China Approves Sanofi-Licensed Medicines Myqorzo and Redemplo, Expanding Treatment Options for Rare and Complex Diseases

China’s National Medical Products Administration (NMPA) has granted regulatory approval to two innovative medicines licensed by Sanofi—Myqorzo (aficamten) and Redemplo (plozasiran). The approvals mark an important milestone in expanding access to advanced therapies for patients in China living with serious and often underdiagnosed conditions, including obstructive hypertrophic cardiomyopathy (oHCM) and familial chylomicronaemia syndrome (FCS).

Myqorzo has been approved for the treatment of adults with obstructive hypertrophic cardiomyopathy, a genetic cardiovascular disorder characterized by abnormal thickening of the heart muscle. Redemplo has received approval for use in adult patients with familial chylomicronaemia syndrome to reduce dangerously high triglyceride levels, in conjunction with dietary control. Both medicines address significant unmet medical needs and represent important scientific advances in their respective therapeutic areas.

Commenting on the approvals, Olivier Charmeil, Executive Vice President of General Medicines at Sanofi, emphasized the company’s long-term commitment to innovation in China. “We are pleased to bring Myqorzo and Redemplo to patients in Greater China. Both medicines represent important advances in treatment options and address unmet medical needs among people living with complex conditions,” he said. “These approvals underscore Sanofi’s sustained commitment to introducing innovative therapies that can meaningfully improve patient outcomes in China.”

Myqorzo: A Novel Therapy for Obstructive Hypertrophic Cardiomyopathy

Myqorzo (aficamten) is a selective, small-molecule cardiac myosin inhibitor designed to improve functional capacity and relieve symptoms in patients with obstructive hypertrophic cardiomyopathy. HCM is the most common monogenic inherited cardiovascular disorder and is characterized by abnormal thickening of the myocardium, or heart muscle. In the obstructive form of the disease, thickened heart muscle interferes with normal blood flow from the heart, leading to symptoms such as chest pain, shortness of breath, dizziness, and fainting, particularly during physical activity.

The NMPA approval of Myqorzo was supported by positive results from the pivotal Phase 3 SEQUOIA-HCM clinical trial. The study demonstrated that aficamten significantly improved clinical outcomes in patients with symptomatic oHCM by reducing left ventricular outflow tract obstruction and improving exercise capacity and quality of life.

Myqorzo was discovered following an extensive chemical optimization program aimed at achieving an optimal therapeutic index and favorable pharmacokinetic properties. The medicine works by reducing the number of active actin-myosin cross-bridges during each cardiac cycle, thereby suppressing the excessive myocardial contractility associated with HCM. Preclinical studies showed that Myqorzo binds directly to cardiac myosin at a selective allosteric site, preventing myosin from entering a force-producing state.

Globally, Myqorzo has received multiple regulatory recognitions. It has been designated as a breakthrough therapy and orphan drug in the United States, as well as a breakthrough therapy in China. The medicine is already approved in the United States and China, and in December 2025, the European Medicines Agency’s Committee for Medicinal Products for Human Use issued a positive opinion recommending marketing authorization in the European Union, with a final decision anticipated in early 2026.

Sanofi acquired exclusive rights in December 2024 to develop and commercialize Myqorzo in Greater China for both obstructive and non-obstructive forms of HCM through an agreement with Corxel Pharmaceuticals, which had previously obtained the rights from Cytokinetics.

Understanding Hypertrophic Cardiomyopathy

Hypertrophic cardiomyopathy affects patients across all age groups and can lead to serious complications if left untreated. The disease has two primary forms: obstructive HCM, which affects approximately two-thirds of patients, and non-obstructive HCM, which accounts for the remaining one-third. Patients with HCM face elevated risks of atrial fibrillation, stroke, mitral valve disease, heart failure, and sudden cardiac death, particularly among young individuals and athletes.

Redemplo: A Breakthrough Treatment for Familial Chylomicronaemia Syndrome

Redemplo (plozasiran) is an innovative small-interfering RNA (siRNA) therapy developed to address the underlying cause of familial chylomicronaemia syndrome, a rare and severe genetic disorder. FCS is characterized by extremely elevated triglyceride levels, often exceeding 880 mg/dL (9.94 mmol/L), which significantly increase the risk of acute pancreatitis and other serious complications.

Redemplo works by suppressing the production of apolipoprotein C-III (apoC-III), a liver-produced protein that plays a key role in regulating triglyceride metabolism. By silencing apoC-III, Redemplo enables sustained and clinically meaningful reductions in triglyceride levels, helping to reduce the risk of life-threatening complications.

The approval of Redemplo by the NMPA was based on positive results from the pivotal Phase 3 PALISADE study, which evaluated the therapy in patients with genetically confirmed or clinically diagnosed FCS. The trial demonstrated substantial triglyceride reductions and a favorable safety profile.

Redemplo has received multiple regulatory designations, including breakthrough therapy, fast track, and orphan drug status in the United States; orphan designation in the European Union; and breakthrough therapy designation in China. The medicine is already approved for the treatment of FCS in the United States, Canada, and China, with regulatory review ongoing in the European Union.

Advancing Innovation for Patients in China

The approvals of Myqorzo and Redemplo reflect Sanofi’s broader strategy to expand access to cutting-edge therapies for patients with rare and complex diseases in China. By bringing forward medicines that target disease mechanisms at a molecular level, Sanofi aims to improve clinical outcomes and quality of life for patients who previously had limited treatment options.

As China continues to strengthen its regulatory environment for innovative medicines, these approvals highlight the growing role of advanced therapies in addressing unmet medical needs and improving standards of care across multiple disease areas.

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