uniQure: Hindi na sumasang-ayon ang FDA na sapat ang Phase I/II data para sa AMT-130 BLA

Binawi ng FDA ang dating patnubay at ngayon ay nagsasaad na ang Phase I/II data kumpara sa external control ay maaaring hindi sapat bilang pangunahing ebidensya para sa BLA submission. Hindi na malinaw ang timing ng planadong BLA filing para sa AMT-130. Hahanapin ng uniQure ang final meeting minutes at agarang follow-up discussions sa FDA upang matukoy ang landas pasulong.

Mahahalagang fact

Na-file: Nob 3, 2025, 12:06 PM
Kaganapan: Iba pa
Direksyon: Negatibo
Kumpanya: QUREuniQure
Asset: AMT-130
Pinagmulan: SEC 8-K
Kumpiyansa: May source

Sipi mula sa source

EX-99.1 2 qure-20251103xex99d1.htm EX-99.1 Exhibit 99.1 uniQure Provides Regulatory Update on AMT-130 for Huntington ’ s Disease Lexington, MA and Amsterdam, the Netherlands, November 3, 2025 — uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that it received feedback from the U.S. Food and Drug Administration (FDA) during a recent pre-Biologics License Application (BLA) meeting regarding AMT-130, an investigational gene therapy for Huntington ’ s disease (HD). Though final meeting minutes have not yet been received, based on the discussions at the meeting, uniQure believes that the FDA currently no longer agrees that data from the Phase I/II studies of AMT-130 in comparison to an ex

SEC 8-K