Talk Bio

On March 2, 2026, uniQure announced that the FDA deems Phase 1/2 data from its single-arm trial of AMT-130 insufficient for marketing approval, recommending a prospective, randomized, double-blind, sham surgery-controlled study 1 2 3 4 5 AMT-130 is an investigational one-time gene therapy delivered via brain surgery to lower mutant huntingtin protein levels in Huntington's disease patients 2 3 5 Following a January 30, 2026 Type A meeting, uniQure plans to request another FDA meeting in Q2 2026 to discuss Phase 3 study designs and alternative paths 1 3 4 5 FDA Commissioner Marty Makary appeared to criticize AMT-130 in a recent CNBC interview, referencing skull-drilling procedures without clear benefit 4 Despite positive three-year data showing slowed disease progression, analysts note FDA's stance contrasts with promised flexibility for rare diseases 3 4 6 Sources: 1. https://www.statnews.com/2026/03/02/uniqure-huntingtons-disease-fda-blocked/ 2. https://en.hdbuzz...