UniQure Stock Soars 38.55% on FDA Breakthrough Therapy Designation

On April 18, 2025, uniQure's stock surged by 38.55% in pre-market trading, marking a significant rise for the biotechnology company.

UniQure's stock price experienced a notable increase following the FDA's grant of Breakthrough Therapy designation to AMT-130, a treatment for Huntington's disease. This designation was based on clinical evidence from phase I/II trials, which demonstrated a meaningful slowing of disease progression. The designation is in addition to the Regenerative Medicine Advanced Therapy (RMAT) designation, Orphan Drug designation, and Fast Track designation, all previously granted by the FDA to AMT-130.

Huntington's disease is a rare, inherited neurodegenerative disorder for which there are currently no disease-modifying therapies available. The Breakthrough Therapy designation is expected to expedite the development and review of AMT-130, potentially bringing a new treatment option to patients suffering from this debilitating condition.