> ## Content Index
> Fetch the complete content index at: https://www.fdaweb.com/llms.txt
> Use this file to discover other available public pages before exploring further.

# Advocacy Push Precedes FDA Meeting on Stalled Gene Therapy
- URL: https://www.fdaweb.com/advocacy-push-precedes-fda-meeting-on-stalled-gene-therapy/
- Published: 2026-01-26T12:00:00.000Z
- Updated: 2026-09-14T15:31:31.000Z
- Author: David McFarland
- Tags: Biologics, #legacy-id-D5160543

A coordinated advocacy campaign by the Huntington’s disease community helped set the stage for a high-priority meeting between FDA and uniQure to discuss the regulatory path forward for the company’s experimental gene therapy AMT-130.

UniQure said earlier this month that it had secured a Type A meeting with, a designation reserved for urgent issues that present significant obstacles to drug development. The meeting is intended to address next steps for AMT-130, an investigational gene therapy being studied as a potential disease-modifying treatment for Huntington’s disease.

In November, UniQure said FDA had indicated during a recent pre-BLA meeting that the agency may no longer view the company’s Phase 1-2 data for its Huntington’s disease (HD) therapy AMT-130, analyzed against an external control, as sufficient to serve as primary evidence to support a planned submission.

The company said at the time that the feedback marks 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. As a result, the timeline for a BLA submission is now unclear, it said.

In September, the company reported favorable data from the study. The study met its prespecified primary endpoint, with high-dose AMT-130 demonstrating a statistically significant (75%) slowing of disease progression as measured by the composite Unified Huntington’s Disease Rating Scale at 36 months compared to a propensity score-matched external control, according to the company.

The Type A meeting follows an unusually large grassroots mobilization by patients, families, and advocacy groups after regulatory setbacks for the program. Over the past two months, two online petitions urging the FDA to reconsider its position on AMT-130 gathered more than 48,000 signatures, according to advocacy organizers. On 1/22, representatives from several Huntington’s disease organizations — including Help 4 HD International, the Huntington’s Disease Youth Organization, the Huntington’s Disease Society of America, HD Reach, and the Huntington’s Disease Foundation — traveled to FDA headquarters in Silver Spring, MD, to hand-deliver printed copies of the petitions to agency officials.

Advocates also contacted members of Congress and held meetings with lawmakers to emphasize the lack of approved disease-modifying therapies for Huntington’s disease, a fatal, inherited neurodegenerative disorder.

UniQure has said it plans to include a representative of the Huntington’s disease community in the Type A meeting, allowing patient perspectives to be presented directly to FDA regulators.