Cytokinetics Announces FDA Approval of MYQORZO™ (aficamten) for the Treatment of Adults with Symptomatic Obstructive Hypertrophic Cardiomyopathy to Improve Functional Capacity and Symptoms

Core Insights - The U.S. FDA has approved MYQORZO™ (aficamten) for the treatment of adults with symptomatic obstructive hypertrophic cardiomyopathy (oHCM), marking it as the company's first FDA-approved medicine [1][2][22] - MYQORZO is an allosteric and reversible inhibitor of cardiac myosin motor activity, designed to improve functional capacity and symptoms in patients with oHCM [1][22] - The approval is based on the positive results from the pivotal Phase 3 clinical trial, SEQUOIA-HCM, which demonstrated significant improvements in exercise capacity and symptom reduction [6][5] Company Overview - Cytokinetics is a specialty cardiovascular biopharmaceutical company focused on developing new medicines for diseases of cardiac muscle dysfunction [27] - The company has over 25 years of experience in scientific innovations in muscle biology and is advancing a pipeline that includes MYQORZO and other investigational drugs [27] Product Details - MYQORZO is available in 5 mg, 10 mg, 15 mg, and 20 mg tablet forms and is expected to be available in the U.S. in the second half of January 2026 [1][3] - The drug is associated with a straightforward dosing regimen and does not require drug-drug interaction monitoring, contributing to its predictable safety profile [2] Clinical Trial Insights - In the SEQUOIA-HCM trial, MYQORZO improved peak oxygen uptake (pVO2) by 1.8 mL/kg/min compared to placebo, with a statistically significant p-value of 0.000002 [6] - The treatment was well-tolerated, with serious adverse events occurring in 5.6% of patients on MYQORZO compared to 9.3% on placebo [7] Safety Information - MYQORZO carries a Boxed WARNING for the risk of heart failure due to its effect on reducing left ventricular ejection fraction (LVEF) [10][22] - Echocardiogram assessments are required prior to and during treatment to monitor for systolic dysfunction, with specific dosing adjustments based on LVEF levels [11][15] Market Context - Hypertrophic cardiomyopathy (HCM) is the most common monogenic inherited cardiovascular disorder, with over 300,000 diagnosed patients in the U.S. and an estimated additional 400,000-800,000 undiagnosed [25][24] - Approximately half of HCM patients have obstructive HCM (oHCM), highlighting the need for effective treatment options like MYQORZO [25]