uniQure’s AMT-130 gene therapy for Huntington’s disease has received Breakthrough Therapy designation from the FDA, adding to its existing RMAT, Orphan Drug, and Fast Track designations. This designation is based on Phase I/II trial data showing a dose-dependent slowing of disease progression compared to natural history, measured by the cUHDRS scale, in 45 treated patients. The FDA intends for the Breakthrough Therapy designation to accelerate the development and review of promising treatments for serious conditions.
This designation is crucial for advancing a potential treatment for Huntington’s disease, a devastating neurodegenerative disorder currently lacking disease-modifying therapies. It signifies the FDA’s recognition of AMT-130’s potential based on promising early clinical data, offering hope for patients and families affected by this debilitating condition. The expedited review process associated with the designation could significantly shorten the time it takes for AMT-130 to reach patients if further trials are successful.
AMT-130 has demonstrated dose-dependent slowing of Huntington’s disease progression after 24 months in ongoing Phase I/II trials. This is notable considering the progressive and irreversible nature of the disease. The Breakthrough Therapy designation affords uniQure intensive FDA guidance and involvement of senior managers, facilitating a potentially more efficient development program. A Biologics License Application (BLA) submission guidance and update are expected in the second quarter of 2025.
This Breakthrough Therapy designation represents a major step forward for AMT-130 and for the Huntington’s disease community. While further clinical data are needed to confirm the initial findings, the designation increases the likelihood of an expedited regulatory pathway, potentially bringing this much-needed therapy to patients sooner. This development also strengthens uniQure’s position in the gene therapy field, showcasing its capability to develop innovative treatments for severe neurological conditions.
Jon Napitupulu is Director of Media Relations at The Clinical Trial Vanguard. Jon, a computer data scientist, focuses on the latest clinical trial industry news and trends.
