Aficamten gains breakthrough designation for obstructive HCM
Cytokinetics announced that the FDA has granted breakthrough therapy designation for aficamten, a next-generation cardiac myosin inhibitor, for the treatment of symptomatic obstructive hypertrophic cardiomyopathy.
The decision is based on the results from the REDWOOD-HCM phase 2 trial of aficamten (Cytokinetics) in patients with symptomatic obstructive HCM, which were presented at the Heart Failure Society of America Annual Scientific Meeting in September 2021. For the REDWOOD-HCM trial, researchers assigned 41 patients with symptomatic obstructive HCM, left ventricular ejection fraction of 60% or more and resting LV outflow tract gradient of 50 mm Hg or more to aficamten or placebo.
As Healio previously reported, treatment with aficamten was associated with improvements in HF symptoms and hemodynamics at 10 weeks and resulted in no treatment-related serious adverse events or excess incidence of adverse events compared with placebo.
Breakthrough therapy designation is designed to expedite the development and review of therapies that are intended to treat a serious condition where preliminary clinical evidence indicates potentially substantial improvement over available therapies on a clinically significant endpoint.