CAMBRIDGE, Mass.--(BUSINESS WIRE)--Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced U.S. Food and Drug Administration (FDA) approval of an expansion to the labeled indication for ELEVIDYS (delandistrogene moxeparvovec-rokl) to include individuals with Duchenne muscular dystrophy (DMD) with a confirmed mutation in the DMD gene who are at least 4 years of age.

马萨诸塞州剑桥市。-（商业新闻短讯）--Sarepta Therapeutics，Inc.（纳斯达克：SRPT）是罕见疾病精确遗传医学的领导者，今天宣布美国食品和药物管理局（FDA）批准扩大ELEVIDYS（delandistrogene moxeparvovec rokl）的标记适应症，以包括至少4岁的Duchenne肌肉萎缩症（DMD）患者，该患者的DMD基因突变已确认。

Confirming the functional benefits, the FDA granted traditional approval for ambulatory patients. The FDA granted accelerated approval for non-ambulatory patients. Continued approval for non-ambulatory Duchenne patients may be contingent upon verification of clinical benefit in a confirmatory trial.

FDA确认了功能益处，批准了门诊患者的传统批准。FDA批准了非门诊患者的加速批准。继续批准非门诊Duchenne患者可能取决于验证性试验中临床获益的验证。

ELEVIDYS is contraindicated in patients with any deletion in exon 8 and/or exon 9 in the DMD gene..

对于DMD基因第8外显子和/或第9外显子缺失的患者，禁止使用ELEVIDYS。。

“Representing many years of dedicated research, development, investment and creative energy, the expansion of the ELEVIDYS label to treat Duchenne patients aged 4 and above, regardless of ambulatory status, is a defining moment for the Duchenne community. Today also stands as a watershed occasion for the promise of gene therapy and a win for science,” said Doug Ingram, president and chief executive officer, Sarepta.

Sarepta总裁兼首席执行官道格·英格拉姆（DougIngram）表示：“ELEVIDYS标签的扩展代表了多年来专注于研究、开发、投资和创新的活力，可以治疗4岁及以上的杜兴病患者，无论其门诊状况如何，这对杜兴社区来说都是一个决定性的时刻。今天也是基因治疗承诺和科学胜利的分水岭。”。

“At this pivotal moment, I want to give warm thanks to Drs. Jerry Mendell and Louise Rodino-Klapac for their dogged, 20-year pursuit of a gene therapy to treat this ruthless and life-robbing disease, to the FDA for following the scientific evidence to speed delivery of a therapy for a life-threatening rare disease to waiting patients, and to the many clinical investigators and courageous Duchenne families who have participated in the multiple studies that led to this important day.”.

“在这个关键时刻，我要热烈感谢Jerry Mendell博士和Louise Rodino Klapac博士，感谢他们20年来坚持不懈地寻求基因疗法来治疗这种残忍和夺命的疾病，感谢FDA遵循科学证据，加速向等待治疗的患者提供一种威胁生命的罕见疾病的治疗，感谢许多临床研究人员和勇敢的Duchenne家庭，他们参与了导致这一重要日子的多项研究。”。

“Today’s expansion of the ELEVIDYS label represents the culmination of my 50-year pursuit of a treatment for Duchenne patients and, along with my colleague Dr. Louise Rodino-Klapac, a nearly 20-year effort to optimize and develop a gene therapy that could be safely and effectively delivered to muscle,” said Jerry Mendell, M.D., co-inventor of ELEVIDYS and senior advisor, Medical Affairs, Sarepta.

“今天ELEVIDYS标签的扩展代表了我50年来对Duchenne患者治疗的追求的高潮，并且与我的同事Louise Rodino Klapac博士一起，经过近20年的努力，优化和开发了一种可以安全有效地传递给肌肉的基因疗法，”ELEVIDYS的共同发明人兼Sarepta医学事务高级顾问Jerry Mendell医学博士说。

“The initial approval of ELEVIDYS was a significant milestone, and the expanded indication means clinicians now have a treatment option for the great majority of boys and young men living with Duchenne. This expansion speaks to the success of the science, the evidence and the improvements in the trajectory of the disease we have seen to date across studies.”.

“ELEVIDYS的初步批准是一个重要的里程碑，适应症的扩大意味着临床医生现在可以为大多数患有杜兴氏病的男孩和年轻男性提供治疗选择。这种扩大说明了科学的成功，证据以及我们迄今为止在各研究中看到的疾病轨迹的改善。”。

Consistent with the accelerated approval pathway, Sarepta has committed to conduct and submit the results of a randomized, controlled trial to verify and confirm the clinical benefit of ELEVIDYS in patients with Duchenne muscular dystrophy who are non-ambulatory. ENVISION (Study SRP-9001-303), a global, randomized, double-blind, placebo-controlled Phase 3 study of ELEVIDYS in non-ambulatory and older ambulatory individuals with Duchenne, is underway and intended to serve as this postmarketing requirement..

与加速批准途径一致，Sarepta承诺进行并提交一项随机对照试验的结果，以验证和确认ELEVIDYS对非门诊杜兴氏肌营养不良症患者的临床益处。ENVISION（研究SRP-9001-303）是一项全球性的，随机的，双盲的，安慰剂对照的3期研究，针对非门诊和老年门诊杜兴氏病患者的ELEVIDYS，正在进行中，旨在作为上市后的要求。。

As part of a collaboration agreement signed in 2019, Sarepta is working with Roche to transform the future for the Duchenne community, enabling those living with the disease to maintain and protect their muscle function. Sarepta is responsible for regulatory approval and commercialization of ELEVIDYS in the U.S., as well as manufacturing.

作为2019年签署的合作协议的一部分，萨雷塔正在与罗氏合作，改变杜兴社区的未来，使患有这种疾病的人能够维持和保护他们的肌肉功能。Sarepta负责ELEVIDYS在美国的监管批准和商业化以及制造。

Roche is responsible for regulatory approvals and bringing ELEVIDYS to patients across the rest of the world..

罗氏负责监管部门的批准，并将ELEVIDYS带给世界各地的患者。。

Patients and physicians can access more information about ELEVIDYS at www.SareptAssist.com or by calling 1-888-727-3782.

患者和医生可以在www.SareptAssist.com或致电1-888-727-3782获得有关ELEVIDYS的更多信息。

Conference call details

电话会议详细信息

At 8:30 a.m. ET on June 21, 2024, Sarepta will host a conference call and webcast to discuss this update.

美国东部时间2024年6月21日上午8:30，萨雷塔将主持电话会议和网络广播，讨论此次更新。

The event will be webcast live under the investor relations section of Sarepta’s website at https://investorrelations.sarepta.com/events-presentations and following the event a replay will be archived there for one year. Interested parties participating by phone will need to register using this online form.

该活动将在Sarepta网站的投资者关系部分进行在线直播https://investorrelations.sarepta.com/events-presentations活动结束后，重播将在那里存档一年。通过电话参与的相关方需要使用此在线表格进行注册。

After registering for dial-in details, all phone participants will receive an auto-generated e-mail containing a link to the dial-in number along with a personal PIN number to use to access the event by phone..

注册拨入详细信息后，所有电话参与者将收到一封自动生成的电子邮件，其中包含拨入号码的链接以及用于通过电话访问活动的个人PIN码。。

About ELEVIDYS (delandistrogene moxeparvovec-rokl)

关于ELEVIDYS（delandistrogene moxeparvovec rokl）

ELEVIDYS (delandistrogene moxeparvovec-rokl) is a single-dose, adeno-associated virus (AAV)-based gene transfer therapy for intravenous infusion designed to address the underlying genetic cause of Duchenne muscular dystrophy – mutations or changes in the DMD gene that result in the lack of dystrophin protein – through the delivery of a transgene that codes for the targeted production of ELEVIDYS micro-dystrophin in skeletal muscle..

ELEVIDYS（delandistrogene moxeparvovec rokl）是一种基于腺相关病毒（AAV）的单剂量基因转移疗法，用于静脉输注，旨在解决杜兴氏肌营养不良症的潜在遗传原因-DMD基因突变或变化导致肌营养不良蛋白缺乏-通过传递编码骨骼肌中靶向产生ELEVIDYS微肌营养不良蛋白的转基因。。

ELEVIDYS is indicated for the treatment of Duchenne muscular dystrophy (DMD) in individuals at least 4 years of age.

ELEVIDYS适用于治疗至少4岁的杜氏肌营养不良症（DMD）。

For patients who are ambulatory and have a confirmed mutation in the DMD gene.

对于非卧床且DMD基因突变已确认的患者。

For patients who are non-ambulatory and have a confirmed mutation in the DMD gene.

对于非门诊且DMD基因突变已确认的患者。

The DMD indication in non-ambulatory patients is approved under accelerated approval based on expression of ELEVIDYS micro-dystrophin (noted hereafter as “micro-dystrophin”) in skeletal muscle. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial..

基于ELEVIDYS微肌营养不良蛋白（以下称为“微肌营养不良蛋白”）在骨骼肌中的表达，非门诊患者的DMD适应症在加速批准下获得批准。是否继续批准该适应症可能取决于验证性试验中临床益处的验证和描述。。

IMPORTANT SAFETY INFORMATION

重要安全信息

CONTRAINDICATION:

禁忌症：

ELEVIDYS is contraindicated in patients with any deletion in exon 8 and/or exon 9 in the DMD gene.

ELEVIDYS在DMD基因第8外显子和/或第9外显子缺失的患者中禁用。

WARNINGS AND PRECAUTIONS:

警告和注意事项：

Infusion-related Reactions:

输液相关反应：

Infusion-related reactions, including hypersensitivity reactions and anaphylaxis, have occurred during or up to several hours following ELEVIDYS administration. Closely monitor patients during administration and for at least 3 hours after the end of infusion. If symptoms of infusion-related reactions occur, slow, or stop the infusion and give appropriate treatment.

。在给药期间和输注结束后至少3小时密切监测患者。如果出现输液相关反应的症状，请减慢或停止输液并给予适当的治疗。

Once symptoms resolve, the infusion may be restarted at a lower rate..

一旦症状消失，输液可能会以较低的速度重新开始。。

ELEVIDYS should be administered in a setting where treatment for infusion-related reactions is immediately available.

ELEVIDYS应该在可以立即治疗输液相关反应的环境中使用。

Discontinue infusion for anaphylaxis.

停止输注过敏反应。

Acute Serious Liver Injury:

急性严重肝损伤：

Acute serious liver injury has been observed with ELEVIDYS, and administration may result in elevations of liver enzymes (such as GGT, GLDH, ALT, AST) or total bilirubin, typically seen within 8 weeks.

ELEVIDYS已观察到急性严重肝损伤，给药可能导致肝酶（如GGT，GLDH，ALT，AST）或总胆红素升高，通常在8周内出现。

Patients with preexisting liver impairment, chronic hepatic condition, or acute liver disease (e.g., acute hepatic viral infection) may be at higher risk of acute serious liver injury. Postpone ELEVIDYS administration in patients with acute liver disease until resolved or controlled.

先前存在肝功能损害，慢性肝病或急性肝病（例如急性肝病毒感染）的患者可能有更高的急性严重肝损伤风险。推迟ELEVIDYS在急性肝病患者中的给药，直到解决或控制。

Prior to ELEVIDYS administration, perform liver enzyme test and monitor liver function (clinical exam, GGT, and total bilirubin) weekly for the first 3 months following ELEVIDYS infusion. Continue monitoring if clinically indicated, until results are unremarkable (normal clinical exam, GGT, and total bilirubin levels return to near baseline levels)..

在ELEVIDYS给药之前，在ELEVIDYS输注后的前3个月内，每周进行肝酶测试并监测肝功能（临床检查，GGT和总胆红素）。如果临床指示，继续监测，直到结果不明显（正常临床检查，GGT和总胆红素水平恢复到接近基线水平）。。

Systemic corticosteroid treatment is recommended for patients before and after ELEVIDYS infusion. Adjust corticosteroid regimen when indicated. If acute serious liver injury is suspected, consultation with a specialist is recommended.

建议在ELEVIDYS输注前后对患者进行全身皮质类固醇治疗。根据需要调整皮质类固醇治疗方案。如果怀疑急性严重肝损伤，建议咨询专家。

Immune-mediated Myositis:

In clinical trials, immune-mediated myositis has been observed approximately 1 month following ELEVIDYS infusion in patients with deletion mutations involving exon 8 and/or exon 9 in the DMD gene. Symptoms of severe muscle weakness, including dysphagia, dyspnea, and hypophonia, were observed.

在临床试验中，在ELEVIDYS输注后约1个月，在DMD基因第8外显子和/或第9外显子缺失突变的患者中观察到免疫介导的肌炎。观察到严重肌肉无力的症状，包括吞咽困难，呼吸困难和声音不足。

Limited data are available for ELEVIDYS treatment in patients with mutations in the DMD gene in exons 1 to 17 and/or exons 59 to 71. Patients with deletions in these regions may be at risk for a severe immune-mediated myositis reaction.

对于外显子1至17和/或外显子59至71中DMD基因突变的患者，ELEVIDYS治疗的数据有限。这些区域缺失的患者可能有发生严重免疫介导的肌炎反应的风险。

Advise patients to contact a physician immediately if they experience any unexplained increased muscle pain, tenderness, or weakness, including dysphagia, dyspnea, or hypophonia, as these may be symptoms of myositis. Consider additional immunomodulatory treatment (immunosuppressants [e.g., calcineurin-inhibitor] in addition to corticosteroids) based on patient’s clinical presentation and medical history if these symptoms occur..

如果患者出现任何无法解释的肌肉疼痛，压痛或无力增加，包括吞咽困难，呼吸困难或声音不足，建议患者立即联系医生，因为这些可能是肌炎的症状。如果出现这些症状，请根据患者的临床表现和病史考虑额外的免疫调节治疗（免疫抑制剂[例如钙调神经磷酸酶抑制剂]以及皮质类固醇）。。

Myocarditis:

心肌炎：

Acute serious myocarditis and troponin-I elevations have been observed following ELEVIDYS infusion in clinical trials.

在临床试验中，ELEVIDYS输注后观察到急性严重心肌炎和肌钙蛋白-I升高。

If a patient experiences myocarditis, those with pre-existing left ventricle ejection fraction (LVEF) impairment may be at higher risk of adverse outcomes. Monitor troponin-I before ELEVIDYS infusion and weekly for the first month following infusion and continue monitoring if clinically indicated. More frequent monitoring may be warranted in the presence of cardiac symptoms, such as chest pain or shortness of breath..

如果患者患有心肌炎，则先前存在左心室射血分数（LVEF）损伤的患者可能有更高的不良后果风险。在ELEVIDYS输注前和输注后第一个月每周监测肌钙蛋白-I，并在临床指示的情况下继续监测。如果存在心脏症状，例如胸痛或呼吸急促，可能需要更频繁的监测。。

Advise patients to contact a physician immediately if they experience cardiac symptoms.

如果患者出现心脏症状，建议他们立即联系医生。

Preexisting Immunity against AAVrh74:

In AAV-vector based gene therapies, preexisting anti-AAV antibodies may impede transgene expression at desired therapeutic levels. Following treatment with ELEVIDYS, all patients developed anti-AAVrh74 antibodies.

在基于AAV载体的基因疗法中，先前存在的抗AAV抗体可能会在所需的治疗水平上阻碍转基因表达。用ELEVIDYS治疗后，所有患者均产生了抗AAVrh74抗体。

Perform baseline testing for presence of anti-AAVrh74 total binding antibodies prior to ELEVIDYS administration.

在ELEVIDYS给药之前，对抗AAVrh74总结合抗体的存在进行基线测试。

ELEVIDYS administration is not recommended in patients with elevated anti-AAVrh74 total binding antibody titers greater than or equal to 1:400.

对于抗AAVrh74总结合抗体滴度大于或等于1:400的患者，不建议使用ELEVIDYS。

Adverse Reactions:

不良反应：

The most common adverse reactions (incidence ≥5%) reported in clinical studies were vomiting, nausea, liver injury, pyrexia, and thrombocytopenia.

临床研究中报告的最常见不良反应（发生率≥5%）是呕吐，恶心，肝损伤，发热和血小板减少症。

Report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch or call 1-800-FDA-1088. You may also report side effects to Sarepta Therapeutics at 1-888-SAREPTA (1-888-727-3782).

向FDA报告处方药的负面副作用。访问www.fda.gov/medwatch或致电1-800-fda-1088。您也可以在1-888-Sarepta（1-888-727-3782）向Sarepta Therapeutics报告副作用。

For further information, please see the full Prescribing Information.

有关更多信息，请参阅完整的处方信息。

About Sarepta Therapeutics

Sarepta is on an urgent mission: engineer precision genetic medicine for rare diseases that devastate lives and cut futures short. We hold leadership positions in Duchenne muscular dystrophy (DMD) and limb-girdle muscular dystrophies (LGMDs), and we currently have more than 40 programs in various stages of development.

萨雷塔正在执行一项紧急任务：为罕见疾病设计精确的基因医学，这些疾病会破坏生命并缩短未来。我们在杜兴氏肌营养不良症（DMD）和肢带型肌营养不良症（LGMD）方面担任领导职务，目前我们有40多个项目处于不同的发展阶段。

Our vast pipeline is driven by our multi-platform Precision Genetic Medicine Engine in gene therapy, RNA and gene editing. For more information, please visit www.sarepta.com or follow us on LinkedIn, X (formerly Twitter), Instagram and Facebook..

我们在基因治疗，RNA和基因编辑方面的多平台精密遗传医学引擎推动了我们庞大的管道。有关更多信息，请访问www.sarepta.com或在LinkedIn、X（以前的Twitter）、Instagram和Facebook上关注我们。。

Internet Posting of Information

网上发布信息

We routinely post information that may be important to investors in the 'For Investors' section of our website at www.sarepta.com. We encourage investors and potential investors to consult our website regularly for important information about us.

。

Forward-Looking Statements

前瞻性声明

This press release contains “forward-looking statements.” Any statements that are not statements of historical fact may be deemed to be forward-looking statements. Words such as “believe,” “anticipate,” “plan,” “expect,” “will,” “may,” “intend,” “prepare,” “look,” “potential,” “possible” and similar expressions are intended to identify forward-looking statements.

本新闻稿包含“前瞻性声明”。任何不是历史事实声明的声明都可能被视为前瞻性声明。诸如“相信”、“预期”、“计划”、“预期”、“将”、“可能”、“打算”、“准备”、“展望”、“潜力”、“可能”等词语以及类似的表达方式旨在识别前瞻性陈述。

These forward-looking statements include, without limitation, statements relating to our future operations, business plans, priorities, research and development programs; the potential benefits and risks of ELEVIDYS; and continued approval for non-ambulatory Duchenne patients may be contingent upon verification of clinical benefit in a confirmatory trial..

这些前瞻性声明包括但不限于与我们未来运营、商业计划、优先事项、研究和开发计划有关的声明；ELEVIDYS的潜在利益和风险；对于非门诊Duchenne患者的持续批准可能取决于在验证性试验中验证临床益处。。

Actual results could materially differ from those stated or implied by these forward-looking statements as a result of such risks and uncertainties. Known risk factors include the following: continued approval for non-ambulatory patients may be contingent upon verification of clinical benefit; we may not be able to comply with all FDA requests in a timely manner or at all; the possible impact of regulations and regulatory decisions by the FDA and other regulatory agencies on our business, as well as the development of our product candidates and our financial and contractual obligations; our dependence on certain manufacturers to produce our products and product candidates, including any inability on our part to accurately anticipate product demand and to secure in a timely manner manufacturing capacity to meet product demand, may impair the availability of product to successfully support various programs; our data may not be sufficient for obtaining regulatory approval; the results of future research may not be consistent with past positive results or may fail to meet regulatory approval requirements for the safety and efficacy of product candidates; the commencement and completion of our clinical trials and announcement of results may be delayed or prevented for a number of reasons, including, among others, denial by the regulatory agencies of permission to proceed with our clinical trials, or placement of a clinical trial on hold, challenges in identifying, recruiting, enrolling and retaining patients to participate in clinical trials and inadequate quantity or quality of supplies of a product candidate or other materials necessary to conduct clinical trials; different methodologies, assumptions and applications we use to assess particular safety or efficacy parameter.

由于此类风险和不确定性，实际结果可能与这些前瞻性声明所述或暗示的结果存在重大差异。已知的风险因素包括：继续批准非门诊患者可能取决于临床获益的验证；我们可能无法及时或根本无法遵守FDA的所有要求；FDA和其他监管机构的法规和监管决定可能对我们的业务产生的影响，以及我们候选产品的开发以及我们的财务和合同义务；我们依赖某些制造商生产我们的产品和候选产品，包括我们无法准确预测产品需求并及时确保制造能力以满足产品需求，这可能会损害产品的可用性，从而无法成功支持各种计划；我们的数据可能不足以获得监管部门的批准；未来研究的结果可能与过去的积极结果不一致，或者可能不符合候选产品安全性和有效性的监管批准要求；我们的临床试验的开始和完成以及结果的宣布可能会因多种原因而延迟或阻止，其中包括监管机构拒绝批准继续进行我们的临床试验，或暂停临床试验，识别，招募，招募和保留患者参加临床试验的挑战，以及候选产品或进行临床试验所需的其他材料的供应数量或质量不足；我们用于评估特定安全性或有效性参数的不同方法，假设和应用。

Any of the foregoing risks could materially and adversely affect the Company’s business, results of operations and the trading price of Sarepta’s common stock. For a detailed description of risks and uncertainties Sarepta faces, you are encouraged to review the SEC filings made by Sarepta. We caution investors not to place considerable reliance on the forward-looking statements contained in this press release.

上述任何风险都可能对公司的业务、经营业绩和萨雷塔普通股的交易价格产生重大不利影响。有关Sarepta面临的风险和不确定性的详细描述，鼓励您查看Sarepta向SEC提交的文件。我们提醒投资者不要过分依赖本新闻稿中的前瞻性声明。

Sarepta does not undertake any obligation to publicly update its forward-looking statements based on events or circumstances after the date hereof, except as required by law..

除法律要求外，Sarepta没有义务根据本协议日期后的事件或情况公开更新其前瞻性声明。。