PTC Therapeutics收到CHMP对Sephience™（sepiapterin）用于治疗苯丙酮尿症（PKU）儿童和成人患者的积极意见-动脉网

PTC Therapeutics Receives Positive CHMP Opinion for Sephience™ (sepiapterin) for the Treatment of Children and Adults Living with Phenylketonuria (PKU)

CISION 等信源发布 2025-04-25 21:00

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- Label includes full spectrum of PKU patients -

- 标签涵盖PKU患者的全部范围 -

- European launch preparations underway -

- 欧洲上市准备工作正在进行中 -

WARREN, N.J.

沃伦，新泽西州

，

April 25, 2025

2025年4月25日

/PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ:

PTCT

) announced today that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has issued a positive opinion on the marketing authorization application for Sephience™ (sepiapterin) for the treatment of children and adults living with phenylketonuria (PKU). The opinion includes a broad label inclusive of all ages and disease severities..

）今天宣布，欧洲药品管理局（EMA）人用医药产品委员会（CHMP）已对Sephience™（sepiapterin）用于治疗苯丙酮尿症（PKU）儿童和成人的上市许可申请发表了积极意见。该意见包括涵盖所有年龄和疾病严重程度的广泛标签。

'This positive opinion is great news for the PKU patient community in

“这一积极的意见对于苯丙酮尿症（PKU）患者群体来说是个好消息，

Europe

欧洲

for whom there remains a significant unmet medical need,' said Matthew B. Klein, M.D., Chief Executive Officer of PTC Therapeutics. 'We look forward to initiating the European launch immediately following the adoption of this positive opinion by the European Commission. The data collected to date continue to support the potential for Sephience to provide a safe and effective treatment that not only lowers phenylalanine levels but also enables diet liberalization even for those with the most severe disease subtypes.'.

“对于那些仍有重大未满足医疗需求的患者，”PTC Therapeutics首席执行官Matthew B. Klein博士说道，“我们期待在欧洲委员会采纳这一积极意见后立即启动欧洲市场推广。迄今为止收集的数据继续支持Sephience作为一种安全有效治疗的潜力，它不仅能够降低苯丙氨酸水平，还能够实现饮食宽松，即使是针对病情最严重的亚型患者。”

Launch planning in

启动计划于

Europe

欧洲

is well underway with priorities in

正在顺利进行中，并优先考虑

Germany

德国

and other key European markets where named patient access will be immediately available.

和其他可立即获得指定患者使用权的关键欧洲市场。

PTC expects the European Commission to ratify the marketing authorization for Sephience in approximately two months. The decision will be applicable to all 27 European Union member states, as well as

PTC预计欧洲委员会将在大约两个月内批准Sephience的上市许可。该决定将适用于所有27个欧盟成员国，以及

Iceland

冰岛

，

Norway

挪威

and

和

Liechtenstein

列支敦士登

。

The New Drug Application (NDA) for sepiapterin is under review by the U.S. Food and Drug Administration (FDA) with a Prescription Drug User Fee Act (PDUFA) target action date of

Sepiapterin的新药申请（NDA）正在由美国食品药品监督管理局（FDA）审查，处方药用户收费法案（PDUFA）的目标行动日期为

July 29, 2025

2025年7月29日

. Review of approval applications is ongoing in several other countries including

. 审批申请正在包括以下几个国家进行中

Japan

日本

and Brazil.

和巴西。

About Sephience™ (sepiapterin)

关于Sephience™（sepiapterin）

Sephience, an oral formulation of synthetic sepiapterin, has a dual mechanism of action to increase activity of the phenylalanine hydroxylase (PAH) enzyme. First, Sephience is a precursor compound that is rapidly absorbed and converted intracellularly to tetrahydrobiopterin (BH4), a critical cofactor of PAH.

Sephience 是一种合成蝶呤的口服制剂，具有双重作用机制来增加苯丙氨酸羟化酶 (PAH) 的活性。首先，Sephience 是一种前体化合物，能够被快速吸收并在细胞内转化为四氢生物蝶呤 (BH4)，而 BH4 是 PAH 的关键辅因子。

Sephience also has an independent pharmacological chaperone effect, correcting PAH misfolding to enhance the enzyme function. Through this dual mechanism of action, Sephience effectively reduces blood phenylalanine (Phe) levels and has the potential to treat a broad range of PKU patients..

Sephience还具有独立的药理伴侣效应，可纠正PAH错误折叠以增强酶功能。通过这种双重作用机制，Sephience有效降低血液苯丙氨酸（Phe）水平，并有潜力治疗广泛的PKU患者。

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）可能会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 don't 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的新生儿最初没有症状，但症状通常会逐渐显现，生命头几年因苯丙氨酸毒性水平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 that discovers, develops and commercializes clinically differentiated medicines that provide benefits to children and adults living with rare disorders. PTC's ability to innovate new therapies and to globally commercialize products is the foundation that drives investment in a robust and diversified pipeline of transformative medicines.

PTC是一家全球生物制药公司，致力于发现、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创新能力以及在全球范围内推广新产品的能力，是推动其对一系列变革性药物进行稳健而多样化投资的基础。

To learn more about PTC, please visit us at .

要了解有关PTC的更多信息，请访问我们。

www.ptcbio.com

and follow us on Facebook, X, and LinkedIn.

并在Facebook、X和LinkedIn上关注我们。

For More Information:

更多信息：

Investors:

投资者：

Ellen Cavaleri

艾伦·卡瓦莱里

+1 (615) 618-6228

ecavaleri@ptcbio.com

Media:

媒体：

Jeanine Clemente

珍妮·克莱门特

+1 (908) 912-9406

jclemente@ptcbio.com

Forward-Looking Statement

前瞻性声明

This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. All statements contained in this release, other than statements of historic fact, are forward-looking statements, including statements regarding: the future expectations, plans and prospects for PTC, including with respect to the expected timing of clinical trials and studies, availability of data, regulatory submissions and responses, commercialization and other matters with respect to its products and product candidates; expectations with respect to Sephience, including any regulatory decision made by the European Commission; PTC's strategy, future operations, future financial position, future revenues, projected costs; and the objectives of management.

本新闻稿包含《1995年私人证券诉讼改革法案》所指的前瞻性陈述。本新闻稿中包含的所有陈述，除历史事实陈述外，均为前瞻性陈述，包括关于以下方面的陈述：PTC的未来预期、计划和前景，包括临床试验和研究的预期时间、数据的可用性、监管提交和回应、商业化及其他与其产品和候选产品相关的事项；关于Sephience的预期，包括欧盟委员会作出的任何监管决定；PTC的战略、未来运营、未来财务状况、未来收入、预计成本；以及管理层的目标。

Other forward-looking statements may be identified by the words, 'guidance', 'plan,' 'anticipate,' 'believe,' 'estimate,' 'expect,' 'intend,' 'may,' 'target,' 'potential,' 'will,' 'would,' 'could,' 'should,' 'continue,' and similar expressions..

其他前瞻性陈述可能通过“指导”、“计划”、“预期”、“相信”、“估计”、“预测”、“打算”、“可能”、“目标”、“潜力”、“将”、“会”、“能够”、“应该”、“继续”等词语和类似表述加以识别。

PTC's actual results, performance or achievements could differ materially from those expressed or implied by forward-looking statements it makes as a result of a variety of risks and uncertainties, including those related to: the outcome of pricing, coverage and reimbursement negotiations with third party payors for PTC's products or product candidates that PTC commercializes or may commercialize in the future; expectations with respect to Sephience, including any regulatory submissions and potential approvals, commercialization and the potential achievement of regulatory and sales milestones and contingent payments that PTC may be obligated to make; significant business effects, including the effects of industry, market, economic, political or regulatory conditions; changes in tax and other laws, regulations, rates and policies; the eligible patient base and commercial potential of PTC's products and product candidates; PTC's scientific approach and general development progress; and the factors discussed in the 'Risk Factors' section of PTC's most recent Annual Report on Form 10-K, as well as any updates to these risk factors filed from time to time in PTC's other filings with the SEC.

PTC的实际结果、业绩或成就可能与其前瞻性声明中表达或暗示的内容存在重大差异，原因在于多种风险和不确定性，包括以下相关因素：PTC对其已商业化或将要商业化的产品或候选产品与第三方支付方进行的价格、覆盖范围和报销谈判的结果；对Sephience的预期，包括任何监管提交和潜在批准、商业化以及PTC可能需要履行的监管和销售里程碑及或有付款；重大的业务影响，包括行业、市场、经济、政治或监管条件的影响；税收及其他法律、法规、税率和政策的变化；PTC产品和候选产品的适用患者群体及市场潜力；PTC的科学方法和总体开发进展；以及PTC最近一份Form 10-K年报中“风险因素”部分讨论的因素，以及PTC在其他提交给美国证券交易委员会（SEC）的文件中不时更新的这些风险因素。

You are urged to carefully consider all such factors..

您被敦促仔细考虑所有这些因素。

As with any pharmaceutical under development, there are significant risks in the development, regulatory approval, and commercialization of new products. There are no guarantees that any product will receive or maintain regulatory approval in any territory, or prove to be commercially successful, including Sephience..

与任何正在开发的药物一样，新产品的开发、监管审批和商业化过程中存在重大风险。无法保证任何产品会在任何地区获得或保持监管批准，或证明其商业上的成功，包括Sephience。

The forward-looking statements contained herein represent PTC's views only as of the date of this press release and PTC does not undertake or plan to update or revise any such forward-looking statements to reflect actual results or changes in plans, prospects, assumptions, estimates or projections, or other circumstances occurring after the date of this press release except as required by law..

本新闻稿中包含的前瞻性陈述仅代表PTC截至本新闻稿发布日期的观点，PTC不承担更新或修改任何前瞻性陈述的义务，以反映实际结果或计划、前景、假设、估计或预测的变化，或其他在本新闻稿发布日期之后发生的情况，除非法律要求。

SOURCE PTC Therapeutics, Inc.

来源：PTC Therapeutics, Inc.

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