UniQure to seek approval for Huntington’s therapy after FDA U-turn, shares skyrocket

By Kamal Choudhury

June 17 (Reuters) – UniQure said the U.S. health regulator has reversed its earlier position and will accept existing trial data for an accelerated review of its Huntington’s treatment, sending the Dutch drugmaker’s U.S.-listed shares up over 75% to a seven-month high on Wednesday.

The U.S. Food and Drug Administration had indicated late last year that available data was insufficient to support a filing, with one senior regulator describing UniQure’s therapy as a “failed product.”

The regulator has now told the company that a three-year analysis from an early-to-mid-stage trial would be sufficient as the main basis for a marketing application under the accelerated approval pathway, uniQure said. 

Data from the trial published last year showed patients who received a high dose of its therapy, AMT-130, saw a 75% reduction in disease progression.

UniQure plans to submit its application in the third quarter of 2026.

The agency has also agreed to work with uniQure to finalize the design of a required confirmatory study before the filing, including using patients on standard treatment as a comparison instead of a sham procedure.

Earlier this year, the FDA had demanded uniQure run an entirely new clinical trial with one arm of patients receiving a sham surgery, a fake version of the real procedure.

The agency’s proposal drew scrutiny because some patients would undergo invasive brain surgery, including drilling small holes into the skull, without receiving the actual therapy, raising ethical concerns among patient groups and experts.

Guggenheim analyst Debjit Chattopadhyay said the latest decision potentially reflects the new FDA’s appreciation of the challenges in rare disease following the overhaul of its leadership.

Former commissioner Marty Makary and controversial vaccines chief Vinay Prasad exited the agency earlier this year.

“Given Vinay Prasad’s and Marty Makary’s public opinion on this filing and CRL issuance and now the positive uniQure regulatory update for AMT-130, we believe the current FDA, largely in caretaker mode, appears to be more flexible on regulatory paths for applications where concerns were previously raised,” said William Blair analyst Myles Minter.

The agency appears to be trying to repair relations with the rare-diseases sector, with acting FDA Commissioner Kyle Diamantas earlier this month meeting with rare disease advocacy groups in what one attendee called a “breath of fresh air.” 

Huntington’s disease is a fatal inherited neurodegenerative disorder with no approved therapies that slow progression.

(Reporting by Kamal Choudhury in Bengaluru; Editing by Jonathan Ananda and Sahal Muhammed)