The National Medical Products Administration in China has approved two Sanofi-licensed innovative medicines, Myqorzo (aficamten) for the treatment of obstructive hypertrophic cardiomyopathy (oHCM), and Redemplo (plozasiran) for the reduction of triglyceride levels, in adult patients with familial chylomicronaemia syndrome (FCS) on the basis of dietary control. Myqorzo. is a selective, small-molecule cardiac myosin inhibitor to improve functional capacity and relieve symptoms in patients with oHCM, in which the myocardium, the heart muscle, becomes abnormally thick.
It is the most common monogenic inherited cardiovascular disorder. The approval was based on the positive pivotal SEQUOIA-HCM phase 3 study (clinical study identifier: NCT05186818) in patients with symptomatic oHCM. Redemplo.
is a small-interfering RNA (siRNA) medicine, suppressing the production of apoc-III, an important target for reducing triglycerides in patients with FCS. FCS is a severe and rare disease where extremely high triglyceride levels can lead to various serious signs and symptoms including acute and potentially fatal pancreatitis, chronic abdominal pain, diabetes, hepatic steatosis, and cognitive issues. The approval was based on The positive pivotal PALISADE phase 3 study (clinical study identifiers: NCT05089084) in patients with genetically confirmed or clinically diagnosed FCS.
About HCM. HCM is the most common inherited cardiovascular disorder, characterized by abnormal thickening of the heart muscle (myocardium). This leads to the left ventricle becoming smaller and stiffer, impairing its ability to relax and fill with blood, limiting the heart's pumping function and exercise capacity.
HCM symptoms include: chest pain, dizziness, shortness of breath, or fainting during physical activity. HCM has two forms: oHCM (two-thirds of patients), where thickened muscle blocks blood flow, and non-obstructive HCM (one-third), where the muscle is thickened but blood flows normally. HCM patients face serious complications including atrial fibrillation, stroke, and mitral valve disease.
It's a leading cause of sudden cardiac death in young people and athletes due to dangerous heart rhythm abnormalities. Sanofi forward-looking statements.
