Sanofi Secures Chinese Approval for Two Breakthrough Therapies Targeting Rare Cardiovascular and Metabolic Disorders
核心洞察
China's National Medical Products Administration has approved Sanofi's Myqorzo (搜索) (aficamten) for obstructive hypertrophic cardiomyopathy (搜索) and Redemplo (搜索) (plozasiran) for familial chylomicronemia syndrome (搜索).
Myqorzo (搜索) represents a novel cardiac myosin (搜索) inhibitor that addresses the most common inherited cardiovascular disorder, while Redemplo (搜索) offers a siRNA-based approach to managing extremely high triglyceride levels.
Both therapies received breakthrough therapy designations and target severe conditions with significant unmet medical needs, potentially transforming treatment options for Chinese patients.
The National Medical Products Administration in China has approved two innovative Sanofi-licensed medicines that address significant unmet medical needs in rare cardiovascular and metabolic disorders. Myqorzo (搜索) (aficamten) received approval for treating obstructive hypertrophic cardiomyopathy (搜索) (oHCM), while Redemplo (搜索) (plozasiran) was approved for reducing triglyceride levels in adult patients with familial chylomicronemia syndrome (搜索) (FCS).
"We are pleased to bring Myqorzo (搜索) and Redemplo (搜索) to patients in Greater China. Both medicines represent important advances in treatment options and address unmet medical needs among people living with complex conditions," said Olivier Charmeil, Executive Vice President, General Medicines, Sanofi.
Myqorzo Targets Most Common Inherited Heart Disorder
Myqorzo (搜索) is a selective, small-molecule cardiac myosin (搜索) inhibitor designed to improve functional capacity and relieve symptoms in patients with oHCM. The condition affects the myocardium, where abnormal thickening of the heart muscle leads to reduced pumping function and exercise capacity. HCM represents the most common monogenic inherited cardiovascular disorder.
The drug works by reducing the number of active actin-myosin cross bridges during each cardiac cycle, thereby suppressing the myocardial hypercontractility associated with HCM. In preclinical models, Myqorzo (搜索) demonstrated its mechanism by binding directly to cardiac myosin (搜索) at a distinct and selective allosteric binding site, preventing myosin from entering a force-producing state.
HCM manifests in two forms: oHCM affects two-thirds of patients, where thickened muscle blocks blood flow, and non-obstructive HCM affects one-third, where muscle thickening occurs without blocking blood flow. Patients face serious complications including atrial fibrillation (搜索), stroke (搜索), and mitral valve disease. The condition serves as a leading cause of sudden cardiac death in young people and athletes due to dangerous heart rhythm abnormalities.
The Chinese approval was based on the positive pivotal SEQUOIA-HCM phase 3 study (NCT05186818) in patients with symptomatic oHCM. Myqorzo (搜索) previously received breakthrough therapy and orphan drug designations in the US, and breakthrough therapy designation in China. The drug is currently approved in the US and China, with European approval expected in the first quarter of 2026 following a positive opinion from the European Medicines Agency's Committee for Medicinal Products for Human Use.
Redemplo Addresses Severe Lipid Disorder
Redemplo (搜索) represents a small-interfering RNA (siRNA) medicine that suppresses the production of apoC-III (搜索), an important target for reducing triglycerides in patients with FCS. The protein, produced primarily in the liver, raises triglyceride levels by slowing their breakdown and clearance. Through sustained silencing of apoC-III, Redemplo delivers significant reductions in triglyceride levels.
FCS is a severe and rare disease characterized by extremely high triglyceride levels exceeding 880 mg/dL (9.94 mmol/L). These severe elevations can lead to acute and potentially fatal pancreatitis (搜索), chronic abdominal pain, diabetes (搜索), hepatic steatosis, and cognitive issues.
The approval was based on the positive pivotal PALISADE phase 3 study (NCT05089084) in patients with genetically confirmed or clinically diagnosed FCS. Redemplo (搜索) has received breakthrough therapy, fast track, and orphan drug designations in the US, orphan designation in the EU, and breakthrough therapy designation in China. The drug is currently approved in the US, Canada, and China, with regulatory review ongoing in the EU.
Strategic Market Expansion
Sanofi acquired the rights to develop and commercialize these therapies in Greater China through strategic partnerships. In December 2024, the company obtained exclusive rights to Myqorzo (搜索) through an agreement with Corxel Pharmaceuticals (搜索), who had acquired them from Cytokinetics (搜索). In August 2025, Sanofi acquired rights to Redemplo (搜索) from Visirna Therapeutics (搜索), a majority-owned subsidiary of Arrowhead Pharmaceuticals.
The approvals underscore Sanofi's commitment to providing transformative medicines to Chinese patients in disease areas with large unmet medical needs, reinforcing the company's long-term strategy in the region.