Why Did uniQure Soar 35.14%? FDA Breakthrough Therapy Designation for AMT-130

On April 17, 2025, uniQure's stock surged by 35.14% in pre-market trading, marking a significant rise that has captured the attention of investors and analysts alike.

The surge in uniQure's stock price can be attributed to the recent breakthrough therapy designation granted by the FDA for AMT-130, a gene therapy developed by uniQure for the treatment of Huntington's disease. This designation is based on clinical evidence from Phase I/II trials, which demonstrated meaningful slowing of disease progression. The FDA's decision underscores the potential of AMT-130 to address a critical unmet medical need, as there are currently no approved therapies to delay the onset or slow the progression of Huntington's disease.

UniQure's chief medical officer, Walid Abi-Saab, highlighted the significance of this designation, noting that it reflects both the urgent need for effective treatments and the encouraging interim data from the trials. The company is now poised to work closely with the FDA to expedite the development and review of AMT-130, with the goal of bringing this innovative therapy to patients as quickly as possible.

In addition to the Breakthrough Therapy designation, AMT-130 has also received Regenerative Medicine Advanced Therapy (RMAT) designation, Orphan Drug designation, and Fast Track designation from the FDA. These designations are intended to expedite the development and review of investigational therapeutic candidates that show substantial improvement over available therapies. UniQure is expected to provide additional regulatory updates and guidance on the Biologics License Application submission in the second quarter of 2025.