FDA rejects uniQure AMT-130 Phase I/II data as sufficient for approval filing

Reuters

Mar 02

FDA rejects uniQure AMT-130 Phase I/II data as sufficient for approval filing

uniQure NV said it received final meeting minutes from the U.S. FDA following a Type A meeting held on Jan. 30, 2026 regarding AMT-130 for Huntington’s disease. The FDA stated it cannot agree that Phase I/II data compared to an external control are sufficient to provide the primary evidence of effectiveness needed to support a marketing application, and strongly recommended a prospective, randomized, double-blind, sham surgery-controlled study. uniQure said it will continue engaging with the FDA on Phase III development considerations and plans to request a Type B meeting in the second quarter of 2026 to discuss potential study design approaches.

Disclaimer: This news brief was created by Public Technologies (PUBT) using generative artificial intelligence. While PUBT strives to provide accurate and timely information, this AI-generated content is for informational purposes only and should not be interpreted as financial, investment, or legal advice. uniQure NV published the original content used to generate this news brief via GlobeNewswire (Ref. ID: 202603020700PRIMZONEFULLFEED9663615) on March 02, 2026, and is solely responsible for the information contained therein.

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