uniQure Stock Tumbled 55% Today: What’s The FDA Angle?

The company recently met with the FDA and believes that the agency no longer considers the data from an early-to-mid-stage trial of its investigational gene therapy sufficient for approval.

• It is now unclear when the company would apply for approval of the gene therapy, it said.
• uniQure, however, added that it intends to communicate with the FDA to determine a path forward toward approval of the therapy. 
• uniQure CEO termed the meeting outcome “unexpected.”
  

uniQure N.V. (QURE) no longer believes that the U.S. Food and Drug Administration considers data from an early-to mid-stage trial of its investigational gene therapy for Huntington’s disease sufficient for approval, the company said on Monday.

The company recently met with the agency, and although the minutes of the meeting have not been received, the company believes that the FDA no longer considers the data from the AMT-130 trial adequate to provide primary evidence in support of an application for approval.

As a result, it is now unclear when the company would apply for approval of the gene therapy, it said. uniQure, however, added that it intends to communicate with the FDA to determine a path forward toward approval of the therapy.

CEO Reacts

uniQure CEO termed the meeting outcome “unexpected.”

“We are surprised by the FDA’s feedback at the recent pre-BLA meeting, which is a drastic change from the guidance the FDA provided in November 2024 that data from the ongoing Phase I/II studies, compared to a natural history external control, may serve as the primary basis for a BLA submission under the Accelerated Approval pathway,” CEO Matt Kapusta said.

The company also stated that it intends to engage in further communications with other regulatory agencies while working with the FDA.

Expert Take

Stifel analyst Annabel Samimy said that the news regarding the FDA's reversal on the approvability of uniQure's AMT-130 based on early to mid-stage trial data on the surface injects another layer of uncertainty into the workings of the FDA and their receptivity to gene therapy, more specifically.

The analyst, however, does not expect this to impact Regenxbio’s (RGNX) rare disease programs. Meanwhile, Raymond James added that it does not expect the meeting outcome to impact Biohaven (BHVN) either.

How Did Stocktwits Users React?

QURE stock traded 55% lower at the time of writing. On Stocktwits, retail sentiment around QURE stock fell from ‘neutral’ to ‘bearish’ territory over the past 24 hours, while message volume stayed at ‘low’ levels.

QURE stock is up 72% this year and approximately 445% over the past 12 months. 

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