Registro de fuente primaria
Grado decisivoOtro
PTC Therapeutics retira la NDA de Translarna tras la opinión de la FDA
PTC ha retirado su resolicitud de NDA para Translarna (ataluren) en DMD por mutación sin sentido. La FDA indicó que los datos presentados probablemente no cumplen el umbral de evidencia sustancial de eficacia. La retirada pone fin a la actual vía de revisión regulatoria en EE.UU. para el producto.
Datos clave
- Presentado
- 12 feb 2026, 22:10
- Evento
- Otro
- Dirección
- Negativo
- Empresa
PTCTPTC Therapeutics- Activo
- ataluren
- Fuente
- SEC 8-K
- Confianza
- Con fuente
Extracto de la fuente
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Update on Translarna™ WARREN, N.J., Feb. 12, 2026 -- PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today that it has withdrawn the New Drug Application (NDA) resubmission for Translarna™ (ataluren) for the treatment of nonsense mutation Duchenne muscular dystrophy (DMD) following U.S.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