Cytokinetics Receives Positive European Regulatory Opinion for MYQORZO in Obstructive Hypertrophic Cardiomyopathy
核心洞察
The European Medicines Agency (搜索)'s Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion recommending marketing authorization for MYQORZO (搜索) (aficamten) in the European Union for treating symptomatic obstructive hypertrophic cardiomyopathy (搜索).
The recommendation is based on robust clinical evidence from the pivotal Phase 3 SEQUOIA-HCM trial, which demonstrated that MYQORZO (搜索) significantly improved exercise capacity by 1.8 ml/kg/min compared to placebo over 24 weeks.
A final decision from the European Commission is anticipated in the first quarter of 2026, while the FDA is reviewing the drug with a target action date of December 26, 2025.
Cytokinetics (搜索) announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (搜索) (EMA) has adopted a positive opinion recommending marketing authorization in the European Union for MYQORZO (搜索)® (aficamten), a cardiac myosin (搜索) inhibitor, for the treatment of symptomatic (New York Heart Association, NYHA, class II-III) obstructive hypertrophic cardiomyopathy (搜索) (oHCM) in adult patients. A final decision is anticipated from the European Commission in the first quarter of 2026.
"We are pleased with CHMP's positive recommendation based on the robust clinical evidence from SEQUOIA-HCM that demonstrated the safety and efficacy of MYQORZO (搜索) in patients with oHCM, and we are accelerating commercial readiness activities accordingly," said Robert I. Blum, Cytokinetics (搜索)' President and Chief Executive Officer.
Pivotal Trial Results Drive Regulatory Decision
The CHMP recommendation is based on positive results from the pivotal Phase 3 clinical trial, SEQUOIA-HCM, published in the New England Journal of Medicine. The trial demonstrated robust efficacy, safety, and clinically meaningful benefits across symptoms, exercise capacity, hemodynamics, and biomarker endpoints.
Results from SEQUOIA-HCM showed that treatment with MYQORZO (搜索) for 24 weeks significantly improved exercise capacity compared to placebo, increasing peak oxygen uptake (pVO2) measured by cardiopulmonary exercise testing (CPET) by 1.8 ml/kg/min compared to baseline in patients treated with MYQORZO versus 0.0 ml/kg/min in patients treated with placebo (least square mean difference [95% CI] of 1.74 mL/kg/min [1.04 - 2.44]; p=0.000002).
The treatment effect of MYQORZO (搜索) was consistent across all prespecified subgroups, including age, sex, patient baseline characteristics, and in patients receiving or not receiving background beta-blocker therapy.
Safety Profile Demonstrates Tolerability
MYQORZO (搜索) was well-tolerated in the clinical trial, with no instances of worsening heart failure (搜索) or treatment interruptions due to low left ventricular ejection fraction (LVEF). Treatment emergent serious adverse events occurred in 5.6% and 9.3% of patients on MYQORZO and placebo, respectively. Core lab echocardiographic LVEF was observed to be <50% in 5 patients (3.5%) on MYQORZO compared to 1 patient (0.7%) on placebo.
Mechanism of Action and Drug Development
MYQORZO (搜索) is an investigational selective, small molecule cardiac myosin (搜索) inhibitor discovered following an extensive chemical optimization program conducted with careful attention to therapeutic index and pharmacokinetic properties. The drug was designed to reduce the number of active actin-myosin cross bridges during each cardiac cycle and consequently suppress the myocardial hypercontractility associated with HCM.
In preclinical models, MYQORZO (搜索) reduced myocardial contractility by binding directly to cardiac myosin (搜索) at a distinct and selective allosteric binding site, thereby preventing myosin from entering a force producing state.
Regulatory Status and Ongoing Development
Aficamten is currently under regulatory review in the U.S., where the Food & Drug Administration (搜索) (FDA) is reviewing a New Drug Application (NDA) with a Prescription Drug User Fee Act (PDUFA) target action date of December 26, 2025. Additionally, the Center for Drug Evaluation (CDE) of the China National Medical Products Administration (搜索) (NMPA) is reviewing an NDA for aficamten with Priority Review.
MYQORZO (搜索) received Breakthrough Therapy Designation for the treatment of symptomatic HCM from the FDA and for the treatment of symptomatic obstructive HCM from the NMPA in China.
Aficamten is also currently being evaluated in ACACIA-HCM, a Phase 3 clinical trial in patients with non-obstructive HCM; CEDAR-HCM, a clinical trial in a pediatric population with oHCM; and FOREST-HCM, an open-label extension clinical study in patients with HCM.
Disease Burden and Unmet Medical Need
HCM is the most common monogenic inherited cardiovascular disorder, affecting approximately 1 out of 500 Europeans, according to the European Society of Cardiology guidelines. Hypertrophic cardiomyopathy (搜索) is a disease in which the heart muscle (myocardium) becomes abnormally thick (hypertrophied). The thickening of cardiac muscle leads to the inside of the left ventricle becoming smaller and stiffer, and thus the ventricle becomes less able to relax and fill with blood.
Two-thirds of patients with HCM have obstructive HCM (oHCM), where the thickening of the cardiac muscle leads to left ventricular outflow tract (LVOT) obstruction, while one-third have non-obstructive HCM (nHCM), where blood flow isn't impacted, but the heart muscle is still thickened.
People with HCM are at high risk of also developing cardiovascular complications including atrial fibrillation (搜索), stroke (搜索) and mitral valve disease (搜索). People with HCM are at risk for potentially fatal ventricular arrhythmias and it is one of the leading causes of sudden cardiac death in younger people or athletes.
"This positive opinion from the CHMP is an important milestone toward bringing a new treatment option with distinct attributes to patients with oHCM," said Iacopo Olivotto, M.D., Head of the Cardiomyopathy Center and Professor of Cardiovascular Medicine at the University of Florence, Italy.