一次情報の記録
決定級試験登録の変更
uniQure: FDA、AMT-130のPhase I/II外部対照データを却下
FDAは、外部対照に対するPhase I/IIデータはマーケティング申請を支持するには不十分であると述べ、将来の無作為化二重盲検シャム対照Phase III試験を強く推奨した。uniQureは2026年第2四半期にPhase IIIデザインの選択肢を議論するためType Bミーティングを要請する予定で、規制タイムラインは大幅に延長される見込み。
主要事実
出典の抜粋
EX-99.1 2 qure-20260302xex99d1.htm EX-99.1 Exhibit 99.1 uniQure Announces 2025 Financial Results and Provides Recent Company Updates ~ Held Type A meeting with FDA to discuss AMT-130 for Huntington’s disease; Company evaluating Phase III development considerations and plans to request follow-up Type B meeting in the second quarter of 2026 ~ ~ Completed enrollment of the first cohort in the Phase I/IIa study of AMT-260 in refractory mesial temporal lobe epilepsy, with additional clinical data expected in the first half of 2026~ ~ Presented updated Phase I/II data from AMT-191 in Fabry disease showing durable, dose-dependent increases in α-Gal A enzyme activity ~ ~ Cash, cash equivalents and current investment securities of approximately $622.5 million as of December 31, 2025 expected to f