uniQure: FDA says Phase I/II data unlikely to support AMT-130 BLA

FDA meeting minutes confirm that the Phase I/II data are currently unlikely to serve as primary evidence for a BLA submission. uniQure will request a follow-up FDA meeting in Q1 2026 to discuss next steps for the AMT-130 Huntington's disease program.

Key facts

Filed: Dec 4, 2025, 12:06 PM
Event: Other
Direction: Negative
Company: QUREuniQure
Asset: AMT-130
Source: SEC 8-K
Confidence: Sourced

Source excerpt

EX-99.1 2 qure-20251204xex99d1.htm EX-99.1 Exhibit 99.1 uniQure Provides Regulatory Update on AMT-130 for Huntington ’ s Disease Lexington, MA and Amsterdam, the Netherlands, December 4, 2025 — uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that the company received final meeting minutes from the U.S. Food and Drug Administration (FDA) regarding a pre-Biologics License Application (BLA) meeting held on October 29, 2025 to discuss the application for AMT-130, an investigational gene therapy for Huntington ’ s disease (HD). In the final meeting minutes, and consistent with uniQure ’ s November 3, 2025 press release, the FDA conveyed that data submitted from the Phase I/II studies of AMT-130 are currently unlikely to provide the primary evidence to support a BLA submission. uniQure is carefully evaluating the feedback and plans to urgently request a follow-up meeting with the FDA to take place in the first quarter of 2026. “ We are committed to collaborating with the FDA to advance AMT-130 to patients and their families as rapidly as possible, ” said Matt Kapusta, chief executive officer at uniQure. “ The support we have seen these last weeks from the Huntington ’ s disease community, including patients, families, caregivers, clinicians and advocates reinforces the urgency of the unmet need in Huntington ’ s disease. ” About uniQure uniQure is delivering on the p

SEC 8-K