak oxygen uptake (peak VO₂), symptoms, and health status.
The safety profile was generally similar to that of adults. The company stated that no new signals of adverse effects were observed in adolescent patients.
SCOUT-HCM is a long-term study that will follow up for up to 200 weeks (about 4 years). After a 28-week placebo-controlled period, there will be a 28-week active treatment period, during which patients in the placebo group will also be switched to Camzyos. Following this, an open-label extension evaluation will be conducted for up to 144 weeks.
Joseph Rosano, head of the cardiology department at the Children’s Hospital of Philadelphia (CHOP), who participated in the study, stated, "Treatment for adolescent oHCM often relies on symptom-controlling medications or invasive surgeries," adding, "If approved, it could become an important treatment option."
Hypertrophic cardiomyopathy is a condition where the heart muscle thickens, preventing the heart from relaxing sufficiently and obstructing blood flow. Symptoms include shortness of breath, chest pain, and palpitations, and it is known to be associated with the risk of sudden death, especially during exercise.
Camzyos is a 'myosin inhibitor' that reduces excessive contraction by decreasing the actin-myosin binding that triggers heart muscle contraction, thereby restoring relaxation function. Unlike existing treatments that only alleviate symptoms, it is evaluated as the first treatment targeting the disease mechanism, and it is referred to as 'oral surgery' as it is expected to have effects comparable to surgery with just one pill a day. It received approval from the Ministry of Food and Drug Safety in Korea in May 2023.
Competition is also intensifying. The FDA approved Cytokinetics' obstructive hypertrophic cardiomyopathy treatment 'Mavacamten' (generic name apicamtent) in December 2025, adding another drug in the same class to the market.
