林业发展局批准第一种用于稀有遗传病的SIRNA药物,以减少三甘酸盐和胰腺炎风险。 FDA approves first siRNA drug for rare genetic disorder, reducing triglycerides and pancreatitis risk.

食品和药物管理局批准了plozasiran,作为REDEPROPOL出售,这是首次对家庭血清微生物综合症(一种罕见的遗传障碍)进行SIRNA疗法。 The FDA has approved plozasiran, sold as REDEMPLO, the first siRNA therapy for familial chylomicronemia syndrome (FCS), a rare genetic disorder. 该药物由箭头制药公司研制,以ApoC-III为对象,减少三甘酸盐,每三个月注射一次次皮注射。 The drug, developed by Arrowhead Pharmaceuticals, reduces triglycerides by targeting apoC-III and is administered as a subcutaneous injection every three months. 根据PALISADE第3阶段试验结果核准的试验结果表明,三环丙酸中位数减少了80%,与安慰剂相比,胰腺炎风险降低83%,具有有利的安全特征。 Approved based on phase 3 PALISADE trial results, it showed an 80% median triglyceride reduction and 83% lower pancreatitis risk versus placebo, with a favorable safety profile. 预计该药物将在年终之前在美国提供。 The drug is expected to be available in the U.S. before year-end.