PTC Therapeutics收到Vatiquinone新药申请的完整回应函-动脉网

PTC Therapeutics Receives Complete Response Letter for Vatiquinone NDA

CISION 等信源发布 2025-08-19 20:00

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WARREN, N.J.

沃伦，新泽西州

，

Aug. 19, 2025

2025年8月19日

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

/PRNewswire/ -- PTC Therapeutics, Inc.（纳斯达克：

PTCT

) announced today that the U.S. Food and Drug Administration (FDA) has issued a Complete Response Letter (CRL) related to the New Drug Application (NDA) for vatiquinone for the treatment of children and adults living with Friedreich's ataxia.

）今天宣布，美国食品药品监督管理局（FDA）已针对用于治疗患有弗里德赖希共济失调的儿童和成人的瓦替奎酮新药申请（NDA）发布了完整回复信（CRL）。

'We are of course disappointed by the FDA's decision to not approve vatiquinone,' said

“我们对FDA不批准vatiquinone的决定当然感到失望，”

Matthew B. Klein

马修·B·克莱因

, M.D., Chief Executive Officer of PTC Therapeutics. 'We believe the data collected to date demonstrate that vatiquinone could provide a safe and effective therapy for both children and adults living with Friedreich's ataxia. We plan to meet with the FDA to discuss potential steps to address the issues raised in the CRL.'.

，PTC Therapeutics首席执行官医学博士。“我们认为，迄今为止收集的数据表明，vatiquinone 可以为患有弗里德赖希共济失调的儿童和成人提供安全有效的治疗。我们计划与 FDA 会面，讨论解决 CRL 中提出的问题的潜在步骤。”

The FDA stated in the CRL that substantial evidence of efficacy was not demonstrated for vatiquinone and that an additional adequate and well-controlled study would be needed to support NDA resubmission.

FDA在完整回应函中指出，vatiquinone的疗效并未得到充分证据的支持，并且需要另外一项充分且控制良好的研究来支持新药申请的重新提交。

About Vatiquinone

关于Vatiquinone

Vatiquinone is a small molecule, first-in-class selective inhibitor of 15-Lipoxygenase (15-LO), an enzyme that is a key regulator of the energetic and oxidative stress pathways that are disrupted in Friedreich's ataxia. Inhibition of 15-LO helps to alleviate the consequences of mitochondrial dysfunction and oxidative stress, ultimately decreasing inflammation and oxidative stress and promoting neuronal survival..

Vatiquinone 是一种小分子，是首个选择性抑制 15-脂氧合酶（15-LO）的药物，15-LO 是一种在弗里德赖希共济失调中调控能量和氧化应激通路的关键酶。抑制 15-LO 有助于缓解线粒体功能障碍和氧化应激的后果，最终减少炎症和氧化应激，并促进神经元存活。

1,2,3

Vatiquinone has been evaluated in a number of clinical studies, many focused on pediatric patients, and has demonstrated an impact on mortality risk, and a number of neurological and neuromuscular disease symptoms.

Vatiquinone已经在多项临床研究中得到评估，其中许多研究聚焦于儿科患者，并且已经显示出对死亡风险的影响，以及多种神经和神经肌肉疾病症状的改善。

About Friedreich's Ataxia

关于弗里德赖希共济失调

Friedreich's ataxia (FA) is a rare, physically debilitating, life-shortening, neuromuscular disorder that mainly affects the central nervous system and the heart.

弗里德赖希共济失调（FA）是一种罕见的、导致身体虚弱、缩短寿命的神经肌肉疾病，主要影响中枢神经系统和心脏。

It is the most common hereditary ataxia (abnormal, uncoordinated movements) and is usually caused by a single genetic defect in the frataxin (FXN) gene that leads to reduced production of frataxin, a mitochondrial protein that is important for cellular metabolism and energy production.

它是最常见的遗传性共济失调（异常、不协调的运动），通常由frataxin（FXN）基因中的单一基因缺陷引起，该缺陷导致frataxin（一种对细胞代谢和能量生产重要的线粒体蛋白）的产量减少。

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Decreased frataxin levels are associated with mitochondrial iron accumulation and increased oxidative stress, which can lead to cell death through ferroptosis.

Frataxin水平的降低与线粒体铁积累和氧化应激增加有关，这可能通过铁死亡导致细胞死亡。

6,7,8

Symptoms include progressive loss of coordination and muscle strength leading to poor balance and coordination, difficulty speaking, swallowing, and breathing, curvature of the spine, serious heart conditions, diabetes, and hearing and vision impairment.

症状包括协调性和肌肉力量的逐渐丧失，导致平衡和协调能力差、说话、吞咽和呼吸困难、脊柱弯曲、严重的心脏病、糖尿病以及听力和视力障碍。

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The severity of symptoms and speed of progression varies between people and some symptoms may not be evident in all. Friedreich's ataxia is usually diagnosed in childhood or adolescence.

症状的严重程度和进展速度因人而异，并且某些症状可能并非在所有人身上都明显。弗里德赖希共济失调通常在儿童或青少年时期被诊断出来。

5,11

Approximately 25,000 people have Friedreich's ataxia globally.

全球大约有25,000人患有弗里德赖希共济失调。

About PTC Therapeutics, Inc.

关于PTC治疗学公司

PTC is a global biopharmaceutical company dedicated to the discovery, development and commercialization of clinically differentiated medicines for children and adults living with rare disorders. PTC is advancing a robust and diversified pipeline of transformative medicines as part of its mission to provide access to best-in-class treatments for patients with unmet medical needs.

PTC是一家全球生物制药公司，致力于为患有罕见疾病的儿童和成人发现、开发和商业化临床差异化的药物。PTC正在推进一系列强大且多样化的变革性药物管道，作为其为未满足医疗需求的患者提供最佳治疗方案使命的一部分。

The company's strategy is to leverage its scientific expertise and global commercial infrastructure to optimize value for patients and other stakeholders. To learn more about PTC, please visit .

该公司的战略是利用其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优化价值。欲了解更多信息，请访问PTC官网。

www.ptcbio.com

and follow on Facebook, X, and LinkedIn.

并在Facebook、X和LinkedIn上关注。

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

珍妮娜·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 press release, other than statements of historic fact, are forward-looking statements, including statements with respect to the future expectations, plans and prospects for PTC; PTC's plans for further interactions with the FDA; PTC's plans for vatiquinone including with respect to the expected timing of clinical trials and studies, availability of data, regulatory submissions and responses and other matters; 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PTC未来预期、计划和前景的陈述；PTC与FDA进一步互动的计划；PTC关于vatiquinone的计划，包括临床试验和研究的预期时间、数据可用性、监管提交和回应及其他事项；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以下词语来识别：“指导”、“计划”、“预期”、“相信”、“估计”、“预计”、“意图”、“可能”、“目标”、“潜力”、“将”、“会”、“可以”、“应该”、“继续”以及类似的表达方式。

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 vatiquinone, including any clinical trials, regulatory submissions, commercialization, 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 Quarterly Report on Form 10-Q and 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，原因是多种风险和不确定性，包括：与第三方支付方就PTC已商业化或未来可能商业化的现有产品或候选产品进行的价格、覆盖范围和报销谈判的结果；关于vatiquinone的预期，包括任何临床试验、监管提交、商业化、潜在的监管和销售里程碑以及PTC可能需要支付的附条件款项；重大的业务影响，包括行业、市场、经济、政治或监管环境的影响；税收及其他法律、法规、费率和政策的变化；PTC产品及候选产品的适用患者群体和市场潜力；PTC的科学方法及总体研发进展；以及PTC最近季度报告Form 10-Q和年度报告Form 10-K中的“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 vatiquinone..

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vatiquinone（瓦替喹酮）。

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不承担也不计划更新或修改任何此类前瞻性陈述，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。

References:

参考文献：

Hinman A, et al. PLoS one. 2018;13:e0201369.

Hinman A, 等。PLoS ONE。2018;13:e0201369。

PTC Therapeutics. EPI-743 Pre-Clinical Data Deck.

PTC Therapeutics. EPI-743 临床前数据汇总。

Shrader WD, et al. Bioorg Med Chem Lett. 2011;21:3693–3698.

Shrader WD, 等。《生物有机与医药化学快报》。2011年；21:3693–3698。

Lynch DR, Farmer JM, Balcer LJ, et al. Arch Neurol 2002;59(5):743–747.

林奇 DR，法默 JM，巴尔塞 LJ，等。《神经学文献》2002；59（5）：743-747。

Campuzano V, Montermini L, Lutz Y, et al. Hum Mol Genet 1997;11(6):1771–1780.