优时比KYGEVVI获FDA批准，为首个也是唯一用于治疗患有胸苷激酶2缺乏症的成人和儿童的药物-动脉网

U.S. FDA approves KYGEVVI[®] (doxecitine and doxribtimine), the first and only treatment for adults and children living with thymidine kinase 2 deficiency (TK2d)

优时比等信源发布 2025-11-03 05:07

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可切换为仅中文

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Brussels (Belgium) 03 November 2025, 22:00: (CET)

布鲁塞尔（比利时）2025年11月3日，22:00（欧洲中部时间）

– UCB (Euronext Brussels: UCB), a global biopharmaceutical company, today announced that KYGEVVI

– UCB（欧洲布鲁塞尔证券交易所：UCB），一家全球生物制药公司，今天宣布KYGEVVI

has been granted approval by the U.S. Food and Drug Administration (FDA) for the treatment of adults and pediatric patients living with thymidine kinase 2 deficiency (TK2d), with an age of symptom onset on or before 12 years.

已获得美国食品药品监督管理局 (FDA) 批准，用于治疗患有胸苷激酶 2 缺乏症 (TK2d) 的成人和儿童患者，症状发病年龄在 12 岁或之前。

It is the first and only approved treatment for these patients living with TK2d.

它是首个也是唯一获批的TK2d患者治疗方案。

TK2d is an ultra-rare, life-threatening, genetic mitochondrial disease characterized by progressive (worsening over time) and severe muscle weakness (myopathy) with no approved treatment options beyond supportive care until now.

TK2d 是一种超罕见、危及生命的遗传性线粒体疾病，其特征是进行性（随时间恶化）和严重的肌肉无力（肌病），迄今为止除了支持性护理外尚无获批的治疗方案。

2,3,4,5

It is often fatal, with those experiencing initial symptoms on or before the age of 12 years facing a high risk of premature death (often occurring within 3 years after symptom onset).

它往往是致命的，12岁或之前出现初始症状的患者面临较高的早逝风险（通常在症状出现后的3年内发生）。

It is estimated that the worldwide prevalence of TK2d is 1.64 [0.5, 3.1] cases per 1,000,000 people.

据估计，TK2d 的全球患病率为每 1,000,000 人中 1.64 [0.5, 3.1] 例。

“The approval of doxecitine and doxribtimine represents a pivotal moment for the TK2d community who previously had no FDA-approved treatment options for this rare genetic mitochondrial disease beyond supportive [palliative] care,” said Donatello Crocetta, Chief Medical Officer at UCB. “We extend heartfelt thanks to the patients, families and friends, advocates, healthcare providers and dedicated clinical trial teams who have partnered with us on this important journey.”.

“Doxecitine 和 doxribtimine 的获批对 TK2d 患者群体而言是一个关键时刻，他们此前除了支持性（姑息性）治疗外，没有 FDA 批准的针对这种罕见遗传性线粒体疾病的治疗选择，”UCB 首席医学官唐纳泰罗·克罗切塔表示。“我们衷心感谢与我们在这段重要旅程中合作的患者、家属和朋友、倡导者、医疗保健提供者以及专注的临床试验团队。”

“It’s hard to overstate the importance of this FDA approval for those diagnosed with TK2d. This is an ultra-rare disease community in dire need of treatment options. For too long, caregivers and their families have had to endure the burden of this disease,' said Kristen Clifford, United Mitochondrial Disease Foundation President and CEO.

“对于那些被诊断为TK2d的人来说，这一FDA批准的重要性难以言过其实。这是一个急需治疗选择的超罕见疾病群体。长久以来，护理人员及其家人都不得不承受这种疾病的重担，”联合线粒体疾病基金会总裁兼首席执行官克里斯汀·克利福德说道。

“Having the first-ever FDA-approved therapy for TK2d in this patient population not only meets a critical medical need - it represents something greater - hope for the future.'.

“在这一患者群体中拥有首个FDA批准的TK2d疗法，不仅满足了关键的医疗需求——它代表了更伟大的事物——对未来的希望。”

“I’ve been studying mitochondrial diseases for more than three decades and have witnessed firsthand the impact TK2d has on patients and their families. We have been waiting for an approved treatment for many years, and this approval marks a significant milestone in how we can support and manage this debilitating condition,” said Dr.

“我研究线粒体疾病已经超过三十年，亲眼目睹了TK2d对患者及其家人的影响。我们多年来一直在等待一种获批的治疗方法，而此次批准标志着我们在支持和管理这种衰竭性疾病方面迈出了重要的里程碑，”博士说道。

Michio Hirano, Professor of Neurology and Chief of the Division of Neuromuscular Medicine at Columbia University Irving Medical Center..

哥伦比亚大学欧文医学中心神经病学教授、神经肌肉医学科主任Michio Hirano。

Supporting data

支持数据

The KYGEVVI approval is supported by safety and efficacy data from one Phase 2 clinical study, two retrospective chart review studies, and an expanded access use program*.

KYGEVVI的批准得到了一项二期临床研究、两项回顾性图表审查研究和一个扩展使用计划*的安全性和有效性数据的支持。

1,8,9,10,11

These studies included a total of 82 unique patients treated with KYGEVVI or pyrimidine nucleosides with an age of TK2d symptom onset ≤12 years. Efficacy was assessed by comparing overall survival in these pediatric and adult treated patients to an external control group of untreated patients who were matched to treated patients using age of TK2d symptom onset (≤ 2 years or >2 to ≤ 12 years)..

这些研究总共包括 82 名独特的患者，这些患者接受了 KYGEVVI 或嘧啶核苷治疗，且 TK2d 症状发作年龄 ≤12 岁。疗效评估是通过将这些接受治疗的儿童和成人患者的总生存期与一个外部未治疗患者对照组进行比较，该对照组根据 TK2d 症状发作年龄（≤ 2 岁或 >2 至 ≤ 12 岁）与治疗患者匹配。

A total of 78 matched pairs were identified. The results showed that survival time from treatment start was improved; treatment reduced the overall risk of death from treatment start by approximately 86% (95% CI: 61%, 96%).

共确定了78对匹配对。结果显示，从治疗开始的生存时间有所改善；治疗使从治疗开始的总体死亡风险降低了约86%（95%置信区间：61%，96%）。

Of the 78 treated patients included in the survival analysis, the median age of TK2d symptom onset was 1.5 years (range: 0.01 to 12 years).

在生存分析中纳入的78名治疗患者中，TK2d症状发作的中位年龄为1.5岁（范围：0.01至12岁）。

The median duration of treatment was 4 years (range: 1 day to 12 years) and the median dose received was 762 mg/kg/day (range: 260 to 800 mg/kg/day).

治疗的中位持续时间为4年（范围：1天至12年），接受的中位剂量为762毫克/千克/天（范围：260至800毫克/千克/天）。

The most common adverse reactions (incidence ≥5%) are diarrhea, abdominal pain (including abdominal pain upper), vomiting, alanine aminotransferase increased (ALT), and aspartate aminotransferase increased (AST).

最常见的不良反应（发生率≥5%）是腹泻、腹痛（包括上腹痛）、呕吐、丙氨酸氨基转移酶升高（ALT）和天冬氨酸氨基转移酶升高（AST）。

A regulatory review of doxecitine and doxribtimine is currently underway by the EMA (European Medicines Agency), and further regulatory submissions are planned. KYGEVVI is currently not approved for use in any indication by any regulatory authority outside of the U.S. UCB expects KYGEVVI to be commercially available in the U.S.

欧洲药品管理局（EMA）目前正在对多西汀和多西替明进行监管审查，并计划提交更多的监管文件。KYGEVVI目前尚未获得美国以外任何监管机构的任何适应症批准。UCB预计KYGEVVI将在美国上市销售。

in Q1, 2026. To further its mission of equitable care, UCB will provide a personalized support program for KYGEVVI that places the needs of patients and caregivers at the forefront. .

2026年第一季度。为了进一步实现公平护理的使命，UCB将为KYGEVVI提供一个个性化支持计划，将患者和护理人员的需求置于首位。

In the U.S., KYGEVVI received Orphan Drug, Breakthrough, Priority Review and Rare Pediatric Disease designations from the FDA.

在美国，KYGEVVI 获得了 FDA 授予的孤儿药、突破性疗法、优先审评和罕见儿科疾病认定。

12,13

12，13

With this approval by FDA, UCB was awarded a Rare Pediatric Disease Priority Review Voucher (RPDPRV) redeemable for a priority review for a future marketing application.

随着FDA的批准，UCB获得了一张罕见儿科疾病优先审查券（RPDPRV），可用于未来上市申请的优先审查。

About KYGEVVI

关于KYGEVVI

KYGEVVI is a combination of doxecitine and doxribtimine, both pyrimidine nucleosides, indicated for the treatment of thymidine kinase 2 deficiency (TK2d) in adults and pediatric patients with an age of symptom onset on or before 12 years.

KYGEVVI 是多西西丁和多西布汀的组合，二者均为嘧啶核苷，用于治疗成人及症状发作年龄在 12 岁或之前的儿童患者的胸苷激酶 2 缺乏症 (TK2d)。

Administration of KYGEVVI is intended to incorporate the pyrimidine nucleosides, deoxycytidine and deoxythymidine, into skeletal muscle mitochondrial DNA.1 This action restores mitochondrial DNA copy number in TK2d mutant mice.

KYGEVVI 的给药旨在将嘧啶核苷脱氧胞苷和脱氧胸苷整合到骨骼肌线粒体 DNA 中。这种作用可恢复 TK2d 突变小鼠的线粒体 DNA 拷贝数。

Important safety information for KYGEVVI

KYGEVVI的重要安全信息

Increase in Liver Transaminases

肝转氨酶升高

Elevated liver transaminase [alanine aminotransferase (ALT) and/or aspartate aminotransferase (AST)] levels were reported in patients treated with KYGEVVI. Obtain baseline liver transaminase (ALT, AST) and total bilirubin levels in patients prior to treatment initiation with KYGEVVI. If signs or symptoms consistent with liver injury are observed, interrupt treatment with KYGEVVI until liver transaminase (ALT, AST) and total bilirubin levels have either returned to baseline or stabilized at a new baseline value.

据报道，使用KYGEVVI治疗的患者肝转氨酶[丙氨酸氨基转移酶（ALT）和/或天门冬氨酸氨基转移酶（AST）]水平升高。在开始使用KYGEVVI治疗前，获取患者的基线肝转氨酶（ALT、AST）和总胆红素水平。如果观察到与肝损伤相符的体征或症状，应中断KYGEVVI治疗，直到肝转氨酶（ALT、AST）和总胆红素水平恢复到基线或稳定在新的基线值。

Consider permanently discontinuing KYGEVVI if signs or symptoms consistent with liver injury persist or worsen. Monitor liver transaminases and total bilirubin levels yearly and as clinically indicated..

如果出现与肝脏损伤一致的体征或症状持续存在或恶化，请考虑永久停用KYGEVVI。每年监测肝转氨酶和总胆红素水平，并根据临床需要进行监测。

Gastrointestinal Adverse Reactions

胃肠道不良反应

Diarrhea and vomiting leading to hospitalization, dose reduction, and permanent discontinuation were reported in patients treated with KYGEVVI. Based on the severity of the diarrhea and/or vomiting, reduce the dosage of KYGEVVI or interrupt treatment until diarrhea and/or vomiting improves or returns to baseline.

据报道，接受KYGEVVI治疗的患者出现腹泻和呕吐，导致住院、剂量减少以及永久停药。根据腹泻和/或呕吐的严重程度，减少KYGEVVI的剂量或中断治疗，直至腹泻和/或呕吐改善或恢复至基线水平。

Consider restarting KYGEVVI at the last tolerated dose, and increase the dose as tolerated. For persistent or recurring diarrhea and/or vomiting, consider discontinuing KYGEVVI permanently and provide supportive care with electrolyte repletion as clinically indicated..

考虑以患者最后可耐受的剂量重新开始使用KYGEVVI，并根据耐受情况增加剂量。对于持续或反复出现的腹泻和/或呕吐，考虑永久停用KYGEVVI，并根据临床需要通过补充电解质提供支持性护理。

Please see the full

请参阅完整内容

U.S Prescribing Information

美国处方信息

for additional information. Talk to your healthcare provider about your condition or your treatment.

获取更多信息，请咨询您的医疗保健提供者有关您的病情或治疗方案。

*The KYGEVVI® approval is supported by safety and efficacy data from one Phase 2 clinical study (Trial 1, (NCT03845712), two retrospective chart review studies (Study 1 and Study 2, NCT03701568 and NCT05017818 respectively), and an expanded access use program (NCT06590493).

KYGEVVI® 的获批得到了一项二期临床试验（试验 1，(NCT03845712)）、两项回顾性图表审查研究（研究 1 和研究 2，分别为 NCT03701568 和 NCT05017818）以及一项扩大使用计划（NCT06590493）的安全性和有效性数据的支持。

For more information about the trial visit:

有关试验的更多信息，请访问：

https://clinicaltrials.gov/study/NCT03845712

(NCT03845712);

https://clinicaltrials.gov/study/NCT03701568

(NCT03701568);

https://clinicaltrials.gov/study/NCT05017818

(NCT05017818);

https://clinicaltrials.gov/study/NCT06590493

(NCT06590493).

(NCT06590493)。

For further information, contact UCB:

如需更多信息，请联系UCB：

Global Communications

全球通讯

Nick Francis

尼克·弗朗西斯

T: +44 7769 307745

电话：+44 7769 307745

nick.francis@ucb.com

US Rare Diseases Communications

美国罕见病通讯

Daphne Teo

张达芬

T: +1 770.880.7655

电话：+1 770.880.7655

daphne.teo@ucb.com

Corporate Communications, Media Relations

企业传播、媒体关系

Laurent Schots

劳伦特·肖茨

T +32.2.559.92.64

电话：+32.2.559.92.64

laurent.schots@ucb.com

Investor Relations

投资者关系

Antje Witte

安特耶·维特

T +32.2.559.94.14

电话：+32.2.559.94.14

antje.witte@ucb.com

About UCB

关于UCB

UCB, Brussels, Belgium (www.ucb.com) is a global biopharmaceutical company focused on the discovery and development of innovative medicines and solutions to transform the lives of people living with severe diseases of the immune system or of the central nervous system. With more than 9 000 people in approximately 40 countries, the company generated revenue of € 6.1 billion in 2024.

优时比公司（UCB），位于比利时布鲁塞尔（www.ucb.com），是一家全球生物制药公司，专注于发现和开发创新药物及解决方案，以改变免疫系统或中枢神经系统严重疾病患者的生活。该公司在约40个国家拥有9000多名员工，并在2024年创造了61亿欧元的收入。

UCB is listed on Euronext Brussels (symbol: UCB). Follow us on Twitter: @UCB_news.

优时比在布鲁塞尔泛欧交易所上市（股票代码：UCB）。关注我们的Twitter：@UCB_news。

Forward-looking statements

前瞻性声明

This document contains forward-looking statements, including, without limitation, statements containing the words “potential”, “believes”, “anticipates”, “expects”, “intends”, “plans”, “seeks”, “estimates”, “may”, “will”, “continue” and similar expressions. These forward-looking statements are based on current plans, estimates and beliefs of management.

本文件包含前瞻性陈述，包括但不限于包含“潜在”、“相信”、“预期”、“预计”、“意图”、“计划”、“寻求”、“估计”、“可能”、“将”、“继续”等词语及类似表达的陈述。这些前瞻性陈述基于管理层当前的计划、估计和信念。

All statements, other than statements of historical facts, are statements that could be deemed forward-looking statements, including estimates of revenues, operating margins, capital expenditures, cash, other financial information, expected legal, arbitration, political, regulatory or clinical results or practices and other such estimates and results.

所有声明，除历史事实声明外，均可能被视为前瞻性声明，包括收入、营业利润率、资本支出、现金、其他财务信息、预期法律、仲裁、政治、监管或临床结果或实践以及其他此类估计和结果。

By their nature, such forward-looking statements are not guaranteeing future performance and are subject to known and unknown risks, uncertainties, and assumptions which might cause the actual results, financial condition, performance or achievements of UCB, or industry results, to be materially different from any future results, performance, or achievements expressed or implied by such forward-looking statements contained in this document. .

由于此类前瞻性陈述的性质，它们并不保证未来的表现，并且受到已知和未知风险、不确定性以及可能导致UCB的实际结果、财务状况、表现或成就（或行业结果）与本文件中包含的此类前瞻性陈述明示或暗示的任何未来结果、表现或成就存在重大差异的因素影响。

Important factors that could result in such differences include but are not limited to: global spread and impacts of wars, pandemics and terrorism, the general geopolitical environment, climate change, changes in general economic, business and competitive conditions, the inability to obtain necessary regulatory approvals or to obtain them on acceptable terms or within expected timing, costs associated with research and development, changes in the prospects for products in the pipeline or under development by UCB, effects of future judicial decisions or governmental investigations, safety, quality, data integrity or manufacturing issues, supply chain disruption and business continuity risks; potential or actual data security and data privacy breaches, or disruptions of UCB’s information technology systems, product liability claims, challenges to patent protection for products or product candidates, competition from other products including biosimilars or disruptive technologies/business models, changes in laws or regulations, exchange rate fluctuations, changes or uncertainties in laws and/or rules pertaining to tax and duties or the administration of such laws and/or rules, and hiring, retention and compliance of employees.

可能导致此类差异的重要因素包括但不限于：战争、疫情和恐怖主义的全球蔓延及影响、总体地缘政治环境、气候变化、总体经济、商业和竞争状况的变化、无法获得必要的监管批准或在可接受的条件或预期时间内获得批准、与研发相关的成本、UCB在研产品或开发中产品的前景变化、未来司法裁决或政府调查的影响、安全性、质量、数据完整性或制造问题、供应链中断和业务连续性风险；潜在或实际的数据安全和隐私泄露，或UCB信息技术系统的中断、产品责任索赔、对产品或候选产品的专利保护挑战、来自其他产品（包括生物仿制药或颠覆性技术/商业模式）的竞争、法律法规的变化、汇率波动、税收和关税相关法律和/或规则及其管理的变化或不确定性，以及员工的招聘、留任和合规问题。

There is no guarantee that new product candidates will be discovered or identified in the pipeline, or that new indications for existing products will be developed and approved. Movement from concept to commercial product is uncertain; preclinical results do not guarantee safety and efficacy of product candidates in humans.

无法保证在研发管线中会发现或识别出新的候选产品，也无法保证现有产品的新增适应症能够被开发并获得批准。从概念到商业产品的推进过程充满不确定性；临床前结果不能保证候选产品在人体中的安全性和有效性。

So far, the complexity of the human body cannot be reproduced in computer models, cell culture systems or animal models. The length of the timing to complete clinical trials and to get regulatory approval for product marketing has.

到目前为止，计算机模型、细胞培养系统或动物模型都无法再现人体的复杂性。完成临床试验和获得产品上市监管批准的时间长度已经。

Given these uncertainties, the public is cautioned not to place any undue reliance on such forward-looking statements. These forward-looking statements are made only as of the date of this document, and do not reflect any potential impacts from the evolving event or risk as mentioned above as well as any other adversity, unless indicated otherwise.

鉴于这些不确定性，公众应注意不要对上述前瞻性声明给予任何过度依赖。这些前瞻性声明仅于本文档发布之日作出，且并未反映上述不断演变的事件或风险以及任何其他不利因素的潜在影响，除非另有说明。

The company continues to follow the development diligently to assess the financial significance of these events, as the case may be, to UCB..

公司继续勤勉地跟进事态发展，以评估这些事件对UCB的财务影响（如有）。

UCB expressly disclaims any obligation to update any forward-looking statements in this document, either to confirm the actual results or to report or reflect any change in its forward-looking statements with regard thereto or any change in events, conditions or circumstances on which any such statement is based, unless such statement is required pursuant to applicable laws and regulations..

UCB明确表示，除非适用法律法规要求，否则不承担更新本文件中任何前瞻性声明的义务，无论这些声明是为确认实际结果，还是为报告或反映与其相关的任何变化，或任何此类声明所基于的事件、条件或情况的任何变化。

References:

参考文献：

KYGEVVI

U.S. Prescribing Information

美国处方信息

Berardo A, et al. Advances in Thymidine Kinase 2 Deficiency: Clinical Aspects, Translational Progress, and Emerging Therapies. J Neuromuscul Dis. 2022;9(2):225-235.

Berardo A, 等。胸苷激酶2缺乏症的进展：临床方面、转化研究进展及新兴疗法。《神经肌肉疾病杂志》。2022年；9(2):225-235。

Wang J, et al. eds. GeneReviews®. [Internet]. Seattle (WA): University of Washington, Seattle; 1993-2024.

王 J 等编。《基因评论》®。[互联网]。西雅图（华盛顿州）：华盛顿大学，西雅图；1993-2024。

Domínguez-González C, et al. Late-onset thymidine kinase 2 deficiency: a review of 18 cases. Orphanet J Rare Dis. 2019;14(1):100.

多明格斯-冈萨雷斯 C 等。迟发性胸苷激酶2缺乏症：18例病例回顾。《孤儿病杂志：罕见病》2019；14(1):100。

National Institute of Health. TK2-related mitochondrial DNA depletion syndrome, myopathic form.

国家卫生研究院。TK2相关的线粒体DNA耗竭综合征，肌病形式。

https://medlineplus.gov/genetics/condition/tk2-related-mitochondrial-dna-depletion-syndrome-myopathic-form/#genes

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访问时间：2025年10月。

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