一次情報の記録
決定級その他
PTC Therapeutics、FDAのフィードバックを受けてTranslarnaのNDAを撤回
PTCは、ナンセンス変異DMD向けTranslarna（ataluren）のNDA再提出を撤回した。FDAは提出データが有効性の実質的証拠基準を満たす可能性が低いと指摘した。この撤回により、同製品の米国での現行規制審査経路は終了した。
主要事実
出典の抜粋
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Update on Translarna™ WARREN, N.J., Feb. 12, 2026 -- PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today that it has withdrawn the New Drug Application (NDA) resubmission for Translarna™ (ataluren) for the treatment of nonsense mutation Duchenne muscular dystrophy (DMD) following U.S.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