一次情報の記録
重要その他
uniQure: FDA、Phase I/IIデータはAMT-130のBLAを支持する可能性が低いと判断
FDAの会議議事録は、Phase I/IIデータが現時点でBLA申請の主要な根拠となる可能性が低いことを確認しています。uniQureは2026年Q1にAMT-130ハンチントン病プログラムの次のステップを議論するため、FDAとのフォローアップミーティングを要請する予定です。
主要事実
出典の抜粋
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