BMS’s Mavacamten Shows Significant LVOT Reduction in First Adolescent oHCM Phase III Study

Bristol‑Myers Squibb (BMS, NYSE: BMY) announced positive topline results from the Phase 3 SCOUT‑HCM study evaluating mavacamten in adolescent patients (aged 12 to <18 years) with symptomatic obstructive hypertrophic cardiomyopathy (oHCM), marking the first cardiac myosin inhibitor (CMI) study in this population.

Regulatory Milestone

Drug Profile & Mechanism of Action

• Mechanism: Cardiac myosin inhibitor (CMI) that reduces myocardial contractility and LVOT obstruction without compromising cardiac output
• Global Registration: Approved in over 50 countries and regions across five continents for adult oHCM
• Innovation: First CMI studied in adolescent oHCM population, addressing a critical unmet need
• Clinical Significance: Demonstrates effective improvement in LVOT obstruction, a key pathophysiological feature of oHCM

Clinical Evidence – Phase 3 SCOUT‑HCM

Market Opportunity & Competitive Landscape

• Competitive Gap: No FDA‑approved pharmacologic therapy specifically for adolescent oHCM; mavacamten would be first‑in‑class
• Next Steps: J&J plans sNDA submission to FDA in Q2 2026; potential PDUFA date Q1 2027

Strategic Positioning

• Manufacturing: Established global supply chain with three production sites capable of supporting pediatric population
• Commercial Readiness: Existing cardiology sales force can seamlessly expand to adolescent indication
• Regulatory Pathway: Breakthrough Therapy Designation likely given first‑in‑class status and unmet need
• Pipeline Expansion: Data supports potential expansion to pediatric population (age 8‑12) and non‑obstructive HCM

Forward‑Looking Statements
This brief contains forward‑looking statements regarding regulatory submissions, commercial forecasts, and market penetration for mavacamten in adolescent oHCM. Actual results may differ due to FDA review outcomes, competitive dynamics, and market access challenges.-Fineline Info & Tech