1차 출처 기록
중대신청 제출
uniQure, 헌팅턴병 대상 ifezuntirgene inilparvovec BLA 제출
uniQure는 헌팅턴병 치료제 ifezuntirgene inilparvovec의 가속 승인을 위해 FDA에 BLA를 제출했습니다. 또한 영국 MHRA에 MAA를 제출했습니다. 두 제출은 외부 대조군 대비 질병 진행 둔화를 보여주는 3년간의 Phase I/II 데이터에 기반합니다.
핵심 사실
출처 발췌
EX-99.1 2 qure-20260902xex99d1.htm EX-99.1 Exhibit 99.1 uniQure Announces Submission of Biologics License Application for Ifezuntirgene Inilparvovec (AMT-130) in Huntington’s Disease ~ Company also announces the submission of a Marketing Authorisation Application for ifezuntirgene inilparvovec in the U.K. ~ ~ Both submissions are supported by the three-year data analysis from the Phase I/II study, in which ifezuntirgene inilparvovec demonstrated a slowing of disease progression ~ Lexington, MA and Amsterdam, the Netherlands, September 2, 2026 — uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced the submission of a Biologics License Application (BLA) to the United States (U.S.) Food and Dru