原始出處紀錄
重大申請已送件
uniQure 提交 ifezuntirgene inilparvovec 用於亨廷頓氏症的 BLA
uniQure 已向 FDA 提交 BLA，尋求 ifezuntirgene inilparvovec 用於亨廷頓氏症的加速核准。該公司也向英國 MHRA 提交 MAA。兩項申請均基於三年期 Phase I/II 資料，顯示疾病進展相較外部對照組有所減緩。
關鍵事實
來源摘錄
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