UniQure’s AMT-130 Receives FDA Breakthrough Designation for Huntington’s Treatment
uniQure’s investigational gene therapy, AMT-130, has received Breakthrough Therapy designation from the U.S. FDA for the treatment of Huntington’s disease, a rare and inherited neurodegenerative disorder with no approved therapies that alter its progression.
Huntington’s disease is a rare inherited disorder that causes motor problems, behavioural changes, and cognitive decline, leading to progressive physical and mental deterioration.
This recognises the urgent need for effective treatments for Huntington’s disease and the promising interim results from ongoing clinical trials of AMT-130. Data from the Phase I/II trials showed a dose-dependent slowing of disease progression in treated patients over a 24-month period, based on the cUHDRS scale. So far, 45 patients have received AMT-130 in the trials.
This designation is intended to speed up the development and review of treatments for serious conditions, particularly when preliminary evidence suggests that the drug could offer significant improvement over existing therapies. A Breakthrough Therapy designation also provides access to all Fast Track programme benefits, intensive guidance on the development process, and senior-level FDA involvement.
