On January 26, Visirna（Visirna）Announces Its Small Nucleic Acid Class 1 New DrugPlethran Sodium InjectionMarketing Authorization Application(Application No.: JXHS2500018)ObtainAccepted by China NMPA,For the treatment of familial chylomicronemia syndrome（FCS）。VisirnaVisirna's press release pointed out,There are currently no drugs approved for FCS in China. Previously, volanesorsen sodium has been included by the NMPA as a breakthrough therapy drug and granted priority review.Expected to become the first effective drug for FCS approved in China。
Screenshot source:Visirna Official WeChatFCS is a serious and extremely rare genetic disorder, usually caused by multiple single-gene mutations.(e.g., LPL, GPIHBP1, APOC2, APOA5, or LMF1)Caused by loss-of-function mutations.FCS typically leads to extremely elevated fasting TG levels.(Above 880 mg/dL),Severe elevation of TG can lead to various clinical diseases and serious complications, including atherosclerosis, acute pancreatitis, type 2 diabetes, and hepatic steatosis, among others.Pulersilan Sodium（VSA001 ）It is a liver-targeted small interfering RNA (siRNA) drug., through the efficient and long-lasting silencing of apolipoprotein C-III（APOC3）The mRNA level was used to reduce the expression of the APOC3 protein. Further through lipoprotein lipase（LPL）Through dependent and independent dual pathways, effectively reduce serum TG and TG-rich lipoproteins and their degradation residues levels.On January 17, 2025, Visirna partner Arrowhead Pharmaceuticals submitted to the U.S. FDANDA for Plasmin Sodium Treatment of FCS, PDUFA Action Date is November 18, 2025.The marketing application of Plerixafor Sodium in China is mainly based on includingKey Points in Adult Patients with FCSPhase III Trial(PALISADE Study)Positive results from multiple overseas studies, including those in China, and Phase I trials in China.Data from the PALISADE study were presented at the Cardiovascular and Metabolic Pipeline R&D Webinar on June 25, 2024.PALISADE Study（NCT05089084）It is a randomized, double-blind, placebo-controlled Phase III clinical trial. A total of 75 subjects were enrolled in the study.(Distributed across different research centers in 18 countries), randomly assigned to receiveOnce every three monthsVSA001 25 mg(26 cases)、50 mg(24 cases)or placebo(25 cases)Subcutaneous injection treatment for 12 months. Subjects who complete the randomization period are eligible to continue participating in the extension period study.The primary endpoint of the PALISADE study was the median triglyceride level at the 10th month, adjusted for placebo.（TG）Horizontal changes. The results show,Patients treated with 25 mg or 50 mg VSA001 achieved median triglyceride level reductions of 80% and 78%, respectively.。
In addition to achieving the primary endpoint, VSA001 also met all critical secondary endpoints, including those at Month 10 and Month 12.(Average)Percentage change in fasting triglycerides from baseline; Percentage change in fasting APOC3 from baseline at Month 10; Percentage change in fasting APOC3 from baseline at Month 12; Incidence of acute pancreatitis events during the randomized controlled trial period.
Research data also showed that VSA001, administered once every three months, throughout the study period.Continuously Reduce Triglyceride Levels(Median and Mean), and with low variability.
In addition, VSA001 also demonstrated good safety in the PALISADE study.
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