Ionis Pharmaceuticals announced the U.S. Food and Drug Administration (FDA) approval of TRYNGOLZA™ (olezarsen) for the treatment of adults with familial chylomicronemia syndrome (FCS).TRYNGOLZA was granted multiple designations, including Priority Review, Fast Track, Orphan Drug, and Breakthrough Therapy, reflecting its significance in addressing a critical unmet medical need.

Ionis Pharmaceuticals宣布美国食品和药物管理局（FDA）批准TRYNGOLZA™（olezarsen）用于治疗成人家族性乳糜微粒血症综合征（FCS）。。

Regulatory filings for olezarsen are also ongoing for broader indications of sHTG.This rare genetic condition, characterised by severe hypertriglyceridaemia (sHTG), can lead to life-threatening acute pancreatitis (AP). TRYNGOLZA is now the first FDA-approved treatment specifically designed for FCS, offering significant reductions in triglyceride levels and a decreased risk of acute pancreatitis when combined with a strict low-fat diet (≤20 grams of fat per day).

olezarsen的监管文件也正在进行中，以获得更广泛的sHTG指征。这种罕见的遗传病以严重的高甘油三酯血症（sHTG）为特征，可导致危及生命的急性胰腺炎（AP）。TRYNGOLZA现在是FDA批准的第一种专门为FCS设计的治疗方法，与严格的低脂饮食（每天≤20克脂肪）相结合，可以显着降低甘油三酯水平，降低急性胰腺炎的风险。

The treatment is self-administered via a monthly auto-injector.The FDA’s decision follows results from the global, randomised, placebo-controlled Phase 3 Balance clinical trial, which assessed the efficacy and safety of TRYNGOLZA in adults with genetically confirmed FCS and triglyceride levels ≥880 mg/dL. Participants receiving TRYNGOLZA demonstrated a 42.5% mean reduction in triglyceride levels compared to placebo at six months, improving to a 57% reduction at 12 months.

治疗是通过每月自动注射器自我管理的。FDA的决定是根据全球随机安慰剂对照3期Balance临床试验的结果做出的，该试验评估了TRYNGOLZA对经基因证实的FCS和甘油三酯水平≥880 mg/dL的成年人的疗效和安全性。与安慰剂相比，接受TRYNGOLZA治疗的参与者在六个月时甘油三酯水平平均降低了42.5%，在12个月时降低了57%。

Additionally, there was a significant decrease in acute pancreatitis episodes, with fewer patients experiencing these events compared to the placebo group.TRYNGOLZA exhibited a favourable safety profile. Common side effects included injection site reactions, decreased platelet count, and joint pain.

此外，急性胰腺炎发作显着减少，与安慰剂组相比，经历这些事件的患者较少。TRYNGOLZA表现出良好的安全性。常见的副作用包括注射部位反应，血小板计数减少和关节疼痛。

The findings from the Balance trial have been published in The New England Journal of Medicine (NEJM).FCS, a rare and serious genetic condition, disrupts the body’s ability to.

平衡试验的结果发表在《新英格兰医学杂志》（NEJM）上。FCS是一种罕见而严重的遗传病，会破坏身体的能力。