---
schema_version: "secwatch.filing_event.v1"
accession: "0001558370-24-012941"
form_type: "8-K"
ticker: "QURE"
cik: "0001590560"
company_name: "uniQure N.V."
filed_at: "2024-09-23T23:59:59+00:00"
generated_at: "2026-05-31T02:26:31.544480+00:00"
event_type: "regulatory"
sentiment: "positive"
materiality_score: 0.65
calibrated_materiality_score: 0.65
confidence: "high"
source: SEC EDGAR
---

# uniQure receives FDA Orphan Drug Designation for AMT-191 gene therapy for Fabry disease

## Summary
- FDA granted Orphan Drug Designation to AMT-191 for Fabry disease on Sep 23, 2024.
- AMT-191 is a one-time IV AAV5-based gene therapy delivering a GLA transgene.
- First patient dosed in Aug 2024 in a U.S. multi-center open-label Phase I/IIa trial.
- Designation provides tax credits, fee waivers, and 7-year US market exclusivity if approved.
- Initial Phase I/IIa clinical data expected in 2025.

## SEC filing metadata
- accession: 0001558370-24-012941
- form_type: 8-K
- ticker: QURE
- cik: 0001590560
- company_name: uniQure N.V.
- filed_at: 2024-09-23T23:59:59+00:00
- event_type: regulatory
- sentiment: positive
- materiality_score: 0.65
- calibrated_materiality_score: 0.65
- confidence: high
- sec_items: 7.01, 9.01
- EDGAR index: https://www.sec.gov/Archives/edgar/data/1590560/000155837024012941/0001558370-24-012941-index.htm
- EDGAR primary document: https://www.sec.gov/Archives/edgar/data/1590560/000155837024012941/qure-20240923x8k.htm

## Machine-readable alternates
- HTML: https://secwatch.observer/filing/0001558370-24-012941
- JSON: https://secwatch.observer/filing/0001558370-24-012941.json
- Plain text: https://secwatch.observer/filing/0001558370-24-012941.txt

This AI-assisted summary is a reading aid. Review the linked SEC EDGAR filing before relying on any specific claim.