March 2 (Reuters) – UniQure said on Monday the U.S. drug regulator had informed that data from an early- to mid-stage trial of its brain disorder gene therapy would not support its approval application, sending its shares down 40% in premarket trading.
The Dutch company was testing its gene therapy AMT-130 in patients with Huntington’s disease — a fatal, inherited neurodegenerative disorder causing progressive breakdown of nerve cells in the brain. Currently, there are no approved treatments to slow the disease’s progression.
UniQure said the U.S. Food and Drug Administration recommended it to run a new study that randomly assigns patients to get either the gene therapy, or a sham surgery.
In September, data from the early- to mid-stage study showed a 75% reduction in disease progression in a three-year analysis, based on a widely used clinical scale.
The regulator “seems to be devoid of disease state expertise, especially given its leadership biases against cell and gene therapy,” said Guggenheim analyst Debjit Chattopadhyay.
“FDA leadership speaks eloquently on regulatory flexibility… actions reflect a premeditated bias. Path forward for AMT-130 remains murky to say the least,” Chattopadhyay said.
UniQure had initially planned to submit a marketing application to the FDA in early 2026, with hopes of launching the therapy later in the year following potential approval.
“Totality and durability of our data warrant continued substantive dialogue regarding how the FDA’s stated commitment to regulatory flexibility may be appropriately applied in this setting,” said CEO Matt Kapusta.
The company said it would continue engaging with the FDA regarding late-stage development considerations and plans to request a meeting in the second quarter of 2026 to further discuss potential study design approaches.
(Reporting by Puyaan Singh in Bengaluru; Editing by Shilpi Majumdar)
