PTC Therapeutics在收到FDA反馈后撤回Translarna的NDA

PTC has withdrawn its NDA resubmission for Translarna (ataluren) in nonsense mutation DMD. FDA indicated the submitted data are unlikely to meet the substantial evidence of effectiveness threshold. The withdrawal ends the current U.S. regulatory review path for the product.

关键事实

申报时间: 2026年2月12日 22:10
事件: 其他
方向: 消极
公司: PTCTPTC Therapeutics
资产: ataluren
来源: SEC 8-K
可信度: 有来源

来源摘录

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Update on Translarna™ WARREN, N.J., Feb. 12, 2026 -- PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today that it has withdrawn the New Drug Application (NDA) resubmission for Translarna™ (ataluren) for the treatment of nonsense mutation Duchenne muscular dystrophy (DMD) following U.S.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