NEEDHAM, Mass., May 7, 2024 /PRNewswire/ -- Stealth Biotherapeutics, 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') has now determined that its New Drug Application ('NDA') for elamipretide for the treatment of Barth syndrome is entitled to a Priority Review designation.

马萨诸塞州李约瑟市，2024年5月7日/PRNewswire/--Stealth Biotherapeutics是一家临床阶段生物技术公司，专注于发现、开发和商业化针对线粒体功能障碍疾病的新型疗法，今天宣布，美国食品和药物管理局（FDA）现已确定其用于治疗Barth综合征的依拉米普瑞肽新药申请（NDA）有权获得优先审查指定。

Per FDA policy, the user fee goal date of January 29, 2025 is unchanged..

根据FDA政策，2025年1月29日的用户费用目标日期不变。。

The FDA grants Priority Review to applications for therapies that have the potential to provide significant improvements in the treatment, diagnosis, or prevention of serious conditions. Barth syndrome is an ultra-rare cardioskeletal disease which results in frequent pediatric deaths and imposes a significant disease burden on all affected individuals.

FDA优先审查有可能在治疗，诊断或预防严重疾病方面提供显着改善的治疗申请。Barth综合征是一种极为罕见的心脏骨骼疾病，可导致儿科频繁死亡，并对所有受影响的个体造成重大疾病负担。

There are no other therapies approved or known to be in clinical development for this disease..

没有其他疗法被批准或已知正在临床开发中用于这种疾病。。

The FDA's decision to grant Priority Review designation follows its March 29, 2024 filing decision in which the FDA assigned a standard review designation. Elamipretide has previously received Orphan Drug, Fast Track and Rare Pediatric Designations for elamipretide for the treatment of Barth syndrome..

美国食品和药物管理局（FDA）决定授予优先审查指定是在其2024年3月29日提交决定之后做出的，在该决定中，FDA指定了标准审查指定。Elamipretide之前曾接受孤儿药，快速通道和罕见的儿科指定的Elamipretide治疗Barth综合征。。

'We are pleased that the FDA has recognized the serious nature of Barth syndrome, the urgency of the unmet medical need, and the potential for elamipretide, if approved, to significantly improve treatment of this devastating disease,' said Reenie McCarthy, Chief Executive Officer. 'We welcome our continued constructive engagement with the Agency as we complete the review process and plan for an anticipated advisory committee meeting later this year.'.

首席执行官瑞妮·麦卡锡（ReenieMcCarthy）说：“我们很高兴美国食品和药物管理局（FDA）已经认识到巴特综合征的严重性，医疗需求尚未得到满足的紧迫性，以及依拉米普瑞特（elamipretide）如果获得批准，有可能显着改善这种毁灭性疾病的治疗。”我们欢迎在完成审查过程并计划今年晚些时候召开预期的咨询委员会会议时，继续与该机构进行建设性接触。”。

If approved, this would be the first marketing authorization for elamipretide, a first-in-class mitochondrial targeted therapeutic. Elamipretide is also in development for primary mitochondrial myopathy, with pivotal data from the fully-enrolled Phase 3 NuPOWER trial expected in late 2024, and dry age-related macular degeneration, with pivotal Phase 3 clinical trials initiating this quarter..

如果获得批准，这将是elamipretide的首次上市授权，elamipretide是一流的线粒体靶向治疗药物。Elamipretide也正在开发原发性线粒体肌病，预计2024年底将进行全面登记的3期NuPOWER试验的关键数据，以及与年龄相关的干性黄斑变性，关键的3期临床试验将于本季度开始。。

About Barth Syndrome

关于Barth综合征

Barth syndrome is an ultra-rare genetic condition characterized by cardiac abnormalities 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 5.

Barth综合征是一种极为罕见的遗传病，其特征是心脏异常导致运动不耐症，肌肉无力，衰弱性疲劳，心力衰竭，反复感染和生长迟缓。这种疾病与预期寿命缩短有关，85%的早期死亡发生在5岁之前。

Barth syndrome occurs almost exclusively in males and is estimated to affect one in 1,000,000 males worldwide or around 150 individuals in the United States. There are currently no FDA- or EMA-approved therapies for patients with Barth syndrome. Elamipretide has Orphan Drug, Fast Track and Rare Pediatric Designation from the FDA and Orphan Drug Designation from the EMA for the treatment of Barth syndrome. .

Barth综合征几乎只发生在男性身上，据估计，全世界每100万男性中就有一人或美国约150人受到影响。目前还没有FDA或EMA批准的Barth综合征患者治疗方法。Elamipretide具有FDA的孤儿药，快速通道和罕见儿科指定以及EMA的孤儿药指定，用于治疗Barth综合征。。

About Stealth BioTherapeutics

关于隐形生物疗法

Stealth BioTherapeutics is a clinical-stage biotechnology company focused on the discovery, development, and commercialization of novel therapies for diseases involving mitochondrial dysfunction. Mitochondria, found in nearly every cell in the body, are the body's main source of energy production and are critical for normal organ function.

Stealth BioTherapeutics是一家临床阶段的生物技术公司，专注于发现，开发和商业化涉及线粒体功能障碍的疾病的新型疗法。线粒体几乎存在于身体的每个细胞中，是身体产生能量的主要来源，对正常的器官功能至关重要。

Dysfunctional mitochondria characterize a number of rare genetic diseases and are involved in many common age-related diseases, typically involving organ systems with high energy demands such as the heart, the eye, and the brain. The Company believes its lead product candidate, elamipretide, has the potential to treat rare diseases including Barth syndrome and primary mitochondrial myopathy and common age-related diseases including dry age-related macular degeneration.

功能失调的线粒体是许多罕见遗传疾病的特征，并参与许多常见的与年龄有关的疾病，通常涉及具有高能量需求的器官系统，例如心脏，眼睛和大脑。该公司认为其主要候选产品elamipretide有潜力治疗罕见疾病，包括Barth综合征和原发性线粒体肌病，以及常见的年龄相关疾病，包括干性年龄相关性黄斑变性。

The Company is also evaluating a topical ophthalmic formulation of its second-generation clinical-stage candidate, SBT-272, for dry age-related macular degeneration. The Company has a deep pipeline of novel compounds under evaluation for rare neurological and cardiac disease indications following promising preclinical data..

该公司还正在评估其第二代临床阶段候选药物SBT-272的局部眼科制剂，用于治疗干性年龄相关性黄斑变性。根据有希望的临床前数据，该公司拥有一系列新型化合物，正在评估罕见的神经和心脏疾病适应症。。