PTC Therapeutics 撤回 Translarna NDA 申請，因 FDA 回饋意見

PTC 已撤回其 Translarna（ataluren）用於無義突變 DMD 的 NDA 重新提交。FDA 表示提交的資料不太可能達到有效性實質證據門檻。此撤回結束了該產品目前在美國的監管審查途徑。

關鍵事實

申報時間: 2026年2月12日下午10:10
事件: 其他
方向: 負面
公司: PTCTPTC Therapeutics
資產: ataluren
來源: SEC 8-K
可信度: 有出處

來源摘錄

EX-99.1 2 tmb-20260212xex99d1.htm EX-99.1 Exhibit 99.1 PTC Therapeutics Provides Regulatory Update on Translarna™ WARREN, N.J., Feb. 12, 2026 -- PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today that it has withdrawn the New Drug Application (NDA) resubmission for Translarna™ (ataluren) for the treatment of nonsense mutation Duchenne muscular dystrophy (DMD) following U.S. Food and Drug Administration (FDA) feedback on the application review. “FDA shared that based on its review to date, the data in the NDA submission are unlikely to meet the Agency’s threshold of substantial evidence of effectiveness to support approval of Translarna. We have therefore made the decision to withdraw the NDA submission,” said Matthew B. Klein, M.D., Chief Executive Officer of PTC Therapeutics. “W

SEC 8-K