By Christy Santhosh
(Reuters) -UniQure said the U.S. Food and Drug Administration no longer agreed that clinical data for the Dutch company’s gene therapy for Huntington’s disease may be adequate for a marketing application, triggering a 57% slump in its U.S.-listed shares.
Monday’s update follows feedback received during a recent meeting with the health regulator, the company said.
The agency advised UniQure that the data would likely be not adequate to serve as the primary basis for a marketing application, also called the Biologics License Application, under the accelerated approval pathway.
“We are surprised by the feedback, which is a drastic change from the guidance the FDA provided in November 2024,” UniQure CEO Matt Kapusta said in a statement.
The FDA did not immediately respond to a Reuters request for comment.
The company plans to “urgently engage” with the agency to determine what additional analyses or studies may be needed to advance the therapy, AMT-130, toward a potential filing.
On September 24, UniQure reported trial data that showed AMT-130 had slowed progression of the brain disorder by 75%, helping shares triple in value.
Analysts on Monday described the development as a major regulatory setback.
Guggenheim analyst Debjit Chattopadhyay said it shows that the FDA now views the statistical analysis as “post hoc” while Cantor Fitzgerald analyst Kristen Kluska said it makes the timing of a BLA submission uncertain, as it is not clear if more trials or data will need to be collected.
UniQure had planned to submit the marketing application in early 2026 and hoped to launch the therapy later that year, if approved.
Huntington’s disease is a fatal genetic disorder that causes the progressive breakdown of nerve cells in the brain, leading to movement, cognitive and psychiatric problems.
In an early-to-mid-stage study, patients who received a high dose of AMT-130 showed a 75% reduction in disease progression in a three-year analysis, based on a widely used clinical scale.
(Reporting by Christy Santhosh in Bengaluru; Editing by Krishna Chandra Eluri and Sahal Muhammed)
