uniQure N.V. (NASDAQ:QURE) surged around 15% in early trading on Friday after the company said it has secured a Type A meeting with the U.S. Food and Drug Administration to discuss its Biologics License Application for AMT-130.
The scheduled meeting will centre on the data package supporting a potential accelerated approval pathway for AMT-130, uniQure’s experimental gene therapy for Huntington’s disease. The development marks a meaningful regulatory milestone for a treatment aimed at a condition with few therapeutic options.
“We look forward to a constructive discussion with the FDA as we work toward a timely resolution regarding an accelerated approval pathway for AMT-130,” said Matt Kapusta, chief executive officer at uniQure. “The Huntington’s disease community, including patients and clinicians, has emphasized the profound unmet medical need and the importance of timely access to potentially disease-modifying therapies such as AMT-130.”
uniQure said it remains focused on enabling patient access while continuing to work closely with the regulator, and plans to provide a further update once official minutes from the meeting are received.
Huntington’s disease is a rare inherited neurodegenerative condition characterised by the gradual loss of nerve cells in the brain, leading to movement difficulties, cognitive impairment and psychiatric symptoms. There are currently no approved treatments that can slow or stop the progression of the disease.