LianBio (Nasdaq: LIAN), a biotechnology company dedicated to bringing innovative medicines to patients in China and other major Asian markets, today announced that the Center for Drug Evaluation (CDE) of the National Medical Products Administration (NMPA) granted Breakthrough Therapy Designation in China for mavacamten for the treatment of patients with obstructive hypertrophic cardiomyopathy (oHCM).
Breakthrough Therapy Designation was supported by data from the global Phase 3 EXPLORER-HCM trial of mavacamten in oHCM patients with New York Heart Association (NYHA) class II–III symptoms. In the EXPLORER-HCM trial, mavacamten met all primary and secondary endpoints with statistical significance and demonstrated clinically meaningful improvement in functional status, symptoms, and quality of life.
“In a global Phase 3 trial, mavacamten demonstrated the potential to change the course of disease and restore heart function in patients with oHCM,” said Yizhe Wang, Ph.D., Chief Executive Officer of LianBio. “LianBio’s Phase 3 trial of mavacamten in Chinese patients with symptomatic oHCM is ongoing, and we believe Breakthrough Therapy Designation will allow us to continue to accelerate the development of mavacamten in China. We look forward to working with CDE to deliver this important medicine to patients in need.”
There are approximately 1.1 million1 to 2.8 million2 people living with HCM in China, with no existing effective drug treatment options beyond limited symptomatic relief.
Breakthrough Therapy Designation in China is designed to expedite the development and review of investigational therapeutics for the treatment of serious and life-threatening diseases that have demonstrated preliminary evidence indicating advantages of the therapy over currently available treatment options. Drugs with Breakthrough Therapy Designation can access additional communication channels and technical guidance from CDE in addition to potential accelerated approval pathways.
LianBio is currently conducting EXPLORER-CN, a Phase 3 multicenter, double-blind, randomized, placebo-controlled registrational study designed to evaluate the safety and efficacy of mavacamten in Chinese patients with symptomatic oHCM. EXPLORER-CN will enroll approximately 81 patients. The primary endpoint is the change in Valsalva left ventricular outflow tract (LVOT) gradient from baseline to week 30. Eligible patients will continue in a long-term extension treatment period.
About Hypertrophic Cardiomyopathy
Hypertrophic cardiomyopathy, or HCM, is a chronic, progressive disease in which excessive contraction of the heart muscle and reduced ability of the left ventricle to fill can lead to the development of debilitating symptoms and cardiac dysfunction. HCM is estimated to affect one in every 500 people globally.
The most frequent cause of HCM is mutations in the heart muscle proteins of the sarcomere. In both obstructive or non-obstructive HCM patients, exertion can result in fatigue or shortness of breath, interfering with a patient’s ability to participate in activities of daily living. HCM has also been associated with increased risks of atrial fibrillation, stroke, heart failure and sudden cardiac death.
About Mavacamten
Mavacamten is a potential first-in-class, oral, allosteric modulator of cardiac myosin, under investigation for the treatment of conditions in which excessive cardiac contractility and impaired diastolic filling of the heart are the underlying cause. Mavacamten reduces cardiac muscle contractility by inhibiting excessive myosin-actin cross-bridge formation that results in hypercontractility, left ventricular hypertrophy and reduced compliance. In clinical and preclinical studies, mavacamten has consistently reduced biomarkers of cardiac wall stress, lessened excessive cardiac contractility and increased diastolic compliance.
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