The FDA has accepted a supplemental New Drug Application (sNDA) from Bristol Myers Squibb for Camzyos (mavacamten), which would allow treatment of symptomatic obstructive hypertrophic cardiomyopathy (oHCM) in adolescents.
Acceptance of the sNDA for priority review is based on positive results from the phase 3 SCOUT-HCM trial. The drug met its primary endpoint, reducing Valsalva left ventricular outflow tract (LVOT) gradient by a statistically-significant and clinically-meaningful degree at Week 28 compared to a placebo. The drug also showed a safety profile consistent with that in adults — it has approval for treating adults with symptomatic New York Heart Association (NYHA) class II-III oHCM.
In April 2022, the FDA approved Camzyos capsules for the treatment of adults with symptomatic New York Heart Association (NYHA) class II-III obstructive hypertrophic cardiomyopathy (obstructive HCM) to improve functional capacity and symptoms. Camzyos is the first and only FDA-approved allosteric and reversible inhibitor selective for cardiac myosin that targets the underlying pathophysiology of obstructive HCM.
If the sNDA receives approval, Camzyos will become the first pharmacological therapy for oHCM in patients aged 12 to 17 years. Its PDUFA date is set for September 30, 2026.