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# FDA Wants Randomized Trial for Huntington’s Gene Therapy
- URL: https://www.fdaweb.com/fda-wants-randomized-trial-for-huntingtons-gene-therapy/
- Published: 2026-03-02T12:00:00.000Z
- Updated: 2026-09-14T13:34:40.000Z
- Author: David McFarland
- Tags: Biologics, #legacy-id-D5160739

uniQure says FDA does not agree that data from the company’s Phase 1-2 studies — compared against an external control cohort — are sufficient to constitute the primary evidence of effectiveness required for a BLA for its Huntington’s disease gene therapy AMT-130\. The agency strongly recommended that a prospective, randomized, double-blind, sham surgery-controlled trial be conducted before seeking approval, according to the company’s characterization of meeting minutes from a January Type A meeting with FDA.

The Type A meeting followed a pre-BLA meeting in October when FDA reportedly first questioned whether the earlier trial data were sufficient to support a submission ([see story](https://fdaweb.com/login.php?sa=v&aid=D5160150&cate=&stid=%241%24Ec5.7Y..%24inn0KSYVfFdestv5P54vG.&ref=fdaweb.com)). At the time, the company said the feedback marked a “key shift” from prior communications over the past year, during which the agency had suggested the current data could form the basis for an accelerated approval filing.

UniQure says it is now evaluating Phase 3 development considerations and plans to request a follow-up Type B meeting in the second quarter to further discuss potential study design approaches.

This recent setback comes on the heels of last week’s public comments by FDA commissioner **Marty Makary** during an interview on CNBC ([see story](https://www.fdaweb.com/makary-defends-approval-standards-and-prasads-role/)) where he defended the agency’s recent decisions to reject or withhold approval for several rare disease drug candidates, emphasizing the agency’s obligation to ensure clear clinical benefit before granting marketing authorization. In his remarks, he alluded to therapies involving highly invasive procedures — including drilling a “burr hole” in the skull to deliver treatment — as examples of cases where the agency resisted external pressure to approve despite what he characterized as insufficient evidence of benefit. Although he did not explicitly name AMT-130, the description closely mirrored that investigational therapy’s surgical delivery method.

The comments reverberated through financial markets, where uniQure’s stock experienced sharp declines following the broadcast. Analysts following the coverage highlighted that public commentary from an FDA commissioner about drugs under active review is unusual and underscores broader debate within the agency over evidentiary standards for rare disease therapies. Some industry observers noted that it remains unclear whether Makary’s critique was aimed specifically at AMT-130 or at other products facing similar clinical evidence challenges, with attention also on another rare disease therapy receiving scrutiny.