uniQure seeks U.S. and U.K. approval for Huntington's gene therapy

uniQure has submitted a Biologics License Application to the U.S. Food and Drug Administration (FDA) seeking accelerated approval for ifezuntirgene inilparvovec, also known as AMT-130. This one-time gene therapy is intended to treat the underlying cause of Huntington’s disease, an inherited neurodegenerative disorder for which there is currently no approved therapy to slow disease progression.

The company has also filed a marketing application with the United Kingdom’s Medicines and Healthcare products Regulatory Agency (MHRA). uniQure has requested a priority review from the FDA, which could shorten the review process to approximately six months following the initial 60-day filing review period.

The filings are supported by three-year trial data indicating that the disease progressed more slowly in patients treated with AMT-130 compared to untreated patients. If approved, the therapy would be administered via MRI-guided stereotactic neurosurgery into the brain. While the filing reduces regulatory uncertainty, practical challenges regarding specialized administration centers and insurance coverage remain for patients.