一次情報の記録
決定級その他
uniQure: FDA、Phase I/IIデータがAMT-130のBLAに十分であることに同意せず
FDAは以前の指針を撤回し、外部対照群との比較によるPhase I/IIデータがBLA申請の主要根拠として十分でない可能性があると述べている。AMT-130のBLA申請予定時期は現時点で不明。uniQureは最終会議録を取得し、FDAと緊急フォローアップ協議を行い、進むべき道筋を特定する予定。
主要事実
出典の抜粋
EX-99.1 2 qure-20251103xex99d1.htm EX-99.1 Exhibit 99.1 uniQure Provides Regulatory Update on AMT-130 for Huntington ’ s Disease Lexington, MA and Amsterdam, the Netherlands, November 3, 2025 — uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that it received feedback from the U.S. Food and Drug Administration (FDA) during a recent pre-Biologics License Application (BLA) meeting regarding AMT-130, an investigational gene therapy for Huntington ’ s disease (HD). Though final meeting minutes have not yet been received, based on the discussions at the meeting, uniQure believes that the FDA currently no longer agrees that data from the Phase I/II studies of AMT-130 in comparison to an ex