{"title":"Olezarsen: First Approval.","authors":"Yahiya Y Syed","doi":"10.1007/s40265-025-02166-0","DOIUrl":null,"url":null,"abstract":"<p><p>Olezarsen (TRYNGOLZA™) is a first-in-class, GalNAc<sub>3</sub>-conjugated antisense oligonucleotide being developed by Ionis Pharmaceuticals for the treatment of familial chylomicronaemia syndrome (FCS) and severe hypertriglyceridaemia. It binds to apoC-III mRNA, promoting its degradation and lowering serum apoC-III protein levels. This action lowers triglyceride levels by enhancing the clearance of plasma triglyceride-rich lipoproteins such as chylomicrons and very-low-density lipoproteins. GalNAc<sub>3</sub> conjugation improves hepatocyte uptake, which helps localise oligonucleotide treatment to the liver; this enables lower dosing, reduced injection volume and frequency, potentially reducing the risk of adverse events such as thrombocytopenia. Olezarsen received its first approval in the USA on 19 December 2024 under priority review as an adjunct to diet to reduce triglycerides in adults with FCS. This article summarizes the milestones in the development of olezarsen leading to this first approval for FCS.</p>","PeriodicalId":11482,"journal":{"name":"Drugs","volume":" ","pages":"571-576"},"PeriodicalIF":13.0000,"publicationDate":"2025-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":"0","resultStr":null,"platform":"Semanticscholar","paperid":null,"PeriodicalName":"Drugs","FirstCategoryId":"3","ListUrlMain":"https://doi.org/10.1007/s40265-025-02166-0","RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":null,"EPubDate":"2025/3/13 0:00:00","PubModel":"Epub","JCR":"Q1","JCRName":"PHARMACOLOGY & PHARMACY","Score":null,"Total":0}
Olezarsen (TRYNGOLZA™) is a first-in-class, GalNAc3-conjugated antisense oligonucleotide being developed by Ionis Pharmaceuticals for the treatment of familial chylomicronaemia syndrome (FCS) and severe hypertriglyceridaemia. It binds to apoC-III mRNA, promoting its degradation and lowering serum apoC-III protein levels. This action lowers triglyceride levels by enhancing the clearance of plasma triglyceride-rich lipoproteins such as chylomicrons and very-low-density lipoproteins. GalNAc3 conjugation improves hepatocyte uptake, which helps localise oligonucleotide treatment to the liver; this enables lower dosing, reduced injection volume and frequency, potentially reducing the risk of adverse events such as thrombocytopenia. Olezarsen received its first approval in the USA on 19 December 2024 under priority review as an adjunct to diet to reduce triglycerides in adults with FCS. This article summarizes the milestones in the development of olezarsen leading to this first approval for FCS.
期刊介绍：
Drugs is a journal that aims to enhance pharmacotherapy by publishing review and original research articles on key aspects of clinical pharmacology and therapeutics. The journal includes:
Leading/current opinion articles providing an overview of contentious or emerging issues.
Definitive reviews of drugs and drug classes, and their place in disease management.
Therapy in Practice articles including recommendations for specific clinical situations.
High-quality, well designed, original clinical research.
Adis Drug Evaluations reviewing the properties and place in therapy of both newer and established drugs.
AdisInsight Reports summarising development at first global approval.
Moreover, the journal offers additional digital features such as animated abstracts, video abstracts, instructional videos, and podcasts to increase visibility and educational value. Plain language summaries accompany articles to assist readers with some knowledge of the field in understanding important medical advances.
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