Ionis：zilganersen PDUFA 日期定于 2026 年 9 月 22 日

FDA 已将 zilganersen 的 PDUFA 行动日期定于 2026 年 9 月 22 日，并授予优先审评。新闻稿还公布了来自关键研究的额外阳性数据，涵盖运动、患者报告和临床医生报告的多个终点，支持此前公布的顶线结果。

关键事实

申报时间: 2026年4月21日 11:00
事件: PDUFA 日期确定
方向: 积极
公司: IONSIonis
资产: zilganersen
来源: SEC 8-K
可信度: 有来源
日历: FDA 审批决定 (PDUFA) · 2026年9月22日 · 积极
GFAP p值: 0.003
10米步行测试p值: 0.041
GMFM-88 p值: 0.034
GFAP降低: 33.6%
10米步行测试差异: 33.3%
GMFM-88差异: 22.9分

来源摘录

EX-99.1 2 ef20071051_ex99-1.htm EXHIBIT 99.1 Exhibit 99.1 FINAL: April 20, 2026 Ionis presents new data from pivotal study of zilganersen in Alexander disease (AxD) at AAN 2026 Annual Meeting – New data highlight potential treatment benefit across multiple AxD symptom domains, reinforcing zilganersen’s positive impact on people living with this rare, often fatal neurological disease – – PDUFA date set for September 22, 2026 – CARLSBAD, Calif., April 21, 2026 -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced additional positive results from the pivotal study of zilganersen in children and adults living with Alexander disease (AxD), a rare, progressive and often fatal neurological condition with no approved disease-modifying treatments. These findings, which will be prese

SEC 8-K