Published by Global Banking & Finance Review®
Posted on March 2, 2026
2 min readLast updated: April 2, 2026
Add as preferred source on GoogleThe FDA has informed uniQure that its Phase I/II gene therapy data for Huntington’s disease (AMT‑130) are not sufficient to support a marketing application, sending the company’s shares sharply lower.
By Puyaan Singh and Christy Santhosh
March 2 (Reuters) - UniQure said the U.S. drug regulator called for a new study to support the approval of its gene therapy for a brain disorder as the data from a previous trial was insufficient, sending the Dutch drugmaker's U.S.-listed shares more than 36% lower on Monday.
Shares have been under pressure since November, when the U.S. Food and Drug Administration said the data likely would not support an accelerated approval application.
The company's drug for Huntington's disease, named AMT‑130, is administered after neurosurgery directly into the brain's striatum.
The FDA strongly recommended to uniQure a study that randomly assigns patients to get either the therapy or a sham procedure, which may "superficially drill a hole on the skull," said medical chief Walid Abi-Saab on Monday.
This is risky as these patients would not receive AMT-130 while their disease worsens, he said.
FDA Commissioner Dr. Marty Makary last week criticized a drug requiring a "burr hole" in the skull, without naming it or the company, but Wall Street analysts feared the comment referred to AMT‑130.
Leerink analyst Joseph Schwartz said that it seems contradictory for the FDA to be concerned about the morbidity associated with burr holes but still want a sham control arm.
Huntington's disease is a fatal inherited neurodegenerative disorder with no approved therapies that slow progression.
Last year, the company reported trial data showing AMT-130 slowed progression by 75%.
PATH FORWARD WILL LIKELY TAKE YEARS
Wall Street analysts pointed to ethical concerns about the FDA-recommended study and warned that the delay could cost the company its first-mover advantage to rivals developing late‑stage candidates, such as PTC's votoplam, an oral drug that does not require neurosurgery.
A study would likely be costly and a data readout would be multiple years away, said Barclays analyst Eliana Merle.
(Reporting by Puyaan Singh in Bengaluru; Editing by Shilpi Majumdar and Sahal Muhammed)
The FDA informed uniQure that early- to mid-stage trial data was insufficient to support a marketing application for its gene therapy for Huntington's disease.
UniQure's shares slumped nearly 44% in premarket trading after the FDA's feedback.
The FDA recommended that uniQure conduct a new study randomly assigning patients to receive the gene therapy or a sham surgery.
The results from existing studies, when compared to an external control, cannot serve as primary evidence of effectiveness required by the FDA.
Huntington's disease is a fatal, inherited neurodegenerative disorder causing progressive breakdown of nerve cells in the brain.
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