uniQure: FDA menyatakan data Fase I/II tidak mungkin mendukung BLA AMT-130

Risalah pertemuan FDA mengonfirmasi bahwa data Fase I/II saat ini tidak mungkin menjadi bukti utama untuk pengajuan BLA. uniQure akan meminta pertemuan lanjutan dengan FDA pada Q1 2026 untuk membahas langkah selanjutnya bagi program AMT-130 untuk penyakit Huntington.

Fakta utama

Diajukan: 4 Des 2025, 12.06
Peristiwa: Lainnya
Arah: Negatif
Perusahaan: QUREuniQure
Aset: AMT-130
Sumber: SEC 8-K
Keyakinan: Bersumber

Kutipan sumber

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