FDA has reversed course on uniQure’s Huntington’s disease gene therapy, clearing the way for the company to file for US accelerated approval. After a dispute that included scrutiny of the company’s evidentiary base, FDA engagement with uniQure resulted in agreement that an analysis period could support a marketing application. UniQure said it will submit a marketing application in the third quarter for AMT-130 and is pursuing accelerated approval. The decision follows meetings with FDA officials in which the agency deemed uniQure’s three-year analysis acceptable for the filing package, changing the regulatory outlook after prior FDA objections. The update comes as the stock reacted positively, reflecting investor focus on accelerated pathways for rare monogenic CNS disease programs. For the field, it underscores how FDA expectations around acceptable endpoints and follow-up duration can still shift during the pre-approval dialogue.