Study summary
After reversing course, the FDA accepted an accelerated-approval filing for the gene therapy
What was tested
The FDA review path for uniQure's AMT-130 gene therapy for Huntington disease.
What they found
- In March 2026 the FDA said external-control data were not enough and asked for a sham-controlled trial.
- In June 2026 it reversed, allowing an accelerated-approval filing with a confirmatory trial against standard care.
- uniQure submitted the application in 2026 and requested priority review.
What it means
A decision could come in 2027, which would be the first disease-modifying approval for Huntington disease.
Last reviewed 2026-11-15 by the Find a Trial editorial team.
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