基因疗法药物研发商PTC Therapeutics宣布Sephience™获得欧盟委员会批准，治疗患有苯丙酮尿症（PKU）的儿童和成人Sephience™ (sepiapterin) Granted Marketing Authorization by the European Commission for the Treatment of Children and Adults Living with Phenylketonuria (PKU)

动脉网2025-06-25

PTC Therapeutics, Inc. today announced that Sephience™ (sepiapterin) was granted marketing authorization by the European Commission for the treatment of children and adults living with phenylketonuria...

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The Sephience European launch will be initiated in. Sephience 的欧洲上市将在Germany德国in the first half of July.在七月份的上半月。By enhancing the conformational stability of misfolded PAH enzyme and increasing the intracellular concentrations of BH4, Sephience is able to effectively reduce blood Phe levels. Sephience  is approved in the European Economic Area. 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The authorization includes a broad label inclusive of all ages and disease severities.PTC Therapeutics, Inc.今天宣布，Sephience™（sepiapterin）已获得欧盟委员会的上市授权，用于治疗患有苯丙酮尿症（PKU）的儿童和成人。该授权涵盖了所有年龄层和疾病严重程度的广泛标签。'The European approval of Sephience is a great step in our efforts to bring this safe and highly effective therapy to children and adults affected by PKU worldwide,' said“Sephience获得欧洲批准是我们为全球受PKU影响的儿童和成人带来这种安全且高效疗法的努力中的重要一步，”Matthew B. Klein马修·B·克莱因, M.D., Chief Executive Officer of PTC Therapeutics. 'The broad label supports that potential for Sephience to address all key PKU patient segments and become the new standard of care.'，医学博士，PTC Therapeutics首席执行官。 “广泛的应用范围支持Sephience有望解决所有关键的PKU患者群体，并成为新的护理标准。”The European approval is based on the highly statistically significant results from the欧洲的批准是基于高度统计学显著的结果从Phase 3 APHENITY trial as well as evidence of durable treatment effect and the ability of study participants to liberalize their diet in the APHENITY long-term extension study.第三阶段的APHENITY试验以及持久治疗效果的证据和研究参与者在APHENITY长期扩展研究中放宽饮食的能力。The marketing authorization is applicable to all 27 European Union member states as well as营销授权适用于所有27个欧盟成员国以及. The Sephience European launch will be initiated in. Sephience 的欧洲上市将在Germany德国in the first half of July.在七月份的上半月。A New Drug Application (NDA) for sepiapterin remains on schedule for its FDA target action date ofSepiapterin的新药申请（NDA）在其FDA目标行动日期前仍按计划进行。July 29, 20252025年7月29日. Review of approval applications is ongoing in several other countries including。包括其他国家在内的几个国家正在持续审查批准申请About Sephience™ (sepiapterin)关于Sephience™（sepiapterin）Sephience(sepiapterin) is indicated for the treatment of hyperphenylalaninaemia (HPA) in adult and pediatric patients with phenylketonuria (PKU). Sephience is a natural precursor of the enzymatic co-factor BH4, a critical co-factor for phenylalanine hydroxylase (PAH). Sephience acts as a dual pharmacological chaperone (sepiapterin and BH4 each with its own binding affinity to variant PAH), including PAH variants commonly found in PKU and known to be insensitive to BH4, to improve the activity of the defective PAH enzyme, achieving a high concentration of BH4 intracellularly.Sephience（四氢生物蝶呤）用于治疗成年和儿童苯丙酮尿症（PKU）患者的高苯丙氨酸血症（HPA）。Sephience是酶辅因子BH4的天然前体，BH4是苯丙氨酸羟化酶（PAH）的关键辅因子。Sephience充当双重药理学伴侣（四氢生物蝶呤和BH4各自对变异的PAH有不同的结合亲和力），包括在PKU中常见且已知对BH4不敏感的PAH变体，以改善缺陷性PAH酶的活性，在细胞内实现高浓度的BH4。By enhancing the conformational stability of misfolded PAH enzyme and increasing the intracellular concentrations of BH4, Sephience is able to effectively reduce blood Phe levels. Sephience (sepiapterin) is approved in the European Economic Area. Sepiapterin is an investigational new drug in .通过增强错误折叠的PAH酶的构象稳定性并增加细胞内BH4的浓度，Sephience能够有效降低血液中的Phe水平。Sephience（sepiapterin）已在欧洲经济区获批。Sepiapterin是一种研究性新药。About Phenylketonuria关于苯丙酮尿症Phenylketonuria (PKU) is a rare, inherited metabolic disease, which affects the brain. It is caused by a defect in the gene that helps create the enzyme needed to break down phenylalanine (Phe). If left untreated or poorly managed, Phe — an essential amino acid found in all proteins and most foods — can build up to harmful levels in the body.苯丙酮尿症（PKU）是一种罕见的遗传性代谢疾病，会影响大脑。它是由帮助生成分解苯丙氨酸（Phe）所需酶的基因缺陷引起的。如果不治疗或管理不当，Phe（一种存在于所有蛋白质和大多数食物中的必需氨基酸）可能在体内积累到有害水平。This causes severe and irreversible disabilities, such as permanent intellectual disability, seizures, delayed development, memory loss, and behavioral and emotional problems. Newborns with PKU initially do not have any symptoms, but symptoms are usually progressive, and damage caused by toxic levels of Phe in the first few years of life is irreversible.这会导致严重且不可逆的残疾，例如永久性智力障碍、癫痫发作、发育迟缓、记忆力丧失以及行为和情绪问题。患有PKU的新生儿最初没有任何症状，但症状通常会逐渐加重，生命头几年因苯丙氨酸水平过高造成的损害是不可逆的。Diagnosis of PKU usually takes place during newborn screening programs. There are an estimated 58,000 people living with PKU globally..苯丙酮尿症的诊断通常在新生儿筛查项目中进行。全球约有58,000人患有苯丙酮尿症。About PTC Therapeutics, Inc.关于PTC治疗学公司PTC is a global biopharmaceutical company focused on the discovery, development and commercialization of clinically differentiated medicines that provide benefits to children and adults living with rare disorders. PTC's ability to globally commercialize products is the foundation that drives investment in a robust and diversified pipeline of transformative medicines and our mission to provide access to best-in-class treatments for patients who have an unmet medical need.PTC是一家全球生物制药公司，专注于发现、开发和商业化具有临床差异化的药物，为患有罕见疾病的儿童和成人带来益处。PTC在全球范围内实现产品商业化的能力建立了推动投资的基础，构建了强大且多样化的变革性药物管线，并致力于为存在未满足医疗需求的患者提供最佳治疗方案。The company's strategy is to leverage its strong scientific expertise and global commercial infrastructure to maximize value for its patients and other stakeholders. To learn more about PTC, please visit .公司的战略是利用其强大的科学专业知识和全球商业基础设施，为患者和其他利益相关者实现价值最大化。如需了解更多信息，请访问PTC官网。","kind":"news","is_publish_news":true,"is_publish_highlight":false,"is_publish_live":false,"is_publish_wemedia":null,"editions":null,"column":"","sentiment":"1","news_tag":"productRelease","news_rank":0,"isVideo":false,"video":null,"symbols":[],"gpt_button":1,"need_auth":false,"need_login_tip":false,"code":"91000000","status":"200"},"commentList":[],"isCommentEnd":true,"newsSizeData":{"likeSize":0,"commentSize":0,"repostSize":0,"favoriteSize":0,"likeStatus":false,"favoriteStatus":false},"APP":{"userAgent":"Mozilla/5.0 AppleWebKit/537.36 (KHTML, like Gecko; compatible; ClaudeBot/1.0; +claudebot@anthropic.com)","isDev":false,"isTTM":false,"tenantId":"TBCN","deviceId":"web-server-community-laohu8-v3","version":"4.42.0","shortVersion":"4.42.0","platform":"web","vendor":"web","appName":"laohu8","isIOS":false,"isAndroid":false,"isTiger":false,"isTHS":false,"isWeiXin":false,"isWeiXinMini":false,"isWeiBo":false,"isQQ":false,"isBaiduSwan":false,"isBaiduBox":false,"isDingTalk":false,"isToutiao":false,"isOnePlus":false,"isHuaWei":false,"isXiaomi":false,"isXiaomiWebView":false,"isOppo":false,"isVivo":false,"isSamsung":false,"isMobile":false},"href":"/m/news/2546126867","isCrawlerRequest":true}