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news.The US Food and Drug Administration (FDA) has accepted Bristol Myers Squibb’s (BMS) supplemental new drug application (sNDA) for Camzyos (mavacamten) to treat adolescents aged 12 to under 18 years with symptomatic obstructive hypertrophic cardiomyopathy (oHCM).
Camzyos is currently approved for symptomatic NYHA Class II-III oHCM in adults to improve functional capacity and symptoms. Credit: NMK-Studio / Shutterstock.com.
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The regulator granted the application priority review for this indication, assigning a Prescription Drug User Fee Act (PDUFA) date of 30 September 2026.
Camzyos is currently approved for symptomatic New York Heart Association (NYHA) Class II-III oHCM in adults to improve functional capacity and symptoms and has so far been prescribed by more than 4,500 healthcare providers to approximately 25,000 patients in the US.
BMS executive vice-president, chief medical officer and head of development Cristian Massacesi said: “The acceptance of this NDA allows us the potential to extend our leadership in oHCM to a younger patient population with a high unmet medical need.
“We are encouraged by the possibility of bringing this potentially paradigm-shifting treatment to adolescents at a time when a condition like oHCM can significantly impact both physical and emotional aspects of their lives.”
The new application was based on results from the randomised, double-blind, placebo-controlled global Phase III SCOUT-HCM trial.
SCOUT-HCM met its primary endpoint, showing a clinically meaningful and statistically significant reduction from baseline in Valsalva left ventricular outflow tract (LVOT) gradient at week 28 compared to placebo.
The safety profile in adolescents was consistent with adults, and no patient experienced left ventricular ejection fraction (LVEF) below 50%.
The trial enrolled 44 adolescents aged 12 to under 18 years with symptomatic oHCM.
It features three successive treatment periods over up to 200 weeks: an initial 28-week placebo-controlled period, a subsequent 28-week active-treatment phase, and an open-label extension lasting 144 weeks.
Hypertrophic cardiomyopathy is a primary cardiac disorder that may arise from genetic or idiopathic causes. Adolescents with oHCM often experience reduced exercise tolerance and related morbidity.
Last month, BMS signed a strategic agreement with Anthropic to implement its Claude platform across its global operations.
