China Approves Myqorzo and Redemplo, Expanding Options in Hypertrophic Cardiomyopathy and Familial Chylomicronaemia Syndrome | iPharmaCenter

China’s National Medical Products Administration has authorised two innovative therapies licensed by Sanofi, strengthening treatment options in serious cardiovascular and lipid disorders.

Myqorzo (aficamten) has been approved for adults with obstructive hypertrophic cardiomyopathy, while Redemplo (plozasiran) has been cleared to reduce triglyceride levels in adults with familial chylomicronaemia syndrome who require dietary management.

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Myqorzo is an orally administered, selective small‑molecule inhibitor of cardiac myosin developed to improve exercise tolerance and alleviate symptoms in obstructive hypertrophic cardiomyopathy, a condition in which the heart muscle becomes abnormally thick and impairs blood flow. The Chinese approval is supported by the pivotal SEQUOIA‑HCM phase 3 trial in symptomatic obstructive disease, which demonstrated clinically meaningful improvements in functional capacity and patient‑reported outcomes.

Redemplo is a small‑interfering RNA therapy that lowers production of apolipoprotein C‑III, a key regulator of triglyceride metabolism and an established target for treating severe hypertriglyceridaemia in familial chylomicronaemia syndrome. The decision in China is based on the PALISADE phase 3 study in patients with genetically confirmed or clinically established familial chylomicronaemia syndrome, which showed robust, sustained reductions in triglycerides.

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Hypertrophic cardiomyopathy is the most common inherited heart muscle disease and is defined by pronounced thickening of the myocardium that reduces the size and compliance of the left ventricle. This stiffening limits filling, diminishes cardiac output and restricts exercise capacity, with symptoms such as chest discomfort, breathlessness, dizziness and fainting during exertion.

Familial chylomicronaemia syndrome is a rare, inherited lipid disorder in which triglyceride levels frequently exceed 880 mg/dL (around 9.9 mmol/L), driven by underlying defects in triglyceride clearance. These extreme elevations are associated with recurrent episodes of potentially life‑threatening pancreatitis, ongoing abdominal pain, disturbances in glucose metabolism, fatty liver and cognitive impairment, and are only partially controlled with strict dietary fat restriction.

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By providing durable suppression of apolipoprotein C‑III, Redemplo has delivered substantial triglyceride reductions in both genetically confirmed and clinically diagnosed familial chylomicronaemia syndrome cohorts, supporting its use as a foundational therapy in this ultra‑rare disease. The medicine has also received expedited‑pathway and orphan‑disease designations in several major regions and is already available for familial chylomicronaemia syndrome in markets including the United States and Canada, with regulatory evaluation ongoing in Europe.

Sanofi recently strengthened its position in this space through a deal to acquire development and commercial rights for Redemplo in Greater China from Visirna Therapeutics, a subsidiary of Arrowhead Pharmaceuticals.