GV101获FDA快速通道认定：弗里德赖希共济失调口服新药将启动2期试验
位于纽约的临床阶段生物技术公司 Graviton BioScience 于11月3日宣布，食品药品监管局（FDA）已授予其在研口服药物 GV101 快速通道认定（Fast Track Designation），用于治疗罕见遗传病弗里德赖希共济失调（Friedreich's ataxia，FA）。快速通道是 FDA 为治疗严重疾病、解决未满足医疗需求的在研疗法设立的加速开发与审评通道。
弗里德赖希共济失调由 frataxin 基因中的 GAA 三核苷酸重复扩增引起，患者体内 frataxin 蛋白表达减少，线粒体功能受损，进而累及心血管、神经与肌肉骨骼系统。患者会逐渐失去协调能力、出现肌无力、言语含糊与吞咽困难，许多人最终依赖轮椅，心肌肥厚等心脏并发症占该病相关死亡的一半以上，患者多在50岁前去世。美国约有5,000名患者，全球估计约15,000名；症状通常在儿童或青少年时期出现。
GV101 是一种强效、高选择性的口服 ROCK2 抑制剂。公司称，抑制 ROCK2 可以同时提高 frataxin 的转录与翻译，从该病的遗传根源入手，有望成为改变疾病进程的疗法；临床前研究显示 GV101 能提高 frataxin 的信使核糖核酸与蛋白水平，并具有神经保护作用。
公司创始人兼首席执行官 Samuel Waksal 表示，获得快速通道认定是 GV101 的重要里程碑，凸显了针对该病底层生物学机制疗法的迫切未满足需求；加上近期其新药临床试验申请（IND）已获 FDA 放行，公司正推进2期研究的准备工作。公司计划开展一项为期12周、随机、安慰剂对照的剂量探索2期试验，首批入组预计2026年第四季度在美国启动、随后扩展至全球。公司称，GV101 已在500多名受试者中用药最长24周、延长期研究超过一年，显示出良好的安全性与耐受性。
公司提醒，GV101 为在研产品，尚未获 FDA 或任何监管机构批准，其潜在疗效仍需临床试验验证。
（来源：Graviton BioScience）
GV101 Wins FDA Fast Track Designation: Oral Friedreich's Ataxia Drug Heads to Phase 2 Trial
New York-based clinical-stage company Graviton BioScience announced on November 3 that the U.S. Food and Drug Administration (FDA) granted Fast Track Designation to GV101, its clinical-stage oral selective ROCK2 inhibitor, for the treatment of Friedreich's ataxia (FA). The FDA's Fast Track program is designed to facilitate the development and expedite the review of investigational therapies intended to treat serious conditions and address unmet medical needs.
Friedreich's ataxia is a rare, inherited, multisystem disorder caused by GAA trinucleotide repeat expansions in the frataxin gene that result in reduced production of frataxin, a protein essential for mitochondrial function. Patients experience progressive loss of coordination, weakness, wheelchair dependence, slurred speech and difficulty swallowing; hypertrophic cardiomyopathy is common, and cardiac complications account for more than half of FA-related deaths, with premature mortality usually before age 50. The disease affects approximately 5,000 people in the United States and an estimated 15,000 worldwide, with symptoms typically beginning in childhood or adolescence.
GV101 is a potent, highly selective oral inhibitor of ROCK2. The company says ROCK2 inhibition targets the root genetic cause of the disease by increasing both frataxin transcription and translation, alleviating mitochondrial dysfunction, and that preclinical studies have shown GV101 increases frataxin mRNA and protein levels, offering a potentially disease-modifying approach, alongside neuroprotective effects.
Founder and Chief Executive Officer Samuel Waksal, Ph.D., said receiving Fast Track Designation is an important milestone for GV101 and underscores the significant unmet need for therapies that address the underlying biology of Friedreich's ataxia. Together with the recent clearance of the company's Investigational New Drug application, he said, the designation provides momentum as Graviton prepares a 12-week, randomized, placebo-controlled, dose-finding Phase 2 trial, with first enrollment expected at U.S. sites in the fourth quarter of 2026 and globally shortly thereafter. The company said more than 500 participants have received GV101 in clinical studies for durations of up to 24 weeks, with extension studies exceeding one year, showing a favorable safety and tolerability profile.
The company noted that GV101 is an investigational product and has not been approved by the FDA or any other regulatory authority.
(Source: Graviton BioScience)
来源：Graviton BioScience
发布：GSM·美国（发布链接）
图片来源：实验室技术人员移取样本（药物研发实验示意）。图：美国国家标准与技术研究院 NIST，公共领域，经 Wikimedia Commons / A laboratory technician pipettes a sample (drug research illustration). Photo: National Institute of Standards and Technology, public domain, via Wikimedia Commons
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