Stealth BioTherapeutics宣布 FDA对 Barth综合征申请的行动日期延迟-动脉网

Stealth BioTherapeutics Announces Delay in FDA Action Date for Barth Syndrome Application

CISION 等信源发布 2025-04-29 18:00

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- FDA decision on elamipretide for the treatment of Barth syndrome delayed -

- FDA 对 elamipretide 治疗 Barth 综合征的决定延迟 -

- Labelling discussions underway, but no revised action date communicated by FDA -

- 标签讨论正在进行中，但FDA尚未传达修订后的行动日期 -

NEEDHAM, Mass.

需要汉姆，马萨诸塞州

，

April 29, 2025

2025年4月29日

/PRNewswire/ -- Stealth BioTherapeutics Inc. (the 'Company' or 'Stealth'), a clinical-stage biotechnology company focused on the discovery, development and commercialization of novel therapies for diseases involving mitochondrial dysfunction, today announced that the U.S. Food and Drug Administration (FDA) communicated that it would not meet today's prescription drug user fee action (PDUFA) date for its new drug application (NDA) for its investigational medicine, elamipretide, for the treatment of Barth syndrome.

/PRNewswire/ -- 专注于发现、开发和商业化治疗线粒体功能障碍相关疾病新型疗法的临床阶段生物技术公司Stealth BioTherapeutics Inc.（“公司”或“Stealth”）今天宣布，美国食品药品监督管理局（FDA）表示将无法在今天完成对其研究性药物elamipretide（用于治疗Barth综合征）的新药申请（NDA）的处方药用户费用法案（PDUFA）日期的审批。

Barth syndrome is an ultra-rare, life threatening pediatric mitochondrial disease which is known to affect less than 150 individuals in .

巴特综合征是一种超罕见、危及生命的儿童线粒体疾病，已知患者人数不到150人。

the United States

美国

and less than 300 individuals worldwide.

全球不到300人。

'We appreciate the diligent efforts of the FDA to progress its review of our new drug application, including its confirmation of post-marketing requirements and its recent initiation of labeling discussions,' said

“我们感谢FDA为推进对我们新药申请的审查所做的不懈努力，包括其对上市后要求的确认以及最近启动的标签讨论，”

Reenie McCarthy

琳妮·麦卡锡

, Chief Executive Officer. 'We hope to gain more information on the revised action date in the coming days to inform our ability to continue supporting expanded and emergency access to elamipretide while advancing towards a potential FDA approval.'

，首席执行官。“我们希望在未来几天内获得更多关于修订后行动日期的信息，以帮助我们继续支持扩大和紧急使用 elamipretide，同时朝着潜在的 FDA 批准迈进。”

Stealth's NDA was submitted to the FDA on

Stealth的NDA已提交给FDA

January 29, 2024

2024年1月29日

and filed by the FDA for standard review on

并由FDA提交以进行标准审查

March 29, 2024

2024年3月29日

with a PDUFA date of

PDUFA日期为

January 29, 2025

2025年1月29日

. On

。打开

May 6, 2024

2024年5月6日

, FDA granted priority review. On

，FDA授予优先审查。在

October 10, 2024

2024年10月10日

, the Cardiovascular and Renal Drugs Advisory Committee (CRDAC) met and concluded that elamipretide is effective for the treatment of patients with Barth syndrome. On

，心血管和肾脏药物咨询委员会 (CRDAC) 召开会议并得出结论，elamipretide 对 Barth 综合征患者的治疗有效。

January 14, 2025

2025年1月14日

, the FDA notified Stealth that new analyses requested by FDA in late November and early

，FDA通知Stealth，FDA在11月下旬和12月初要求的新分析

December 2024

2024年12月

required a delay of the PDUFA date to

需要推迟PDUFA日期以

April 29, 2025

2025年4月29日

。

Elamipretide has received Orphan Drug, Fast Track, Priority Review, and Rare Pediatric Designations from the FDA and Orphan Drug Designation from the European Medicines Agency (EMA) for the treatment of Barth syndrome. More than 35 patients worldwide have received expanded or emergency access to elamipretide for the treatment of Barth syndrome, including many critically ill infants and toddlers affected by the disease..

Elamipretide 已获得美国食品药品监督管理局（FDA）授予的孤儿药、快速通道、优先审评和罕见儿科疾病认定，以及欧洲药品管理局（EMA）授予的孤儿药认定，用于治疗巴特综合征。全球已有超过 35 名患者通过扩大使用或紧急途径获得 elamipretide 治疗巴特综合征，其中包括许多受该疾病影响的危重婴儿和幼儿。

About Barth Syndrome

关于巴特综合征

Barth syndrome is an ultra-rare genetic mitochondrial disease leading to exercise intolerance, muscle weakness, debilitating fatigue, heart failure, recurrent infections, and delayed growth. The disease is associated with reduced life expectancy, with 85% of early deaths occurring by age five. Barth syndrome occurs primarily in males and is estimated to affect one in 1,000,000 males worldwide or around 150 individuals in the .

巴特综合征是一种超罕见的遗传性线粒体疾病，会导致运动不耐受、肌肉无力、严重疲劳、心力衰竭、反复感染和生长迟缓。该疾病与预期寿命缩短相关，85%的早亡发生在五岁之前。巴特综合征主要发生在男性中，估计全球每1,000,000名男性中有一人受到影响，大约有150人。

United States

美国

. There are no approved therapies for the treatment of Barth syndrome.

巴特综合征的治疗目前尚无获批的疗法。

About Stealth BioTherapeutics

关于Stealth BioTherapeutics

Our mission is to develop novel therapies to improve the lives of patients living with diseases of mitochondrial dysfunction. Our lead product candidate, elamipretide, is under review for Barth syndrome and in late-stage development for primary mitochondrial myopathy and dry age-related macular degeneration, where we observed a highly protective effect on photoreceptor loss in Phase 2.

我们的使命是开发新型疗法，以改善线粒体功能障碍疾病患者的生活。我们的主要候选产品elamipretide正在接受巴特综合征的审查，并在原发性线粒体肌病和干性年龄相关性黄斑变性的晚期开发阶段，在二期研究中我们观察到其对光感受器损失具有显著的保护作用。

We are evaluating a topical ophthalmic formulation of our second-generation clinical-stage candidate, bevemipretide (SBT-272), for dry age-related macular degeneration. We have a deep pipeline of novel compounds under evaluation for rare neurological, cardiac and myopathic diseases. .

我们正在评估第二代临床阶段候选药物贝米普肽（SBT-272）的眼用局部制剂，用于治疗干性年龄相关性黄斑变性。我们拥有一个深层次的新型化合物管线，正在评估其在罕见神经、心脏和肌病疾病中的应用。

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Anna Stallmann Communications

安娜·斯塔尔曼通讯

Anna Stallmann

安娜·施塔尔曼

anna@annacomms.com

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