sepapterin：第一次批准。

IF 14.4 1区医学 Q1 PHARMACOLOGY & PHARMACY

Drugs Pub Date : 2025-10-15 DOI:10.1007/s40265-025-02247-0

Yvette N Lamb

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引用次数: 0

摘要

PTC Therapeutics正在开发一种用于治疗苯丙酮尿症（PKU）的口服制剂sepiapterin （Sephience™）。sepapterin是四氢生物terin （BH4）的天然前体，是苯丙氨酸羟化酶（PAH）介导的苯丙氨酸代谢所必需的。sepapterin可增强PAH活性，而PKU患者缺乏PAH活性。Sepiapterin于2025年6月19日在欧盟获得首次批准，用于成人和儿科PKU患者的高苯丙氨酸血症（HPA）。随后，该药物于2025年7月28日在美国获得批准，用于治疗成人和≥1个月大的sepiapterin反应性PKU患者的HPA。本文总结了sepapterin在成人和儿童PKU患者中首次被批准用于HPA的发展里程碑。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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Sepiapterin: First Approval.

An oral formulation of sepiapterin (Sephience™) is being developed by PTC Therapeutics for the treatment of phenylketonuria (PKU). Sepiapterin is a natural precursor of enzyme cofactor tetrahydrobiopterin (BH₄), which is required for phenylalanine hydroxylase (PAH)-mediated phenylalanine metabolism. Sepiapterin enhances PAH activity, which is deficient in patients with PKU. Sepiapterin received its first approval on 19 June 2025 in the European Union, for use in hyperphenylalaninaemia (HPA) in adult and paediatric patients with PKU. This was followed by approval in the USA on 28 July 2025, for the treatment of HPA in adult and paediatric patients ≥ 1 month of age with sepiapterin-responsive PKU. This article summarizes the milestones in the development of sepiapterin leading to its first approval for HPA in adult and paediatric patients with PKU.

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来源期刊

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.