FDA Defends Call For Placebo Study In uniQure Huntington's Gene Therapy Trial

Core Viewpoint - The FDA has requested a new placebo-controlled study for uniQure N.V.'s gene therapy for Huntington's disease, stating that the existing trial design does not adequately demonstrate the treatment's effectiveness in slowing disease progression [1][2]. Group 1: FDA's Position - The FDA advised against using Phase 1/2 study data as primary evidence for the marketing application of AMT-130, recommending a prospective, randomized, double-blind, sham surgery-controlled study instead [2]. - Historically, the FDA has required randomized, placebo-controlled trials for evaluating therapies for Huntington's disease, and comparisons against external patient datasets are deemed insufficient [3]. Group 2: UniQure's Previous Study - UniQure reported statistically significant results showing a slowing of disease progression over three years using a clinical rating scale, but this was based on comparisons with external datasets rather than a randomized placebo group [4]. - In late 2024, uniQure claimed to have reached an agreement with the FDA on key components for an accelerated approval application, but the FDA clarified that the existing study would not suffice for approval [5]. Group 3: Dispute Over Procedure Description - During a financial results conference call, uniQure's chief medical officer described the FDA's request for a placebo procedure as "superficially drill a hole on the skull," which the FDA official disputed, suggesting a randomized trial with a small scalp incision instead [6]. - A spokesperson for the Department of Health and Human Services criticized uniQure's characterization of the FDA's request on social media, labeling it as false [7]. Group 4: Market Reaction - Following the news, uniQure's shares increased by 18.81% to $10.74, approaching its 52-week low of $7.76 [7].