FDA 受理 Biohaven 罕见病新药 Vyglxia 重新提交的上市申请：目标审评日期为2027年第一季度
美国制药公司 Biohaven 10月22日通过向美国证券交易委员会提交的 8-K 文件披露，美国食品药品监管局（FDA）已受理其重新提交的新药上市申请，该药为 Vyglxia（通用名 troriluzole），拟用于治疗脊髓小脑性共济失调（SCA）。FDA 将该申请的处方药使用者费用目标日期定在2027年第一季度，并计划召开外周与中枢神经系统顾问委员会会议听取外部意见，会议日期将于近期公布。
FDA 认为，这次重新提交构成对该局2025年11月4日发出的完整回复函的完整回应。Biohaven 表示，重新提交的材料中包含了新的、积极的支持性分析，但文件未披露这些分析的具体内容，也未报告新的试验数据。公司同时提示，目标日期只是审评目标，并不保证 FDA 届时采取行动或批准该药，审评期可能延长，FDA 也可能要求补充信息或再次发出完整回复函。
脊髓小脑性共济失调是一种罕见的遗传性神经退行性疾病，可危及生命，其特征是自主运动控制能力逐渐丧失，小脑与脑干出现萎缩，患者会出现平衡、协调、行走与言语方面的障碍。troriluzole 是一种在研口服药物，目前尚未在美国获批用于任何适应症。
消息公布后，Biohaven 股价在10月22日盘前交易中一度上涨约39%，至16.35美元。该公司总部位于 Connecticut 州 New Haven，为临床阶段生物制药公司。
FDA Accepts Biohaven's Resubmitted Vyglxia Application for Rare Disease SCA, Sets Q1 2027 Goal Date
Biohaven disclosed in a Form 8-K filing dated October 22, 2026, that the U.S. Food and Drug Administration (FDA) has accepted for review its resubmitted new drug application for Vyglxia (troriluzole) for the treatment of spinocerebellar ataxia (SCA). The FDA assigned a user fee goal date in the first quarter of 2027 and plans to convene its Peripheral and Central Nervous System Advisory Committee to review the application, with the meeting date to be announced in the near term.
The FDA considers the resubmission a complete response to the complete response letter it issued on November 4, 2025. Biohaven said the resubmission included new positive supportive analyses, though the filing did not describe the analyses or report trial data. The company noted that the goal date does not guarantee FDA action or approval, that the review may be extended, and that the agency could request more information or issue another complete response letter.
Spinocerebellar ataxia is a rare, life-threatening inherited neurodegenerative disease characterized by progressive loss of voluntary motor control and atrophy of the cerebellum and brainstem, affecting balance, coordination, walking and speech. Troriluzole is an investigational oral drug and has not been approved for any indication in the United States. Biohaven shares rose about 39% in premarket trading on October 22 to $16.35 following the announcement, according to Dow Jones Newswires.
来源：GSM·美国（原文链接）
图片来源：图：美国食品药品监管局 FDA 总部入口（FDA 官方图，公共领域）
