Record mula sa primary source
Decision-gradeIba pa
Binawi ng PTC Therapeutics ang NDA ng Translarna matapos ang feedback ng FDA
PTC has withdrawn its NDA resubmission for Translarna (ataluren) in nonsense mutation DMD.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.
Mahahalagang fact
- Na-file
- Peb 12, 2026, 10:10 PM
- Kaganapan
- Iba pa
- Direksyon
- Negatibo
- Kumpanya
PTCTPTC Therapeutics- Asset
- ataluren
- Pinagmulan
- SEC 8-K
- Kumpiyansa
- May source
Sipi mula sa source
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Update on Translarna™ WARREN, N.J., Feb. 12, 2026 -- PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today that it has withdrawn the New Drug Application (NDA) resubmission for Translarna™ (ataluren) for the treatment of nonsense mutation Duchenne muscular dystrophy (DMD) following U.S.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