原始来源记录
重大申请已提交
Dyne Therapeutics：z-rostudirsen BLA 提交按计划于 2026 年第二季度进行
Dyne 已与 FDA 完成积极的 pre-BLA 会议，仍按计划于 2026 年第二季度提交 z-rostudirsen 用于外显子 51 DMD 的美国加速批准 BLA。公司还计划于 2026 年第二季度启动全球确证性 3 期试验，假设获得优先审评和批准，潜在美国上市时间定为 2027 年第一季度。
关键事实
- 申报时间
- 2026年5月11日 11:35
- 事件
- 申请已提交
- 方向
- 积极
- 来源
- SEC 8-K
- 可信度
- 有来源
来源摘录
EX-99.1 2 dyn-ex99_1.htm EX-99.1 EX-99.1 Exhibit 99.1 Dyne Therapeutics Reports First Quarter 2026 Financial Results and Recent Business Highlights - Positive pre-BLA meeting completed with FDA for z-rostudirsen in exon 51 DMD; on track for BLA submission in Q2 2026 and potential launch in Q1 2027 - - Positive cardiopulmonary results and long-term dystrophin data from Phase 1/2 DELIVER trial of z-rostudirsen in exon 51 DMD presented at MDA conference - - Completion of enrollment in registrational expansion cohort of Phase 1/2 ACHIEVE trial of z-basivarsen in DM1 on track for Q2 2026; global confirmatory Phase 3 HARMONIA trial initiated - - New preclinical data to be presented at ASGCT underscore differentiated capability of clinically validated FORCE TM platform to cross the blood-brain