｜Edited by the Content Team of Zhong肽 BiochemicalJanuary 202527Day，Center for Drug Evaluation (CDE) of the National Medical Products AdministrationThe website shows that Visirna Therapeutics' small interfering RNA (siRNA) therapy, Plozasiran Sodium (Plozasiran, VSA001) Injection, has been submitted for marketing authorization in China to treat familial chylomicronemia syndrome (FCS). The drug has recently been submitted for marketing authorization in the United States and is expected to become the first siRNA therapy approved for FCS.
Plesiran is a liver-targeted small interfering RNA (siRNA) drug developed by Arrowhead Pharmaceuticals. It effectively and persistently silences the mRNA levels of apolipoprotein C-III (APOC3), reducing the expression of the APOC3 protein. Through both lipoprotein lipase (LPL)-dependent and -independent pathways, it efficiently lowers serum triglycerides (TG), TG-rich lipoproteins (TRL), and their degradation remnants. In April 2022, Arrowhead Pharmaceuticals established Visirna through a joint venture with Vivo Capital and granted the latter the rights to develop and commercialize four RNAi therapies in China, including Plesiran.The marketing application of Volanesorsen is mainly based on the positive results of several overseas studies, including the pivotal Phase III trial (PALISADE study), and a Phase I trial in China. The PALISADE study enrolled 75 patients with FCS, with or without genetic diagnosis, to evaluate the efficacy of Volanesorsen (25mg or 50mg, subcutaneous injection, once every 3 months) compared to placebo.The results showed that after 10 months of treatment, the fasting TG levels of patients in the Prusilan group were significantly reduced (P<0.001), with decreases of 80%, 78%, and 17% in the 25mg group, 50mg group, and placebo group, respectively.Source: New England Journal of MedicineIn addition, after 10 months of treatment, the APOC3 levels in patients in the volanesorsen group were significantly reduced (P<0.001), with decreases of 93%, 96%, and 1% in the 25mg group, 50mg group, and placebo group, respectively.In terms of safety, the incidence of adverse events was similar across the three groups (23% vs 20% vs 20%). The incidence of acute pancreatitis (AP) reported by patients in the Pluxiran group was reduced.Source: New England Journal of MedicineIn November 2024, Arrowhead Pharmaceuticals also submitted a new drug application to the FDA for volanesorsen for the treatment of FCS based on the PALISADE study. The application has been recently accepted, with a Prescription Drug User Fee Act (PDUFA) date of November 18, 2025. FCS is a severe rare genetic disorder, with an estimated prevalence of approximately 1 in 1,000,000 according to incomplete statistics. It is typically caused by loss-of-function mutations or compound/double heterozygous mutations in various single genes (e.g., LPL, GPIHBP1, APOC2, APOA5, or LMF1). FCS often leads to extremely elevated fasting TG levels (above 880 mg/dL). Severe TG elevation can result in various clinical conditions and serious complications, including acute pancreatitis, atherosclerosis, type 2 diabetes, obesity, hepatic steatosis, and chronic kidney disease. Currently, there are no drugs approved for FCS available in China.AboutVisirna
Visirna is a small nucleic acid drug therapy company based in China with a global outlook, aiming to build a biopharmaceutical enterprise with comprehensive capabilities in research and development, production, and commercialization. The company was founded in 2022 and has established a long-term strategic partnership with Arrowhead Pharmaceuticals (NASDAQ: ARWR), an internationally leading small nucleic acid drug company. Currently, the company’s pipeline includes four small nucleic acid drugs in clinical development stages targeting cardiovascular metabolism and autoimmune diseases. The company's existing pipeline is in a leading position among similar competing products, with its research targets supported by clear genomic and biological evidence. These drugs utilize chemical modification and delivery technology platforms that have been validated by Arrowhead Pharmaceuticals and independently developed by Visirna. The company adopts a U.S.-China coordinated clinical development and registration strategy, which helps accelerate the registration and market launch of its products under development. At the same time, through the comprehensive integration of internal and external resources, it forms an entire industry chain layout from early-stage R&D, clinical R&D, localized production to commercialization.Source:1.PharmaCube
