Givinostat: First Approval.

IF 13 1区 医学 Q1 PHARMACOLOGY & PHARMACY Drugs Pub Date : 2024-07-01 Epub Date: 2024-07-05 DOI:10.1007/s40265-024-02052-1
Yvette N Lamb
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Abstract

Givinostat (DUVYZAT™), an orally available histone deacetylase inhibitor, is being developed by Italfarmaco for the treatment of muscular dystrophy and polycythemia vera. Givinostat received its first approval on 21 March 2024, in the USA, for the treatment of Duchenne muscular dystrophy (DMD) in patients 6 years of age and older. Approval was based on the results of the multinational phase III EPIDYS trial, in which givinostat recipients showed less decline than placebo recipients in the time taken to perform a functional task. Givinostat represents the first nonsteroidal treatment for DMD to be approved for use in patients irrespective of the specific genetic variant underlying their disease. Givinostat is available as an oral suspension to be administered twice daily with food. The recommended dosage is based on the body weight of the patient. In the EU, regulatory review of givinostat in DMD is currently underway. This article summarizes the milestones in the development of givinostat leading to this first approval for DMD.

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吉维诺司他首次批准。
Givinostat(DUVYZAT™)是一种口服组蛋白去乙酰化酶抑制剂,由 Italfarmaco 公司开发,用于治疗肌肉萎缩症和多血症。吉维诺司他(Givinostat)于 2024 年 3 月 21 日在美国首次获得批准,用于治疗 6 岁及以上杜氏肌营养不良症(DMD)患者。获批的依据是跨国 EPIDYS III 期试验的结果,在该试验中,吉维诺他受试者在完成一项功能性任务所需的时间上比安慰剂受试者下降得更少。吉维司他是首款获准用于DMD治疗的非甾体类药物,不论患者的具体基因变异情况如何。吉维司他是一种口服混悬液,每天两次,与食物一起服用。建议剂量根据患者体重确定。在欧盟,吉维诺他治疗 DMD 的监管审查目前正在进行中。本文总结了吉维司他开发过程中的里程碑事件,这些事件促成了吉维司他在 DMD 领域的首次获批。
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来源期刊
Drugs
Drugs 医学-毒理学
CiteScore
22.70
自引率
0.90%
发文量
134
审稿时长
3-8 weeks
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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