Beren Therapeutics has announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation (BTD) to adrabetadex, an investigational treatment for infantile-onset Niemann-Pick disease type C (NPC).

贝伦治疗公司宣布，美国食品和药物管理局 (FDA) 已授予 adrabetadex 突破性疗法认定 (BTD)，这是一种用于治疗婴儿期发病的尼曼-匹克病C型 (NPC) 的研究性疗法。

Infantile-onset NPC is a rare and severe neurodegenerative condition in which neurological symptoms appear between birth and six years of age. Earlier onset is associated with faster progression and reduced life expectancy, with average survival of around 5.6 years for early-infantile cases and approximately 13.4 years for late-infantile onset.

婴儿期发作的NPC是一种罕见且严重的神经退行性疾病，其神经系统症状在出生至六岁之间显现。发病越早，病情进展越快，预期寿命越短；早期婴儿型病例的平均生存期约为5.6年，而晚期婴儿型发病的平均生存期约为13.4年。

The condition affects multiple organs, with the most serious complications occurring in the brain..

这种病影响多个器官，最严重的并发症发生在大脑。

Adrabetadex (VTS-270) is a mixture of 2-hydroxypropyl-β-cyclodextrin isomers designed to restore intracellular cholesterol transport, directly addressing the underlying cause of NPC. The treatment has generally shown acceptable safety, with common side effects including manageable hearing impairment and occasional fatigue or ataxia following dosing..

Adrabetadex（VTS-270）是一种2-羟丙基-β-环糊精异构体的混合物，旨在恢复细胞内胆固醇运输，直接针对NPC的根本原因。该治疗通常显示出可接受的安全性，常见副作用包括可控的听力损伤以及给药后偶尔出现的疲劳或共济失调。

The designation is intended to accelerate the development of medicines for serious or life-threatening conditions where early clinical evidence indicates the potential for significant benefit over existing options.

该指定旨在加速开发用于严重或危及生命状况的药物，其中早期临床证据表明其相较于现有疗法有显著益处的潜力。

The FDA’s decision was based on an externally controlled survival analysis showing that adrabetadex may improve survival in individuals with infantile-onset NPC, supported by additional biomarker and nonclinical findings. Adrabetadex previously received BTD in 2016 under a different sponsor, but the designation was later withdrawn following results from a Phase 2b/3 trial.

FDA的决定基于一项外部对照的生存分析，该分析显示adrabetadex可能改善婴儿期起病的NPC患者的生存率，并得到了额外的生物标志物和非临床研究结果的支持。Adrabetadex曾在2016年由另一家赞助商获得BTD（突破性疗法认定），但在2b/3期试验结果公布后，该认定被撤销。

Since acquiring the programme in 2021, Beren and Mandos have been working closely with the FDA and the NPC community to advance the therapy and develop new evidence that supports the renewed designation..

自 2021 年收购该计划以来，贝伦和曼多斯一直与 FDA 和 NPC 社区密切合作，以推进该疗法并开发支持重新指定的新证据。

The therapy also holds Orphan Drug and Rare Paediatric Disease designations. Beren plans to submit a New Drug Application (NDA) soon and expects the treatment to be considered for Priority Review.

该疗法还获得了孤儿药和罕见儿科疾病认定。Beren计划很快提交新药申请（NDA），并预计该治疗将被考虑进行优先审查。

Source: businesswire.com

来源：businesswire.com