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Getting the latest healthcare news for you
Getting the latest healthcare news for you
A landmark phase 3 trial just cracked open a door that's been shut for years. The ACACIA-HCM trial showed aficamten significantly improved symptoms and exercise capacity in nonobstructive hypertrophic cardiomyopathy (HCM) — a condition with zero approved therapies. Presented at ESC 2026 and published in the NEJM, these are the first positive controlled trial results ever in this patient population.
For patients with nonobstructive hypertrophic cardiomyopathy (HCM), there have been no approved drugs — until now. The phase 3 ACACIA-HCM trial, presented at the European Society of Cardiology Congress 2026 and simultaneously published in the New England Journal of Medicine, showed that aficamten (Myqorzo), a cardiac myosin inhibitor already approved for obstructive HCM, significantly improved both symptom burden and exercise capacity in nonobstructive HCM patients — a historic first in a controlled trial.
Across 518 adults at 160 sites in 19 countries, aficamten beat placebo on both primary endpoints at 36 weeks, with benefits emerging as early as 12 weeks. A responder analysis found that 53% of aficamten-treated patients achieved a meaningful clinical response across multiple domains, versus just 13% on placebo. The drug was associated with a higher rate of LVEF reductions and early discontinuations, though all three deaths in the trial occurred in the placebo arm.
By the numbers:
Why it matters: Nonobstructive HCM is a progressive, debilitating disease with no proven pharmacologic treatments beyond heart transplantation. These results position aficamten as a potential first-ever approved therapy for this population, pending regulatory review — a meaningful step forward for patients who currently have nowhere else to turn.
