- PDUFA target action date of June 30, 2026 -

- PDUFA目标行动日期为2026年6月30日 -

- Priority Review designation is granted to applications for drugs that, if approved, would be a significant improvement in the safety or effectiveness of treating a serious condition -

- 优先审查资格授予那些如果获得批准，将在治疗严重疾病的安全性或有效性方面取得重大改进的药物申请 -

- Veligrotug now has both Priority Review and Breakthrough Therapy Designations, each following requests which included data on veligrotug’s (i) consistent and robust improvement and resolution of diplopia in chronic TED, and (ii) rapid onset of proptosis response -

- Veligrotug现已获得优先审查和突破性疗法认定，这些请求中包含的数据表明veligrotug（i）在慢性TED中对复视有一致且显著的改善和缓解作用，以及（ii）眼球突出反应的快速起效 -

WALTHAM, Mass.--(BUSINESS WIRE)--

马萨诸塞州沃尔瑟姆——（商业资讯）

Viridian Therapeutics, Inc. (Nasdaq: VRDN), a biotechnology company focused on discovering, developing, and commercializing potentially best-in-class medicines for serious and rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has accepted the Biologics License Application (BLA) for veligrotug for the treatment of thyroid eye disease (TED).

Viridian Therapeutics, Inc.（纳斯达克股票代码：VRDN）是一家专注于发现、开发和商业化潜在同类最佳药物以治疗严重和罕见疾病的生物技术公司，今天宣布美国食品药品监督管理局 (FDA) 已接受 veligrotug 的生物制品许可申请 (BLA)，用于治疗甲状腺眼病 (TED)。

The application has been granted Priority Review, with a Prescription Drug User Fee Act (PDUFA) target action date of June 30, 2026..

该申请已获得优先审查，处方药用户费用法案（PDUFA）的目标行动日期为2026年6月30日。

Priority Review shortens the BLA target review timeline to six months from ten months after the FDA accepts the BLA. This designation is granted to applications for drugs that, if approved, would be a significant improvement in the safety or effectiveness of treating a serious condition. Priority Review is the second FDA designation granted for veligrotug in 2025.

优先审查将生物制品许可申请（BLA）的目标审查时间从FDA接受BLA后的十个月缩短至六个月。这一指定授予那些如果获得批准，将在治疗严重疾病的安全性或有效性方面取得重大改进的药物申请。这是veligrotug在2025年获得的第二项FDA指定。

In May 2025, the agency granted veligrotug Breakthrough Therapy Designation. Each of these requests included phase 3 clinical trial data on veligrotug’s (i) consistent and robust improvement and resolution of diplopia in chronic TED, and (ii) rapid onset of proptosis response..

2025年5月，该机构授予veligrotug突破性疗法认定。这些请求中的每一个都包括了关于veligrotug的3期临床试验数据：（i）在慢性TED中持续且显著改善和解决复视问题，以及（ii）眼球突出反应的快速起效。

“We are thrilled that the FDA granted Priority Review for veligrotug, marking another significant milestone for Viridian and the TED community,” said Steve Mahoney, Viridian’s President and CEO. “This designation is a recognition that, if approved, veligrotug would be a significant improvement in the safety or effectiveness of treating a serious condition.

“我们非常高兴FDA授予veligrotug优先审查资格，这对Viridian和TED社区而言是另一个重要的里程碑，”Viridian总裁兼首席执行官Steve Mahoney表示。“这一指定表明，如果获得批准，veligrotug将在治疗严重疾病的的安全性或有效性方面带来重大改进。”

This achievement also reflects the Viridian team’s excellent execution and dedication. We very much appreciate all of the support we’ve received from patients, investigators, and our clinical partners. We look forward to working closely with the FDA as we advance toward our goal of delivering a potentially transformative therapy to people living with thyroid eye disease and establishing Viridian as a leading commercial company in TED.”.

这一成就也反映了Viridian团队出色的执行力和奉献精神。我们非常感谢来自患者、研究人员和我们的临床合作伙伴的所有支持。我们期待在朝着为甲状腺眼病患者提供一种可能具有变革性的疗法的目标迈进的过程中，与FDA密切合作，并将Viridian确立为TED领域的领先商业公司。”

The veligrotug BLA is supported by positive data from two of the largest phase 3 clinical trials conducted in TED to date. In THRIVE and THRIVE‑2, conducted in active and chronic TED patients, respectively, veligrotug met the primary and all secondary endpoints of each study as well as demonstrated a rapid onset of clinical benefit.

维利罗单抗BLA得到了迄今为止在甲状腺眼病（TED）中进行的两项最大规模的3期临床试验的积极数据支持。在分别针对活动期和慢性TED患者开展的THRIVE和THRIVE-2试验中，维利罗单抗达到了每项研究的主要终点和所有次要终点，并显示出临床获益的快速起效。

For the first time in a phase 3 clinical trial in chronic TED, veligrotug demonstrated statistically significant diplopia response and diplopia resolution. Veligrotug, if approved, would provide patients with an attractive treatment option with a five-infusion treatment course enabling patients to complete treatment in 12 weeks.

在慢性TED的III期临床试验中，veligrotug首次展示了具有统计学意义的复视反应和复视缓解。如果获得批准，veligrotug将为患者提供一个有吸引力的治疗选择，通过五次输注疗程，患者可在12周内完成治疗。

Veligrotug was generally well-tolerated in its phase 3 clinical trials..

维利格罗图在三期临床试验中通常具有良好的耐受性。

Preparations are well underway for veligrotug’s anticipated commercial launch. The company also plans to submit a Marketing Authorization Application to the European Medicines Agency in the first quarter of 2026.

Veligrotug的预期商业发布准备工作正在顺利进行中。该公司还计划在2026年第一季度向欧洲药品管理局提交上市许可申请。

About Veligrotug

关于Veligrotug

Veligrotug is an intravenously delivered, anti-insulin-like growth factor-1 receptor (IGF-1R) antibody in phase 3 development for thyroid eye disease, with the potential to be the IV treatment-of-choice for active and chronic TED patients. Based on clinical data to date, veligrotug has demonstrated robust clinical activity and was generally well-tolerated..

Veligrotug 是一种静脉注射的抗胰岛素样生长因子-1受体（IGF-1R）抗体，目前处于治疗甲状腺眼病的第三阶段开发中，有潜力成为活动性和慢性甲状腺眼病（TED）患者的首选静脉注射治疗方案。根据迄今为止的临床数据，Veligrotug 已展现出强大的临床活性，并且总体耐受性良好。

Both pivotal phase 3 clinical trials, THRIVE and THRIVE-2, reported positive topline data, meeting the primary and all secondary endpoints of each study. In these studies, veligrotug demonstrated a rapid onset of clinical benefit and statistically significant, clinically meaningful effects on multiple diplopia endpoints in both clinical trials, including the first demonstration of diplopia response and resolution in a global chronic TED phase 3 study.

两项关键的三期临床试验，THRIVE 和 THRIVE-2，均报告了积极的顶线数据，达到了各自研究的主要终点和所有次要终点。在这些研究中，veligrotug 展现了快速的临床效益，并在两项临床试验中的多个复视终点上显示出统计学显著且临床意义重大的效果，包括在全球慢性TED三期研究中首次证明了复视反应和缓解。

Following these results, veligrotug was granted Breakthrough Therapy Designation in May 2025. The FDA also granted the veligrotug BLA a Priority Review with a PDUFA target action date of June 30, 2026. Both Breakthrough Therapy and Priority Review Designations were supported by phase 3 clinical trial data on veligrotug’s consistent and robust improvement and resolution of diplopia in chronic TED, and rapid onset of proptosis response..

根据这些结果，veligrotug 于2025年5月获得了突破性疗法认定。FDA还对veligrotug的生物制品许可申请（BLA）给予了优先审查，并将《处方药使用者费用法案》（PDUFA）的目标行动日期定为2026年6月30日。突破性疗法和优先审查认定均得到了veligrotug在慢性甲状腺眼病（TED）中持续且显著改善和解决复视问题、以及眼球突出反应快速起效的第3期临床试验数据的支持。

About Viridian Therapeutics

关于Viridian Therapeutics

Viridian is a biopharmaceutical company focused on discovering, developing, and commercializing potential best-in-class medicines for patients with serious and rare diseases. Viridian’s expertise in antibody discovery and protein engineering enables the development of differentiated therapeutic candidates for previously validated drug targets in commercially established disease areas..

Viridian是一家生物制药公司，专注于为患有严重和罕见疾病的患者发现、开发和商业化潜在的同类最佳药物。Viridian在抗体发现和蛋白质工程方面的专长使得其能够在商业上已确立的疾病领域中，针对先前已验证的药物靶点开发出具有差异化的治疗候选药物。

Viridian is advancing multiple candidates in the clinic for the treatment of patients with thyroid eye disease (TED). The company conducted a pivotal program for veligrotug (VRDN-001), including two global phase 3 clinical trials (THRIVE and THRIVE-2), to evaluate its efficacy and safety in patients with active and chronic TED.

Viridian公司正在推进多个治疗甲状腺眼病（TED）患者的临床候选药物。该公司开展了针对veligrotug（VRDN-001）的关键项目，包括两项全球III期临床试验（THRIVE和THRIVE-2），以评估其在活动性和慢性TED患者中的有效性和安全性。

Both THRIVE and THRIVE-2 reported positive topline data, meeting the primary and all secondary endpoints of each study. Viridian is also advancing VRDN-003 as a potential best-in-class subcutaneous therapy for the treatment of TED, including two ongoing global phase 3 pivotal clinical trials, REVEAL-1 and REVEAL-2, to evaluate the efficacy and safety of VRDN-003 in patients with active and chronic TED..

THRIVE 和 THRIVE-2 均报告了积极的顶线数据，达到了各自研究的主要终点和所有次要终点。Viridian 还在推进 VRDN-003 作为一种潜在的同类最佳皮下治疗药物，用于治疗甲状腺眼病（TED），包括两项正在进行的全球三期关键临床试验 REVEAL-1 和 REVEAL-2，以评估 VRDN-003 在活动性和慢性 TED 患者中的疗效和安全性。

In addition to its TED portfolio, Viridian is advancing a novel portfolio of neonatal Fc receptor (FcRn) inhibitors, including VRDN-006 and VRDN-008, which have the potential to be developed in multiple autoimmune diseases.

除了其TED产品组合外，Viridian还在推进一种新型的新生儿Fc受体（FcRn）抑制剂组合，包括VRDN-006和VRDN-008，这些药物有潜力在多种自身免疫疾病中开发。

Viridian is based in Waltham, Massachusetts. For more information, please visit

Viridian 总部位于马萨诸塞州沃尔瑟姆。欲了解更多信息，请访问

www.viridiantherapeutics.com

www.viridiantherapeutics.com

. Follow Viridian on

关注Viridian

LinkedIn

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Forward Looking Statements

前瞻性声明

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. These statements may be identified by the use of words such as, but not limited to, “anticipate,” “believe,” “become,” “continue,” “could,” “design,” “estimate,” “expect,” “intend,” “may,” “might,” “on track,” “plan,” “potential,” “predict,” “project,” “should,” “target,” “will,” or “would” or other similar terms or expressions that concern our expectations, plans and intentions.

本新闻稿包含1995年《私人证券诉讼改革法案》意义上的前瞻性陈述。这些陈述可以通过使用诸如“预期”、“相信”、“成为”、“继续”、“可能”、“设计”、“估计”、“期望”、“打算”、“或许”、“可能”、“按计划”、“计划”、“潜力”、“预测”、“预计”、“应该”、“目标”、“将”或“会”等词语或其他类似的术语或表达来识别，这些词语或表达涉及我们的期望、计划和意图。

Forward-looking statements are neither historical facts nor assurances of future performance. Instead, they are based on our current beliefs, expectations, and assumptions. Forward-looking statements include, without limitation, statements regarding: preclinical development, clinical development, and anticipated commercialization of Viridian’s product candidates veligrotug, VRDN-003, VRDN-006, and VRDN-008; Viridian’s expectations regarding regulatory interactions and anticipated timing of regulatory submissions, including a Marketing Authorization Application to the European Medicines Agency, and review timelines; veligrotug’s potential to be an attractive treatment option for patients and the IV treatment-of-choice for active and chronic TED; veligrotug’s potential to be a transformative therapy for people living with TED; Viridian’s product candidates potentially being best-in-class; Viridian’s expectations regarding the potential commercialization of veligrotug, if approved and potential to become a leading commercial company in TED..

前瞻性声明既不是历史事实，也不是未来表现的保证。相反，它们是基于我们当前的信念、期望和假设。前瞻性声明包括但不限于以下内容的声明：Viridian的产品候选药物veligrotug、VRDN-003、VRDN-006和VRDN-008的临床前开发、临床开发及预期商业化；Viridian对监管互动的期望以及预期的监管提交时间，包括向欧洲药品管理局提交上市许可申请及审查时间表；veligrotug有望成为患者有吸引力的治疗选择以及活动性和慢性TED的首选静脉注射治疗方案；veligrotug有潜力成为改变TED患者生活的疗法；Viridian的产品候选药物可能成为同类最佳；Viridian对veligrotug潜在商业化的期望（如果获批）及其有望成为TED领域的领先商业公司的潜力。

New risks and uncertainties may emerge from time to time, and it is not possible to predict all risks and uncertainties. No representations or warranties (expressed or implied) are made about the accuracy of any such forward-looking statements. Such forward-looking statements are subject to a number of material risks and uncertainties including but not limited to: potential utility, efficacy, potency, safety, clinical benefits, clinical response, and convenience of Viridian’s product candidates; that results or data from completed or ongoing clinical trials may not be representative of the results of ongoing or future clinical trials; that preliminary data may not be representative of final data; the timing, progress and plans for our ongoing or future research, preclinical, and clinical development programs; changes to trial protocols for ongoing or new clinical trials; expectations and changes regarding the timing for regulatory filings; regulatory interactions; expectations and changes regarding the timing for enrollment and data; uncertainty and potential delays related to clinical drug development; the duration and impact of regulatory delays in our clinical programs; the timing of and our ability to obtain and maintain regulatory approvals for our therapeutic candidates; manufacturing risks; competition from other therapies or products; estimates of market size; other matters that could affect the sufficiency of existing cash, cash equivalents, and short-term investments to fund operations; our financial position and projected cash runway; our future operating results and financial performance; Viridian’s intellectual property position; the timing of preclinical and clinical trial activities and reporting results from same; that our product candidates may not be c.

新的风险和不确定性可能会不时出现，且无法预测所有风险和不确定性。对于任何此类前瞻性陈述的准确性，不作任何明示或暗示的声明或保证。此类前瞻性陈述受多种重大风险和不确定性的影响，包括但不限于：Viridian候选产品的潜在效用、疗效、效力、安全性、临床益处、临床反应和便利性；已完成或正在进行的临床试验的结果或数据可能无法代表正在进行或未来临床试验的结果；初步数据可能无法代表最终数据；我们正在进行或未来的研究、临床前和临床开发计划的时间、进展和规划；对正在进行或新临床试验的试验方案的更改；对监管文件时间的预期和变化；与监管机构的互动；对入组和数据时间的预期和变化；与临床药物开发相关的不确定性和潜在延迟；我们临床项目中监管延迟的持续时间和影响；我们获得和维持治疗候选产品监管批准的时间和能力；生产风险；来自其他疗法或产品的竞争；市场规模的估计；可能影响现有现金、现金等价物和短期投资是否足以支持运营的其他事项；我们的财务状况和预计现金跑道；我们未来的运营结果和财务表现；Viridian的知识产权地位；临床前和临床试验活动的时间以及相关结果报告；我们的候选产品可能无法...

Investors

投资者

Greg Rossino

格雷格·罗西诺

grossino@viridiantherapeutics.com

格罗西诺@维尔迪安治疗学.com

Media

媒体

Lisa Lopez

丽莎·洛佩兹

llopez@viridiantherapeutics.com

洛佩兹@维里迪安制药公司. com

Source: Viridian Therapeutics, Inc.

来源：Viridian Therapeutics, Inc.