Visirna Announces NMPA Acceptance of New Drug Application for Plozasiran

(Suzhou, China, Jan. 26th, 2025) – Visirna Therapeutics (Suzhou) Co., Ltd. (hereinafter referred to as "Visirna") today announced that its class 1 investigational RNA interference (RNAi) therapeutic , Plozasiran, has had its new drug application (NDA) (Acceptance No. JXHS2500018) officially accepted by the National Medical Products Administration (NMPA) for the treatment of familial chylomicronemia syndrome (FCS). Currently, no drug has been approved for the treatment of FCS in China. Plozasiran has previously been granted as Breakthrough Therapy Designation (BTD) and Priority Review to accelerate its marketing approval in China. The NDA submission for Plozasiran in China is primarily based on the positive results from multiple overseas studies, including the pivotal Phase III PALISADE study for FCS, as well as a Phase I study conducted in China. In the PALISADE study, Plozasiran met its primary endpoint and all key secondary endpoints, including statistically significant reductions in triglycerides (TGs), apolipoprotein C-III (APOC3), and the incidence of acute pancreatitis (AP). Dr. Xiaoming Zou, CEO of Visirna, stated, "FCS typically leads to extremely elevated TG levels in patients. Currently, no drugs for the treatment of FCS have been approved in China. The inability to effectively control TG levels significantly increases the treatment burden associated with acute pancreatitis, cardiovascular disease, and diabetes, which severely compromise the patient's health status and potentially pose life-threatening risks. Plozasiran, an innovative oligonucleotide drug targeting APOC3, has demonstrated robust and sustained TG-lowering effects across multiple clinical studies. The drug exhibits favorable safety profile and tolerability, with the convenience of quarterly dosing. It shows promise in addressing the current therapeutic void for FCS treatment in China. This drug represents Visirna's first product to enter the marketing approval review phase since the company's establishment. This milestone signals Visirna's imminent entry into the commercialization stage of oligonucleotide therapeutics in China, positioning the company as a biopharmaceutical enterprise with capabilities spanning the full industry chain of oligonucleotide drug R&D, manufacturing, and commercialization. We are simultaneously exploring plozasiran's therapeutic potential in additional indications, including severe hypertriglyceridemia, with the aim of extending the novel and unique therapeutic benefits of this drug to a broader patient population in China". 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. 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.

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