uniQure: Tinanggihan ng FDA ang Phase I/II external-control data para sa AMT-130

Sinabi ng FDA na ang Phase I/II data kumpara sa external control ay hindi sapat upang suportahan ang marketing application at mariing inirerekomenda ang prospective, randomized, double-blind, sham-controlled Phase III study. Plano ng uniQure na humiling ng Type B meeting sa Q2 2026 upang talakayin ang Phase III design options, na makabuluhang magpapahaba sa regulatory timeline.

Mahahalagang fact

Na-file: Mar 2, 2026, 12:06 PM
Kaganapan: Pagbabago sa trial registry
Direksyon: Negatibo
Kumpanya: QUREuniQure
Asset: AMT-130
Pinagmulan: SEC 8-K
Kumpiyansa: May source

Sipi mula sa source

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

SEC 8-K