Vyondys 53（Golodirsen）注射液

公司名称：Sarepta Therapeutics

批准日期：2019年12月12日

治疗：杜兴氏肌营养不良症

Vyondys 53（golodirsen）是一种磷二酰胺吗啉代寡聚体，用于治疗患有确诊易发生外显子53突变的杜兴氏肌营养不良症（DMD）。

马萨诸塞州坎布里奇，2019年12月12日（环球新闻）-罕见疾病精密基因医学的领导者Sarepta Therapeutics，Inc.（纳斯达克股票代码：SRPT）今天宣布，美国食品药品监督管理局（FDA）已批准的Vyondys 53™（Golodirsen）。 Vyondys 53是来自Sarepta的磷酸二酰胺基吗啉代寡聚物（PMO）平台的反义寡核苷酸，适用于治疗已确诊易发生外显子53突变的杜氏肌营养不良症（DMD）。该适应症是基于在用Vyondys 53治疗的患者中观察到的骨骼肌肌营养不良蛋白产生的统计学显着增加，这合理地有可能预测那些适合外显子53的患者的临床获益。与加速批准途径一致，Vyondys 53的继续批准可能取决于该上市后确认试验中的临床获益。

Sarepta的安慰剂对照上市后确认试验，以支持Vyondys 53加速批准-名为ESSENCE-目前正在招募中，预计将于2024年结束。

用Vyondys 53治疗的患者发生了过敏反应，包括皮疹，发热（发烧），瘙痒，荨麻疹，荨麻疹，皮炎和皮肤剥脱。在动物研究中观察到了肾脏毒性。尽管在Vyondys 53的临床研究中未观察到肾毒性，包括某些致命的肾小球肾炎，但在施用某些反义寡核苷酸后已观察到。至少在20％的Vyondys 53治疗患者中发生的最常见不良反应是头痛（41％），发热（41％），跌倒（29％），腹痛（27） ％），鼻咽炎（27％），咳嗽（27％），呕吐（27％）和恶心（20％）。

向Vyondys 53的神经病学产品部（审查部）提交新药申请（NDA）并对其进行审查后，审查部建议批准该新药申请，药品评估办公室1在2006年发布了完整的答复信（CRL）。 2019年8月。此后，Sarepta提出了有关FDA指南中概述的正式争议解决请求。在审查部门的支持下，新药办公室（OND）主任Peter Stein博士迅速评估并解决了CRL中提出的问题。 OND批准了该公司的上诉，而Sarepta将其NDA重新提交给了审核部门，该部门迅速审核并批准了Vyondys 53。

Sarepta总裁兼首席执行官道格·英格拉姆（Doug Ingram）说：“今天对于Sarepta以及更重要的是对DMD社区来说都是具有纪念意义的。” “ Vyondys 53，是我们第二项批准用于DMD的外显子跳跃RNA治疗方法，可以治疗高达8％的DMD社区，代表那些确诊具有外显子53顺应性突变的患者。现在，与EXONDYS51®（eteplirsen）一起，我们为美国约20％患有DMD的患者提供了治疗选择。”

英格拉姆继续说：“在四个月的时间里，我们开始并完成了正式的争端解决程序，最终批准了我们的上诉，重新提交了我们的NDA并获得了批准-这对正在等待治疗的DMD患者是一个巨大的好处。没有比利·邓恩博士和新药办公室领导下的审查处的承诺，没有史无前例的时机，他们迅速听取并批准了我们的呼吁。与DMD社区一起，我们感谢审核部和OND采取客观，基于证据的方式进行此审核，具有公正性以及他们解决和解决CRL并获得批准的紧迫感，批准。”

父母项目肌肉营养不良症（PPMD）创始主席兼首席执行官Pat Furlong说：“通过Vyondys 53的批准，多达8％的Duchenne家庭将有一种疗法来治疗这种破坏性疾病。” “ 25年来，PPMD一直与研究人员，临床医生，行业和Duchenne社区合作，为所有与Duchenne一起生活的人们寻找治疗方法。尽管我们需要确保患者可以使用这些批准的疗法，但今天我们庆祝这一批准，并感谢Sarepta在结束杜兴的斗争中继续发挥领导作用。”

关于Vyondys 53

Vyondys 53是一种反义寡核苷酸，可用于已确诊DMD基因突变且适合外显子53跳过的患者的Duchenne肌营养不良症的治疗。 Vyondys 53使用Sarepta专有的磷酸二氨基吗啉吗啉代寡聚物（PMO）化学和外显子跳跃技术与肌营养不良蛋白前体mRNA的外显子53结合，从而导致具有遗传突变的患者在mRNA处理过程中将该外显子排斥或“跳过”。适合外显子53跳过。外显子跳跃的目的是允许产生内部截短的肌营养不良蛋白。

Vyondys 53被批准进行加速审查，其依据是外显子53跳过的患者骨骼肌中肌营养不良蛋白产量的增加。继续批准可能取决于验证试验中临床获益的验证。

Vyondys 53符合安全性和有效性的完整法定标准，因此不被认为是试验性或实验性的。

Vyondys的重要安全信息53

经Vyondys 53治疗的患者发生了过敏反应，包括皮疹，发热，瘙痒，荨麻疹，皮炎和皮肤剥脱，其中一些患者需要治疗。如果发生超敏反应，请采取适当的医疗措施，并考虑减慢输注速度或中断Vyondys 53治疗。

在接受戈洛迪森的动物中观察到了肾脏毒性。尽管在Vyondys 53的临床研究中未观察到肾毒性，但在服用某些反义寡核苷酸后已观察到肾毒性，包括可能致命的肾小球肾炎。服用Vyondys 53的患者应监测肾功能。由于骨骼肌质量降低对肌酐测量的影响，肌酐可能不是DMD患者肾功能的可靠测量。建议在治疗开始前通过24小时尿液收集来测量肾小球滤过率（GFR）。建议每月通过试纸尿液监测蛋白尿，每三个月监测一次血清半胱氨酸蛋白酶抑制剂C。如果确诊量油尺蛋白尿大于或等于2或血清半胱氨酸蛋白酶抑制剂C升高，则应进行24小时尿液收集以定量蛋白尿并评估GFR。

在至少20％的接受治疗的患者中观察到的不良反应大于安慰剂（Vyondys 53，安慰剂）：头痛（41％，10％），发热（41％，14％），跌落（29％，19％），腹痛（27％，10％），鼻咽炎（27％，14％），咳嗽（27％，19％），呕吐（27％，19％）和恶心（20％，10％）。

以Vyondys 53治疗的患者发生频率高出5％且发生频率高于安慰剂的其他不良反应包括给药部位疼痛，背痛，疼痛，腹泻，头晕，韧带扭伤，挫伤，流感，口咽痛，鼻炎，皮肤擦伤，耳朵感染，季节性过敏，心动过速，导管部位相关反应，便秘和骨折。

有关更多信息，请参见完整的处方信息。

关于Exondys 51

Exondys 51使用Sarepta专有的二氨基磷酸二酰胺吗啉代低聚物（PMO）化学物质和外显子跳跃技术来跳过肌营养不良蛋白基因的外显子51。外显子51被设计为与肌营养不良蛋白前体mRNA的外显子51结合，从而在具有适合于外显子51跳跃的遗传突变的患者的mRNA加工过程中将该外显子排除在外。外显子跳跃的目的是允许产生内部截短的肌营养不良蛋白。

关于Exondys的重要安全信息51

用Exondys 51治疗的患者发生了超敏反应，包括皮疹和荨麻疹，发热，潮红，咳嗽，呼吸困难，支气管痉挛和低血压。如果发生超敏反应，请采取适当的医疗措施并考虑减慢输注速度或中断输注。 Exondys 51治疗。

用Exondys 51 30 mg或50 mg / kg /周/周通过静脉内（IV）输注治疗的DMD患者（N = 8）的不良反应发生率比安慰剂（N = 4）高出至少25％（研究1，24周（Exondys 51，安慰剂）：平衡障碍（38％，0％），呕吐（38％，0％）和接触性皮炎（25％，0％）。最常见的不良反应是平衡障碍和呕吐。由于患者数量少，这些代表的原始频率可能无法反映实际观察到的频率。不建议每周一次使用Exondys 51的50 mg / kg剂量。

在临床研究中，接受Exondys 51≥30 mg / kg /周，长达208周的88例患者中，≥10％的患者发生以下事件，其发生率高于研究1中相同剂量的患者：呕吐，挫伤，挫伤，关节痛，皮疹，导管部位疼痛和上呼吸道感染。

有关更多信息，请参见完整的处方信息。

Vyondys 53（Golodirsen）注射液

公司名称：Sarepta Therapeutics

批准日期：2019年12月12日

治疗：杜兴氏肌营养不良症

Vyondys 53（golodirsen）是一种磷二酰胺吗啉代寡聚体，用于治疗患有确诊易发生外显子53突变的杜兴氏肌营养不良症（DMD）。

马萨诸塞州坎布里奇，2019年12月12日（环球新闻）-罕见疾病精密基因医学的领导者Sarepta Therapeutics，Inc.（纳斯达克股票代码：SRPT）今天宣布，美国食品药品监督管理局（FDA）已批准的Vyondys 53™（Golodirsen）。 Vyondys 53是来自Sarepta的磷酸二酰胺基吗啉代寡聚物（PMO）平台的反义寡核苷酸，适用于治疗已确诊易发生外显子53突变的杜氏肌营养不良症（DMD）。该适应症是基于在用Vyondys 53治疗的患者中观察到的骨骼肌肌营养不良蛋白产生的统计学显着增加，这合理地有可能预测那些适合外显子53的患者的临床获益。与加速批准途径一致，Vyondys 53的继续批准可能取决于该上市后确认试验中的临床获益。

Sarepta的安慰剂对照上市后确认试验，以支持Vyondys 53加速批准-名为ESSENCE-目前正在招募中，预计将于2024年结束。

用Vyondys 53治疗的患者发生了过敏反应，包括皮疹，发热（发烧），瘙痒，荨麻疹，荨麻疹，皮炎和皮肤剥脱。在动物研究中观察到了肾脏毒性。尽管在Vyondys 53的临床研究中未观察到肾毒性，包括某些致命的肾小球肾炎，但在施用某些反义寡核苷酸后已观察到。至少在20％的Vyondys 53治疗患者中发生的最常见不良反应是头痛（41％），发热（41％），跌倒（29％），腹痛（27） ％），鼻咽炎（27％），咳嗽（27％），呕吐（27％）和恶心（20％）。

向Vyondys 53的神经病学产品部（审查部）提交新药申请（NDA）并对其进行审查后，审查部建议批准该新药申请，药品评估办公室1在2006年发布了完整的答复信（CRL）。 2019年8月。此后，Sarepta提出了有关FDA指南中概述的正式争议解决请求。在审查部门的支持下，新药办公室（OND）主任Peter Stein博士迅速评估并解决了CRL中提出的问题。 OND批准了该公司的上诉，而Sarepta将其NDA重新提交给了审核部门，该部门迅速审核并批准了Vyondys 53。

Sarepta总裁兼首席执行官道格·英格拉姆（Doug Ingram）说：“今天对于Sarepta以及更重要的是对DMD社区来说都是具有纪念意义的。” “ Vyondys 53，是我们第二项批准用于DMD的外显子跳跃RNA治疗方法，可以治疗高达8％的DMD社区，代表那些确诊具有外显子53顺应性突变的患者。现在，与EXONDYS51®（eteplirsen）一起，我们为美国约20％患有DMD的患者提供了治疗选择。”

英格拉姆继续说：“在四个月的时间里，我们开始并完成了正式的争端解决程序，最终批准了我们的上诉，重新提交了我们的NDA并获得了批准-这对正在等待治疗的DMD患者是一个巨大的好处。没有比利·邓恩博士和新药办公室领导下的审查处的承诺，没有史无前例的时机，他们迅速听取并批准了我们的呼吁。与DMD社区一起，我们感谢审核部和OND采取客观，基于证据的方式进行此审核，具有公正性以及他们解决和解决CRL并获得批准的紧迫感，批准。”

父母项目肌肉营养不良症（PPMD）创始主席兼首席执行官Pat Furlong说：“通过Vyondys 53的批准，多达8％的Duchenne家庭将有一种疗法来治疗这种破坏性疾病。” “ 25年来，PPMD一直与研究人员，临床医生，行业和Duchenne社区合作，为所有与Duchenne一起生活的人们寻找治疗方法。尽管我们需要确保患者可以使用这些批准的疗法，但今天我们庆祝这一批准，并感谢Sarepta在结束杜兴的斗争中继续发挥领导作用。”

关于Vyondys 53

Vyondys 53是一种反义寡核苷酸，可用于已确诊DMD基因突变且适合外显子53跳过的患者的Duchenne肌营养不良症的治疗。 Vyondys 53使用Sarepta专有的磷酸二氨基吗啉吗啉代寡聚物（PMO）化学和外显子跳跃技术与肌营养不良蛋白前体mRNA的外显子53结合，从而导致具有遗传突变的患者在mRNA处理过程中将该外显子排斥或“跳过”。适合外显子53跳过。外显子跳跃的目的是允许产生内部截短的肌营养不良蛋白。

Vyondys 53被批准进行加速审查，其依据是外显子53跳过的患者骨骼肌中肌营养不良蛋白产量的增加。继续批准可能取决于验证试验中临床获益的验证。

Vyondys 53符合安全性和有效性的完整法定标准，因此不被认为是试验性或实验性的。

Vyondys的重要安全信息53

经Vyondys 53治疗的患者发生了过敏反应，包括皮疹，发热，瘙痒，荨麻疹，皮炎和皮肤剥脱，其中一些患者需要治疗。如果发生超敏反应，请采取适当的医疗措施，并考虑减慢输注速度或中断Vyondys 53治疗。

在接受戈洛迪森的动物中观察到了肾脏毒性。尽管在Vyondys 53的临床研究中未观察到肾毒性，但在服用某些反义寡核苷酸后已观察到肾毒性，包括可能致命的肾小球肾炎。服用Vyondys 53的患者应监测肾功能。由于骨骼肌质量降低对肌酐测量的影响，肌酐可能不是DMD患者肾功能的可靠测量。建议在治疗开始前通过24小时尿液收集来测量肾小球滤过率（GFR）。建议每月通过试纸尿液监测蛋白尿，每三个月监测一次血清半胱氨酸蛋白酶抑制剂C。如果确诊量油尺蛋白尿大于或等于2或血清半胱氨酸蛋白酶抑制剂C升高，则应进行24小时尿液收集以定量蛋白尿并评估GFR。

在至少20％的接受治疗的患者中观察到的不良反应大于安慰剂（Vyondys 53，安慰剂）：头痛（41％，10％），发热（41％，14％），跌落（29％，19％），腹痛（27％，10％），鼻咽炎（27％，14％），咳嗽（27％，19％），呕吐（27％，19％）和恶心（20％，10％）。

以Vyondys 53治疗的患者发生频率高出5％且发生频率高于安慰剂的其他不良反应包括给药部位疼痛，背痛，疼痛，腹泻，头晕，韧带扭伤，挫伤，流感，口咽痛，鼻炎，皮肤擦伤，耳朵感染，季节性过敏，心动过速，导管部位相关反应，便秘和骨折。

有关更多信息，请参见完整的处方信息。

关于Exondys 51

Exondys 51使用Sarepta专有的二氨基磷酸二酰胺吗啉代低聚物（PMO）化学物质和外显子跳跃技术来跳过肌营养不良蛋白基因的外显子51。外显子51被设计为与肌营养不良蛋白前体mRNA的外显子51结合，从而在具有适合于外显子51跳跃的遗传突变的患者的mRNA加工过程中将该外显子排除在外。外显子跳跃的目的是允许产生内部截短的肌营养不良蛋白。

关于Exondys的重要安全信息51

用Exondys 51治疗的患者发生了超敏反应，包括皮疹和荨麻疹，发热，潮红，咳嗽，呼吸困难，支气管痉挛和低血压。如果发生超敏反应，请采取适当的医疗措施并考虑减慢输注速度或中断输注。 Exondys 51治疗。

用Exondys 51 30 mg或50 mg / kg /周/周通过静脉内（IV）输注治疗的DMD患者（N = 8）的不良反应发生率比安慰剂（N = 4）高出至少25％（研究1，24周（Exondys 51，安慰剂）：平衡障碍（38％，0％），呕吐（38％，0％）和接触性皮炎（25％，0％）。最常见的不良反应是平衡障碍和呕吐。由于患者数量少，这些代表的原始频率可能无法反映实际观察到的频率。不建议每周一次使用Exondys 51的50 mg / kg剂量。

在临床研究中，接受Exondys 51≥30 mg / kg /周，长达208周的88例患者中，≥10％的患者发生以下事件，其发生率高于研究1中相同剂量的患者：呕吐，挫伤，挫伤，关节痛，皮疹，导管部位疼痛和上呼吸道感染。

有关更多信息，请参见完整的处方信息。

Company: Sarepta Therapeutics
Date of Approval: December 12, 2019
Treatment for: Duchenne Muscular Dystrophy

Vyondys 53 (golodirsen) is a phosphordiamidate morpholino oligimer for the treatment of Duchenne muscular dystrophy (DMD) in patients with a confirmed mutation amenable to exon 53 skipping.

FDA Approves Vyondys 53 (golodirsen) Injection for the Treatment of Duchenne Muscular Dystrophy (DMD) in Patients Amenable to Skipping Exon 53

CAMBRIDGE, Mass., Dec. 12, 2019 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has approved Vyondys 53™ (golodirsen). Vyondys 53 is an antisense oligonucleotide from Sarepta’s phosphorodiamidate morpholino oligomer (PMO) platform, indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients with a confirmed mutation amenable to exon 53 skipping. This indication is based on a statistically significant increase in dystrophin production in skeletal muscle observed in patients treated with Vyondys 53, which is reasonably likely to predict clinical benefit for those patients who are exon 53 amenable. Consistent with the accelerated approval pathway, the continued approval of Vyondys 53 may be contingent on confirmation of a clinical benefit in this post-marketing confirmatory trial.

Sarepta’s placebo-controlled, post-marketing confirmatory trial to support the Vyondys 53 accelerated approval – titled ESSENCE – is currently enrolling and expected to conclude by 2024.

Hypersensitivity reactions, including rash, pyrexia (fever), pruritis, urticaria (hives), dermatitis, and skin exfoliation have occurred in patients who were treated with Vyondys 53. Renal toxicity was observed in animal studies. Although not observed in the clinical studies with Vyondys 53, renal toxicity, including potentially fatal glomerulonephritis, has been observed after administration of some antisense oligonucleotides. The most common adverse reactions that occurred in at least 20% of Vyondys 53-treated patients and more frequently than in placebo-treated patients were headache (41%), pyrexia (41%), fall (29%), abdominal pain (27%), nasopharyngitis (27%), cough (27%), vomiting (27%), and nausea (20%).

Following a New Drug Application (NDA) submission to and review by the Division of Neurology Products (the Review Division) for Vyondys 53, which the Review Division recommended for approval, the Office of Drug Evaluation 1 issued a complete response letter (CRL) in August of 2019. Thereafter, Sarepta made a formal dispute resolution request as outlined in relevant FDA Guidance. With the support of the Review Division, the matters raised in the CRL were rapidly evaluated and resolved by Dr. Peter Stein, Director of the Office of New Drugs (OND). OND granted the Company’s appeal and Sarepta re-submitted its NDA to the Review Division, which worked expeditiously to review and approve Vyondys 53.

“Today is monumental for Sarepta and, more importantly, for the DMD community,” said Doug Ingram, president and chief executive officer, Sarepta. “Vyondys 53, our second approved exon-skipping RNA therapy for DMD, may treat up to 8% of the DMD community, representing those patients who have a confirmed exon 53 amenable mutation. Along with EXONDYS 51® (eteplirsen), we now offer treatment options for approximately 20% of those with DMD in the U.S.”

Ingram continued, “In the span of four months, we commenced and completed the formal dispute resolution process culminating in the grant of our appeal, resubmitted our NDA and obtained an approval – a great benefit to DMD patients awaiting treatment. This unprecedented timing could not have been achieved without the commitment of the Review Division under the leadership of Dr. Billy Dunn, and the Office of New Drugs, which expeditiously heard and granted our appeal. Along with the DMD community, we owe our gratitude to both the Review Division and the OND for their objective, evidence-based approach to this review, for their fairness, and for the sense of urgency with which they addressed and resolved the CRL and granted this approval.”

“With the approval of Vyondys 53, up to another 8% of Duchenne families will have a therapy to treat this devastating disease,” said Pat Furlong, founding president and chief executive officer, Parent Project Muscular Dystrophy (PPMD). “For 25 years, PPMD has been working with researchers, clinicians, industry, and the Duchenne community to find treatments for all people living with Duchenne. And while we need to ensure that these approved therapies are accessible for patients, today we celebrate this approval and thank Sarepta for their continued leadership in the fight to end Duchenne.”

Vyondys 53 is priced at parity to EXONDYS 51, the price of which has not increased since its launch in 2016. Patients and physicians can access more information at www.SareptAssist.com or by calling 1-888-727-3782.

Vyondys 53 is an antisense oligonucleotide indicated for the treatment of Duchenne muscular dystrophy in patients who have a confirmed mutation of the DMD gene that is amenable to exon 53 skipping. Vyondys 53 uses Sarepta’s proprietary phosphorodiamidate morpholino oligomer (PMO) chemistry and exon-skipping technology to bind to exon 53 of dystrophin pre-mRNA, resulting in exclusion, or “skipping,” of this exon during mRNA processing in patients with genetic mutations that are amenable to exon 53 skipping. Exon skipping is intended to allow for production of an internally truncated dystrophin protein.

Vyondys 53 is approved under accelerated review based on an increase in dystrophin production in skeletal muscle of patients amenable to exon 53 skipping. Continued approval may be contingent upon verification of a clinical benefit in confirmatory trials.

Vyondys 53 has met the full statutory standards for safety and effectiveness and as such is not considered investigational or experimental.

Important Safety Information for Vyondys 53
Hypersensitivity reactions, including rash, pyrexia, pruritus, urticaria, dermatitis, and skin exfoliation have occurred in Vyondys 53-treated patients, some requiring treatment. If a hypersensitivity reaction occurs, institute appropriate medical treatment and consider slowing the infusion or interrupting the Vyondys 53 therapy.

Renal toxicity was observed in animals who received golodirsen. Although renal toxicity was not observed in the clinical studies with Vyondys 53, renal toxicity, including potentially fatal glomerulonephritis, has been observed after administration of some antisense oligonucleotides. Renal function should be monitored in patients taking Vyondys 53. Because of the effect of reduced skeletal muscle mass on creatinine measurements, creatinine may not be a reliable measure of renal function in DMD patients. Measurement of glomerular filtration rate (GFR) by 24-hour urine collection prior to initiation of therapy is recommended. Monthly monitoring for proteinuria by dipstick urinalysis and monitoring of serum cystatin C every three months is recommended. In the case of a confirmed dipstick proteinuria of 2+ or greater or elevated serum cystatin C, a 24-hour urine collection to quantify proteinuria and assess GFR should be performed.

Adverse reactions observed in at least 20% of treated patients and greater than placebo were (Vyondys 53, placebo): headache (41%, 10%), pyrexia (41%, 14%), fall (29%, 19%), abdominal pain (27%, 10%), nasopharyngitis (27%, 14%), cough (27%, 19%), vomiting (27%, 19%), and nausea (20%, 10%).

Other adverse reactions that occurred at a frequency greater than 5% of Vyondys 53-treated patients and at a greater frequency than placebo were administration site pain, back pain, pain, diarrhea, dizziness, ligament sprain, contusion, influenza, oropharyngeal pain, rhinitis, skin abrasion, ear infection, seasonal allergy, tachycardia, catheter site related reaction, constipation, and fracture.

For further information, please see the full Prescribing Information.

Exondys 51 uses Sarepta’s proprietary phosphorodiamidate morpholino oligomer (PMO) chemistry and exon-skipping technology to skip exon 51 of the dystrophin gene. Exondys 51 is designed to bind to exon 51 of dystrophin pre-mRNA, resulting in exclusion of this exon during mRNA processing in patients with genetic mutations that are amenable to exon 51 skipping. Exon skipping is intended to allow for production of an internally truncated dystrophin protein.

Important Safety Information About Exondys 51
Hypersensitivity reactions, including rash and urticaria, pyrexia, flushing, cough, dyspnea, bronchospasm, and hypotension, have occurred in patients who were treated with Exondys 51. If a hypersensitivity reaction occurs, institute appropriate medical treatment and consider slowing the infusion or interrupting the Exondys 51 therapy.

Adverse reactions in DMD patients (N=8) treated with Exondys 51 30 mg or 50 mg/kg/week by intravenous (IV) infusion with an incidence of at least 25% more than placebo (N=4) (Study 1, 24 weeks) were (Exondys 51, placebo): balance disorder (38%, 0%), vomiting (38%, 0%) and contact dermatitis (25%, 0%). The most common adverse reactions were balance disorder and vomiting. Because of the small numbers of patients, these represent crude frequencies that may not reflect the frequencies observed in practice. The 50 mg/kg once weekly dosing regimen of Exondys 51 is not recommended.

In the 88 patients who received ≥30 mg/kg/week of Exondys 51 for up to 208 weeks in clinical studies, the following events were reported in ≥10% of patients and occurred more frequently than on the same dose in Study 1: vomiting, contusion, excoriation, arthralgia, rash, catheter site pain, and upper respiratory tract infection.

For further information, please see the full Prescribing Information.

Sarepta is at the forefront of precision genetic medicine, having built an impressive and competitive position in Duchenne muscular dystrophy (DMD) and more recently in gene therapies for 6 Limb-girdle muscular dystrophy diseases (LGMD), Charcot-Marie-Tooth (CMT), MPS IIIA, Pompe and other CNS-related disorders, totaling over 20 therapies in various stages of development. The Company’s programs and research focus span several therapeutic modalities, including RNA, gene therapy and gene editing. Sarepta is fueled by an audacious but important mission: to profoundly improve and extend the lives of patients with rare genetic-based diseases. For more information, please visit www.sarepta.com.

Forward-Looking Statement
This press release contains "forward-looking statements." Any statements contained in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Words such as "believes," "anticipates," "plans," "expects," "will," "intends," "potential," "possible" and similar expressions are intended to identify forward-looking statements. These forward-looking statements include statements regarding the immediate commencement of commercial distribution of Vyondys 53 in the U.S.; Vyondys 53’s continued approval for its indication potentially being contingent upon verification of a clinical benefit in confirmatory trials; the potential benefits and risks of Vyondys 53; Vyondys 53’s potential to treat up to another 8% of those living with DMD; the potential of Exondys 51 and Vyondys 53 to treat up to 20% of those with DMD in the U.S.; exon skipping’s intention to allow for production of an internally truncated dystrophin protein; and our mission to profoundly improve and extend the lives of patients with rare genetic-based diseases.

These forward-looking statements involve risks and uncertainties, many of which are beyond Sarepta’s control. Known risk factors include, among others: the planned commercial launch in the U.S. for Vyondys 53 may not be successful for various reasons including the actual market size and drug supply needed may not be consistent with the company’s expectations and its executed commercial readiness plans, the degree to which Vyondys 53 is accepted by patients and prescribed by physicians, manufacturing limitations that may not be anticipated or resolved for in a timely manner or at all, the efficiency of our manufacturing, sales, distribution and specialty pharmacy network in getting Vyondys 53 to the market and future economic, competitive, reimbursement and regulatory conditions that could negatively impact the commercial launch of Vyondys 53; we may not be able to comply with all FDA post-approval commitments and requirements with respect to Exondys 51 and Vyondys 53 in a timely manner or at all; we may not be able to complete clinical trials required by the FDA or other regulatory authorities for approval of our product candidates; the results of our ongoing research and development efforts and clinical trials for our products and product candidates may not be positive or consistent with prior results or demonstrate a safe treatment benefit or support an NDA or a BLA filing, positive advisory committee recommendation or marketing approval by the FDA or other regulatory authority; we may not be able to execute on our business plans including meeting our expected or planned regulatory milestones and timelines, clinical development plans and bringing our product candidates to market, including the commercialization of Vyondys 53, for various reasons, including factors outside of our control, such as possible limitations of company financial and other resources, manufacturing limitations that may not be anticipated or resolved for in a timely manner or at all, and regulatory, court or agency decisions, such as decisions by the United States Patent and Trademark Office with respect to patents that cover our product and product candidates; and those risks identified under the heading “Risk Factors” in Sarepta’s most recent Annual Report on Form 10-K for the year ended December 31, 2018, and most recent Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission (SEC) as well as other SEC filings made by the Company which you are encouraged to review.

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 Sarepta's 2018 Annual Report on Form 10-K and most recent Quarterly Report on Form 10-Q filed with the SEC as well as other SEC filings made by Sarepta. We caution investors not to place considerable reliance on the forward-looking statements contained in this press release. Sarepta does not undertake any obligation to publicly update its forward-looking statements based on events or circumstances after the date hereof.

Source: Sarepta Therapeutics, Inc.

Posted: December 2019