uniQure N.V. (NASDAQ:QURE) shares are rising following a significant development in its efforts to treat Huntington’s disease.

The company has made a key breakthrough with its treatment, AMT-130. The company announced that the FDA has agreed on important elements for an accelerated approval pathway for AMT-130, a potential therapy aimed at slowing the disease’s progression.

The FDA’s decision followed discussions regarding data from ongoing Phase I/II clinical studies. The FDA has confirmed that the data, compared to a natural history control, can be used for the Biologics License Application (BLA), bypassing the need for additional pre-submission studies.

Huntington’s disease is a rare, inherited neurodegenerative disorder that causes progressive motor dysfunction, cognitive decline, and behavioral changes.

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This latest agreement marks a crucial milestone for the Huntington’s disease community, as AMT-130 could soon become a groundbreaking therapy.

The FDA also acknowledged reductions in neurofilament light chain (NfL), a biomarker of neurodegeneration, as supportive evidence of the therapy’s potential benefits.

uniQure continues its efforts in advancing AMT-130, with BLA readiness activities underway and plans to engage further with the FDA in early 2025.

According to Benzinga Pro, QURE stock has gained over 2% in the past year. Investors can gain exposure to the stock via Virtus LifeSci Biotech Clinical Trials ETF (NYSE:BBC).

Price Action: QURE shares are trading higher by 87.8% to $13.70 in the premarket session on Tuesday.

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