一次情報の記録
重要申請提出
uniQure、Huntington病向けifezuntirgene inilparvovecのBLAを提出
uniQureは、Huntington病に対するifezuntirgene inilparvovecの迅速承認を求めるBLAをFDAに提出した。同社はまた、英国MHRAにMAAを提出した。両申請は、外部対照と比較して疾患進行の抑制を示した3年間の第I/II相データに基づいている。
主要事実
出典の抜粋
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