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중대신청 제출
uniQure, 헌팅턴병 대상 AMT-130 BLA 제출 계획 발표
FDA는 3년차 Phase I/II 데이터가 가속승인을 위한 BLA의 주요 근거로 활용될 수 있음을 확인했습니다. 회사는 2026년 3분기에 BLA를 제출할 계획이며, 제출 전에 FDA와 확증 연구 설계에 대해 합의해야 합니다.
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EX-99.1 2 tm2618105d1_ex99-1.htm EXHIBIT 99.1 Exhibit 99.1 uniQure Announces Plan for BLA Submission for AMT-130 in Huntington’s Disease ~ 3-year analysis from the Phase I/II study can serve as the primary basis of a Biologics License Application for accelerated approval with FDA ~ ~ Company intends to submit the BLA in third quarter of 2026 ~ Lexington, MA and Amsterdam, the Netherlands, June 17, 2026 — uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that, during a recent Type B meeting with the U.S. Food and Drug Administration (FDA), the FDA communicated that the 3-year analysis from the Phase I/II study would be acceptable as the primary basis of a Biologics License Application