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uniQure: FDA, Phase I/II 데이터가 AMT-130 BLA를 뒷받침하기 어려울 것으로 판단
FDA 회의록에 따르면 Phase I/II 데이터는 현재 BLA 제출의 주요 근거로 활용되기 어려운 것으로 확인되었습니다. uniQure는 2026년 1분기에 후속 FDA 미팅을 요청하여 AMT-130 헌팅턴병 프로그램의 다음 단계를 논의할 예정입니다.
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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