Stealth BioTherapeutics Inc., a clinical-stage biotechnology company focused on the discovery, development and commercialization of novel therapies for diseases involving mitochondrial dysfunction,  announced that the FDA Cardiovascular and Renal Drugs Advisory Committee (“CRDAC”) voted 10 to 6 in favor of Stealth’s New Drug Application (“NDA”), concluding that elamipretide is effective for the treatment of patients with Barth syndrome.

Stealth BioTherapeutics Inc.是一家专注于线粒体功能障碍疾病新疗法的发现，开发和商业化的临床阶段生物技术公司，宣布FDA心血管和肾脏药物咨询委员会（“CRDAC”）以10票对6票赞成Stealth的新药申请（“NDA”），结论是elamipretide对Barth综合征患者的治疗有效。

The CRDAC discussed the benefits and risks of elamipretide for the treatment of Barth syndrome, including results from the TAZPOWER Part 2 baseline-controlled extension study, the SPIBA-001 Phase III natural history control study, and additional supportive biomarker and preclinical data. The CRDAC’s vote, while not binding, will be considered by the FDA when making its decision regarding the potential approval of elamipretide for Barth syndrome.

CRDAC讨论了elamipretide治疗Barth综合征的益处和风险，包括TAZPOWER第2部分基线对照扩展研究，SPIBA-001 III期自然史对照研究的结果，以及其他支持性生物标志物和临床前数据。CRDAC的投票虽然不具约束力，但FDA将在决定是否批准elamipretide治疗Barth综合征时予以考虑。

If approved, elamipretide would be the first therapy for this ultra-rare, progressive, life-shortening, cardioskeletal disease that affects approximately 150 individuals in the United States. The NDA for elamipretide was granted priority review and has been assigned a Prescription Drug User Fee Act (“PDUFA”) action date of January 29, 2025..

如果获得批准，elamipretide将成为这种超罕见，进行性，寿命缩短的心脏骨骼疾病的第一种治疗方法，该疾病影响美国约150人。elamipretide的NDA被授予优先审查权，并被指定为2025年1月29日的处方药用户费用法案（“PDUFA”）。。

If approved, this would be the first marketing authorization for elamipretide, a first-in-class mitochondria-targeted therapeutic. In addition to Barth syndrome, elamipretide is in Phase 3 trials for primary mitochondrial myopathy, with pivotal data expected by the end of 2024, and for dry age-related macular degeneration..

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。除Barth综合征外，elamipretide正在进行原发性线粒体肌病的3期临床试验，预计2024年底将有关键数据，以及与年龄相关的干性黄斑变性。。

About Barth Syndrome; 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.

关于Barth综合征；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 primarily 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综合征主要发生在男性身上，据估计全球每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拥有FDA指定的孤儿药、快速通道和罕见儿科药物，以及EMA指定的用于治疗Barth综合征的孤儿药。。

Condition: Barth Syndrome

病情：Barth综合征

Type: drug

类型：药物