Fenfluramine Hydrochloride

▎药明康德优时比小分子达3期试验主要终点，上市申请递交在即优时比（UCB）今日宣布，其小分子疗法fenfluramine在治疗CDKL5缺乏症（CDD）的3期临床试验中取得积极结果。公司计划尽快向监管机构提交该药物的上市申请，为CDD患者群体带来这一潜在的全新治疗选择。CDKL5缺乏症是一种超罕见且严重的发育性和癫痫性脑病（DEE），其主要特征包括婴儿期起频繁发作的癫痫，以及严重的全球性神经发育迟缓，进而影响患者的认知功能、运动能力、皮层视觉功能以及睡眠质量。此次公布的研究为一项随机、双盲、安慰剂对照、固定剂量的多中心研究，共纳入87名年龄在1至35岁之间、确诊为CDD且癫痫未受控的患者，评估fenfluramine作为辅助治疗的疗效与安全性。本研究的主要终点是比较fenfluramine组与安慰剂组在药物滴定期及维持期内，与基线相比的可计数运动性癫痫发作频率（CMSF）中位百分比变化。研究结果显示，该试验已达到主要终点及大多数关键次要终点，且fenfluramine具有良好的总体耐受性，其安全性与以往研究中观察到的结果一致。反义寡核苷酸药物获突破性疗法认定，3期试验进行中Ultragenyx Pharmaceutical今日宣布，其在研反义寡核苷酸（ASO）疗法GTX-102（apazunersen）获得美国FDA授予的突破性疗法认定，用于治疗天使综合征（AS）。该认定主要基于一项1/2期临床试验的积极结果。该研究共纳入74名年龄在4至17岁之间、携带母源UBE3A基因完全缺失的天使综合征患者，结果显示，患者在接受治疗最长达3年后，在多个症状领域持续表现出快速且稳定的发育改善。GTX-102通过鞘内给药方式递送，该疗法可以促进神经元细胞中父系UBE3A的等位基因表达，产生患者体内所缺失的关键蛋白产物。该疗法的相关全球3期试验正在进行中。参考资料：[1] UCB announces positive results from GEMZ phase 3 study of fenfluramine in CDKL5 Deficiency Disorder. Retrieved June 27, 2025 from https://www.ucb.com/newsroom/press-releases/article/ucb-announces-positive-results-from-gemz-phase-3-study-of-fenfluramine-in-cdkl5-deficiency-disorder[2] Ultragenyx Receives Breakthrough Therapy Designation for GTX-102 in Angelman Syndrome. Retrieved June 27, 2025 from https://www.globenewswire.com/news-release/2025/06/27/3106516/20739/en/Ultragenyx-Receives-Breakthrough-Therapy-Designation-for-GTX-102-in-Angelman-Syndrome.html免责声明：本文仅作信息交流之目的，文中观点不代表药明康德立场，亦不代表药明康德支持或反对文中观点。本文也不是治疗方案推荐。如需获得治疗方案指导，请前往正规医院就诊。版权说明：欢迎个人转发至朋友圈，谢绝媒体或机构未经授权以任何形式转载至其他平台。转载授权请在「药明康德」微信公众号回复“转载”，获取转载须知。👇 分享，点赞，在看，聚焦全球生物医药健康创新

After scoring approvals in two epilepsy disorders, UCB's Fintepla has excelled in a phase 3 trial in patients with a different epilepsy condition. UCB’s Fintepla has been approved to treat seizures associated with two epilepsy disorders and now has posted data that could pave the way for its use in another epilepsy condition.A phase 3 trial of Fintepla in patients with the ultra-rare CDKL5 deficiency disorder (CDD) has achieved its primary and most of its secondary endpoints, the Brussels-based company said in a June 27 press release.The study enrolled 87 participants between the ages of 1 and 35 who have been diagnosed with CDD and uncontrolled seizures. In comparing the performance of Fintepla to placebo, the primary endpoint was based on the median percent change in countable motor seizure frequency between baseline and the titration-plus-maintenance trial phase. UCB said it will present the results at an upcoming medical conference.In a statement, UCB's Fiona du Monceau, EVP of patient evidence, said the company looks forward to working with regulators to make the drug available "as soon as possible."Fintepla was well-tolerated in the trial, with its safety profile consistent with previous studies. UCB is conducting a 54-week extension phase of the trial to investigate the long-term tolerability of the oral treatment.The median age for CDD onset is 6 weeks, UCB said. The genetic condition causes neurodevelopmental delays resulting in intellectual, motor, cortical visual, and sleep impairments. It is estimated that CDD affects approximately 1 in 40,000 to 60,000 live births.Fintepla was first approved in 2020 to treat seizures associated with Dravet syndrome, a rare, severe form of epilepsy. Two years later, Fintepla gained expansion into a larger epilepsy indication, Lennox-Gastaut syndrome, which affects between 1 to 2 million people worldwide.  Months before the Lennox-Gastaut nod, UCB paid $1.9 billion to acquire Fintepla and its developer, California-based biotech Zogenix. The deal was a strategic fit for UCB, which then had four epilepsy drugs on the market but was losing market exclusivity for the most successful one, Vimpat, a blockbuster that outsold the other three combined.Fintepla appears to be on a blockbuster track of its own, with sales reaching 340 million euros ($368 million) last year, which was a bump from sales of 226 million euros ($243 million) in 2023. After a 2023 settlement in a patent dispute, UCB expects to retain U.S. exclusivity on Fintepla until 2033.