Heart failure & cardiomyopathiesRandomised controlled trial

Aficamten or Metoprolol Monotherapy for Obstructive Hypertrophic Cardiomyopathy

N Engl J MedPublished

  • Randomised controlled trial
  • international
  • double-blind
  • double-dummy

Authors' conclusion

Among patients with symptomatic obstructive HCM, aficamten monotherapy was superior to metoprolol monotherapy in improving peak oxygen uptake and hemodynamics and decreasing symptoms.

Study context

Population
Adults with symptomatic obstructive hypertrophic cardiomyopathy (HCM)
Participants
88 vs 87
Comparison
Aficamten monotherapy vs metoprolol monotherapy
  • Aficamtenn = 88
  • Metoprololn = 87reference
Primary endpoint
Change in peak oxygen uptake
Follow-up
Week 24

Primary result

As reported in the abstract

24 weeks

Least-squares mean between-group difference

2.3 ml per kilogram per minute

95% CI 1.5–3.1

P < 0.001

By group

Aficamten

1.1

95% CI 0.5–1.7 · ml per kilogram of body weight per minute

Metoprolol

−1.2

95% CI −1.7–-0.8 · ml per kilogram per minute

Aficamten vs Metoprolol
least-squares mean between-group difference 2.3 ml per kilogram per minute (95% CI 1.5–3.1); P < 0.001

Secondary outcomes

New York Heart Association (NYHA) class, Kansas City Cardiomyopathy Questionnaire clinical summary score (KCCQ-CSS), LV outflow tract gradient, NT-proBNP, left atrial volume index · week 24

Significantly greater improvements with aficamten than with metoprolol

LV mass index · week 24

No significant difference

Safety

Adverse events

Appeared similar in the two treatment groups

Original publication

Aficamten or Metoprolol Monotherapy for Obstructive Hypertrophic Cardiomyopathy. N Engl J Med. Published 30 August 2025. doi:10.1056/nejmoa2504654

Summarised from the published abstract; every number is checked against it