UniQure (QURE) said Thursday its experimental gene therapy, AMT-130, received breakthrough therapy designation from the US Food and Drug Administration for the treatment of Huntington's disease, a neurodegenerative disorder, sending shares up nearly 35% in premarket activity.
The company said the decision is based on interim data from an ongoing phase I/II trial, which showed that AMT-130 may slow disease progression in a dose-dependent manner.
The new designation adds to the therapy's existing regenerative medicine advanced therapy designation, orphan drug designation, and fast-track designation, it added.
Breakthrough therapy designation speeds up the development and review of treatments showing early signs of significant benefit for severe conditions.
The company said it expects to deliver an additional regulatory update and guidance on the Biologics License Application submission in the second quarter of 2025.
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