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uniQure Huntington’s disease gene therapy regulatory filings

Updated 2 times since CLSTR started tracking revisions of this situation.

What changed

2026-09-30 15:13 UTC → 2026-10-05 01:41 UTC · added removed

uniQure has initiated regulatory approval processes for AMT-130, a gene therapy designed to treat the underlying cause of Huntington’s disease. The company submitted a Biologics License Application to the U.S. Food and Drug Administration (FDA) requesting accelerated approval and a priority review, while also filing a marketing application with the United Kingdom’s Medicines and Healthcare products Regulatory Agency (MHRA). Updated Phase I/II clinical data through the 4-year mark indicates that the therapy continues to show a slowing of disease progression. progression, though recent results are mixed. While 36-month data showed high-dose patients demonstrated an 80% slowing of decline on the Composite Unified Huntington’s Disease Rating Scale (cUHDRS) and a 67% slowing on Total Functional Capacity (TFC), the 48-month analysis of 12 high-dose patients showed a 44% slowing of decline on the Composite Unified Huntington’s Disease Rating Scale (cUHDRS) cUHDRS that did not reach statistical significance, significance (p-value of 0.144). However, the Total Functional Capacity (TFC) TFC metric showed a 61% slowing at 48 months. This follows earlier 36-month data where high-dose patients demonstrated an 80% slowing of cUHDRS decline and a 67% slowing on TFC compared to a natural-history cohort. Following the validation of its marketing authorization application by the MHRA, uniQure expects an acceptance decision from the FDA during the fourth quarter. The company also quarter and plans to begin screening patients for a confirmatory study before the end of the year. Despite the nuanced long-term data, Barclays has maintained a buy recommendation for the company. Financially, uniQure reported a revenue increase to 5.84 million USD in the second quarter of 2026, while its GAAP net loss expanded to 81.06 million USD.

Versions

  1. 2026-10-05 01:41 UTC uniQure Huntington’s disease gene therapy regulatory filings
  2. 2026-09-30 15:13 UTC uniQure Huntington’s disease gene therapy regulatory filings
  3. 2026-09-29 20:53 UTC uniQure Huntington’s disease gene therapy regulatory filings

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