Catatan sumber primer
PenentuLainnya
PTC Therapeutics menarik NDA Translarna setelah umpan balik FDA
PTC telah menarik pengajuan ulang NDA untuk Translarna (ataluren) pada DMD mutasi nonsense. FDA mengindikasikan bahwa data yang diajukan kemungkinan tidak memenuhi ambang bukti efektivitas yang substansial. Penarikan ini mengakhiri jalur tinjauan regulasi AS saat ini untuk produk tersebut.
Fakta utama
- Diajukan
- 12 Feb 2026, 22.10
- Peristiwa
- Lainnya
- Arah
- Negatif
- Perusahaan
PTCTPTC Therapeutics- Aset
- ataluren
- Sumber
- SEC 8-K
- Keyakinan
- Bersumber
Kutipan sumber
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Update on Translarna™ WARREN, N.J., Feb. 12, 2026 -- PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today that it has withdrawn the New Drug Application (NDA) resubmission for Translarna™ (ataluren) for the treatment of nonsense mutation Duchenne muscular dystrophy (DMD) following U.S.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