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Heart failure & cardiomyopathiesRandomised controlled trial

Aficamten for Symptomatic Nonobstructive Hypertrophic Cardiomyopathy

N Engl J MedPublished

  • Randomised controlled trial
  • phase 3
  • multinational
  • double-blind
  • placebo-controlled
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Authors' conclusion

Among patients with symptomatic nonobstructive HCM, treatment with aficamten resulted in a significantly greater change in exercise capacity and patient-reported health status than placebo at 36 weeks.

Study context

Population
Adults with symptomatic nonobstructive hypertrophic cardiomyopathy (HCM); mean age 55.1 years, 53.6% women
Participants
258 vs 259
Comparison
Aficamten vs placebo
  • Aficamtenn = 258
  • Placebon = 259reference
Primary endpoint
Dual primary: change from baseline in peak oxygen uptake and in KCCQ-CSS (0–100, higher = better health status)
Follow-up
Week 36

Primary result

As reported in the abstract

36 weeks

Aficamten

11.4 points (95% CI, 9.6 to 13.2)

Kansas City Cardiomyopathy Questionnaire clinical summary score (KCCQ-CSS) change · Peak oxygen uptake mean change 0.64 ml/kg/min (95% CI, 0.32 to 0.95)

Placebo

8.4 points (95% CI, 6.6 to 10.2)

KCCQ-CSS change · Peak oxygen uptake mean change −0.03 ml/kg/min (95% CI, −0.35 to 0.28)

Aficamten vs PlaceboLeast-squares mean difference, points (95% CI)P value
KCCQ-CSS
3.0(0.5–5.5)
P = 0.02
Aficamten vs PlaceboLeast-squares mean difference, ml/kg/min (95% CI)P value
peak oxygen uptake
0.67(0.22–1.11)
P = 0.003

Safety

Reversible reduction in LVEF to <50%

Serious adverse events

Original publication

Aficamten for Symptomatic Nonobstructive Hypertrophic Cardiomyopathy. N Engl J Med. Published 28 August 2026. doi:10.1056/nejmoa2603021
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Summarised from the published abstract; every number is checked against it