Camzyos shows sustained efficacy and safety in 56‑week pediatric oHCM trial
Bristol Myers Squibb presented results from the Phase 3 SCOUT‑HCM study of Camzyos (mavacamten) in children 12‑17 years with symptomatic obstructive hypertrophic cardiomyopathy. All 44 enrolled patients completed the 28‑week double‑blind period, and 43 continued into the active‑treatment phase through week 56. The drug lowered the Valsalva left‑ventricular outflow‑tract gradient by an average of 48.1 mmHg and no participant’s left‑ventricular ejection fraction fell below 50% during the study. Safety signals were limited, with a small number of treatment‑related adverse events and two serious events occurring only in the continuous‑treatment group.
Why it matters
The data reinforce Camzyos’ therapeutic value for pediatric oHCM, supporting its recent FDA approval for children weighing ≥30 kg. Investors may see continued demand for the product and potential expansion of the market beyond the adult indication.
Key facts
- 1The SCOUT‑HCM trial enrolled 44 pediatric patients aged 12 to under 18 years. stocktitan.net
- 2Forty‑three participants completed the 28‑week double‑blind placebo‑controlled period and entered the active‑treatment phase through week 56. stocktitan.net
- 3Patients receiving continuous Camzyos experienced a mean reduction of 48.1 mmHg in Valsalva LVOT gradient after 56 weeks. stocktitan.net
- 4Patients who crossed over from placebo to Camzyos at week 28 showed a comparable gradient reduction. stocktitan.net
- 5No patient’s left‑ventricular ejection fraction dropped below 50% during the 56‑week evaluation. stocktitan.net
- 6Treatment‑related adverse events occurred in five patients in the continuous‑treatment arm over the 56‑week period, and in one patient in the crossover arm over 28 weeks. investing.com
Summary written by AlphAI AI Desk from 5 of 5 sources. Not investment advice. Figures are as stated by the linked sources.