FDA requires Phase III trials uniQure(QURE.US) Huntington's disease gene therapy launch faces obstacles, stock plunges approximately 33%

K-LinePoet · 2026-03-12T18:05:33+00:00

Gene therapy company uniQure's Huntington's disease candidate drug AMT-130 failed to obtain accelerated approval from the U.S. FDA and will require Phase III clinical trials, causing the stock price to plunge approximately 33%. The CEO stated that Phase I/II data showed the drug can slow disease progression, but the FDA determined that existing data is insufficient to support a marketing application.

K-LinePoet

2026-03-12 18:05:33

Abstract generation in progress

Gene therapy company uniQure (QURE.US) originally hoped that its Huntington’s disease candidate drug AMT-130 would receive accelerated approval from the U.S. FDA, but this expectation was recently dashed. The FDA explicitly informed the biopharmaceutical company that it needs to conduct a randomized, double-blind, sham-controlled Phase III clinical trial.

Following this news, the company’s stock price closed down about 33% on Monday.

According to a press release from the company, uniQure had previously planned to apply for approval based on data from a Phase I/II study comparing it to external controls. However, the FDA believes that the existing data are not sufficient “as the primary evidence of effectiveness needed to support the BLA submission for AMT-130.”

uniQure stated it will request a Type B meeting with the FDA in the second quarter to discuss the next steps.

In a subsequent conference call, CEO Matthew Kapusta revealed that Phase I/II data showed the drug could slow disease progression by 75% and reduce the decline in overall functional capacity by 60%. However, he also acknowledged that during a pre-BLA meeting held last October, “the FDA indicated that the data submitted at that time… was unlikely to serve as the primary basis for a BLA submission.”

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