The U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation to aficamten, a cardiac myosin inhibitor, for the treatment of symptomatic obstructive hypertrophic cardiomyopathy (oHCM). Hypertrophic cardiomyopathy (HCM) is a condition characterized by the abnormal thickening of the heart muscle, leading to the inner ventricles becoming smaller and less flexible and causing them to be unable to relax and fill with blood. Due to this thickening, the heart’s function of pumping blood becomes restricted, resulting in chest pain, shortness of breath, and potential for severe outcomes related to stroke, arrhythmias, and death. The FDA grants Breakthrough Therapy Designation in order to expedite the development and review of therapeutics that are designed to treat serious conditions when preliminary clinical evidence suggests that the drug may offer significant improvement when compared to currently available therapeutics in critical clinical endpoints. This designation given to aficamten is based on the results of the REDWOOD-HCM Phase II clinical trial in patients with symptomatic oHCM which showed that when compared to baseline, treatment with aficamten resulted in clinically significant reductions in the average resting ventricular outflow tract pressure gradient, a clinical marker of oHCM severity. The drug is designed to inhibit the number of active cardiac myosin cross bridges during cardiac cycles, which are responsible for the contractibility of the muscle, and thus suppress the hypercontractility that is characteristic of HCM. As a biopharmaceutical company committed to discovering, developing, and commercializing first in-class muscle activators and inhibitors as treatments for conditions marked by debilitating disruptions in muscle performance.  

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