一次情報の記録
重要申請提出
uniQure、Huntington病向けAMT-130のBLA提出を計画
FDAは、3年間のPhase I/IIデータが加速承認を求めるBLAの主要根拠となり得ることを確認した。同社は2026年第3四半期にBLAを提出する予定であり、提出前にFDAと確認試験のデザインについて合意する必要がある。
主要事実
出典の抜粋
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