UniQure’s AMT-130 Receives FDA Breakthrough Therapy Designation for Huntington’s Disease
Saturday, April 19, 2025
uniQure N.V, a biotechnology company focused on developing gene therapies for serious medical conditions, has announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to its gene therapy candidate AMT-130. This investigational therapy is being developed for the treatment of Huntington’s disease, a rare genetic brain disorder for which no disease-modifying treatments currently exist.
The Breakthrough Therapy designation adds to the list of regulatory recognitions already received by AMT-130, which includes the Regenerative Medicine Advanced Therapy (RMAT) designation, Orphan Drug designation, and Fast Track designation, all previously granted by the FDA.
The latest designation is based on interim clinical data from ongoing Phase I/II studies. As of July 2024, findings showed that AMT-130 may help slow the progression of Huntington’s disease in a dose-dependent manner, as observed in treated patients when compared to historical data. So far, 45 patients have been treated with AMT-130 in these clinical trials.
The Breakthrough Therapy designation is intended to speed up the development and review of treatments for serious conditions where early clinical data suggests significant improvement over existing options. It also allows for closer collaboration with the FDA, including guidance from senior regulatory officials.
Huntington’s disease is a rare inherited disorder caused by a mutation in the huntingtin gene. This condition leads to the build-up of abnormal proteins in the brain, resulting in movement issues, changes in behaviour, and cognitive decline. The disease typically worsens over time and has a significant impact on both physical and mental health. An estimated 70,000 people in the United States and Europe are currently living with Huntington’s disease, and many more are at risk of developing it. Despite its known genetic cause, there are still no approved treatments that can delay or slow the disease’s progression.
Source: globenewswire.com
