Plozasiran (VSA001) Granted Priority Review in China for patients with Familial Chylomicronemia Syndrome (FCS)

SUZHOU, CHINA – January 7th, 2025, Visirna announced that the Center for Drug Evaluation (CDE) of National Medical Products Administration (NMPA) of China granted priority review for Plozasiran (VSA001) for the treatment of patients with Familial Chylomicronemia Syndrome (FCS). Previously, the CDE granted Breakthrough Therapy Designation (BTD) for VSA001. There are currently no domestically approved and marketed drugs available in China for FCS. Priority review constitutes a programmatic approach specifically designed to accelerate the drug approval process. The policy aims to ensure the rapid approval and market availability of drugs that address significant unmet clinical needs or offer notable clinical advantages, thereby benefiting patients in urgent need of medication. According to the "Procedures for Priority Review and Approval of Drug Marketing Authorization," applications included in the priority review program are subject to a review period of 130 working days. Center for Food and Drug Inspection of NMPA, National Institutes for Food and Drug Control, and the Chinese Pharmacopoeia Commission will prioritize the inspection, testing, and approval of generic names for these drugs. Plozasiran is a hepatocyte-targeting small interfering RNA (siRNA) designed to inhibit the expression of apolipoprotein C3 (APOC3) by efficiently and consistently silencing messenger RNA (mRNA) of APOC3. The levels of serum TG, TG-rich lipoprotein (TRL), and TRL remnants are effectively lowered via both lipoprotein lipase (LPL)-independent and LPL-dependent pathways. FCS is a severe rare genetic disease, with an estimated prevalence of approximately 1/1,000,000. It is typically caused by mutations or compound/heterozygous mutations in multiple single genes (e.g., LPL, GPIHBP1, APOC2, APOA5, or LMF1). FCS usually leads to extremely elevated fasting TG levels (above 880 mg/dL). Severe TG elevation can cause various clinical diseases and severe complications, including acute pancreatitis, atherosclerosis, type 2 diabetes, obesity, fatty liver, and chronic kidney disease. Currently, there are no approved drugs specifically for FCS. Visirna was founded in 2022 in a strategic partnership with Arrowhead Pharmaceuticals (NASDAQ: ARWR). Headquartered in China with a global perspective, Visirna strives to emerge as a frontrunner in the advancement of siRNA therapeutics. The existing product portfolio comprises three clinical stage siRNA candidates with a focus on cardiovascular and metabolic ailments. By effectively integrating internal expertise with external resources, Visirna has cultivated a robust suite of industry capabilities spanning drug discovery, clinical advancement, domestic manufacturing, and commercialization. For more information, please visit www. visirna.com

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