uniQure: FDA sabi na ang Phase I/II data ay hindi malamang na suportahan ang AMT-130 BLA

Kinumpirma ng FDA meeting minutes na ang Phase I/II data ay kasalukuyang hindi malamang na magsilbing pangunahing ebidensya para sa BLA submission. Hihingi ang uniQure ng follow-up FDA meeting sa Q1 2026 para talakayin ang mga susunod na hakbang para sa AMT-130 Huntington's disease program.

Mahahalagang fact

Na-file: Dis 4, 2025, 12:06 PM
Kaganapan: Iba pa
Direksyon: Negatibo
Kumpanya: QUREuniQure
Asset: AMT-130
Pinagmulan: SEC 8-K
Kumpiyansa: May source

Sipi mula sa source

EX-99.1 2 qure-20251204xex99d1.htm EX-99.1 Exhibit 99.1 uniQure Provides Regulatory Update on AMT-130 for Huntington ’ s Disease Lexington, MA and Amsterdam, the Netherlands, December 4, 2025 — uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that the company received final meeting minutes from the U.S. Food and Drug Administration (FDA) regarding a pre-Biologics License Application (BLA) meeting held on October 29, 2025 to discuss the application for AMT-130, an investigational gene therapy for Huntington ’ s disease (HD). In the final meeting minutes, and consistent with uniQure ’ s November 3, 2025 press release, the FDA conveyed that data submitted from the Phase I/II studies o

SEC 8-K