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• In March, the FDA said the data were not sufficient for BLA approval and recommended a larger randomized study.
• uniQure said it plans to submit the BLA in the third quarter of 2026.
• The regulatory alignment comes after a leadership reshuffle at the FDA last month.

Shares of uniQure (QURE) surged 70% in pre-market trading on Wednesday, after the firm’s biologics license application for accelerated approval of Huntington’s disease therapy got a significant regulatory boost.

At the time of writing, QURE stock was trading at its highest levels in over seven months.

FDA Reopens The Door For AMT-130

On Wednesday, uniQure said that the U.S. Food and Drug Administration (FDA) is willing to consider three-year data from its Phase I/II study as the primary basis for a BLA of AMT-130, marking a positive shift for the company after the FDA indicated in March that the available early-stage data were not sufficient for approval and recommended a larger randomized study.

uniQure said it plans to submit the BLA in the third quarter of 2026. Approval of the BLA would allow uniQure to market the therapy.

AMT-130 has already received Regenerative Medicine Advanced Therapy (RMAT), Breakthrough Therapy, and Fast Track designations from the FDA.

QURE’s AMT-130 Plans Beyond U.S.

Recently, uniQure announced plans to seek approval for AMT-130 in the U.K. later this year. The company expects to submit its application to the MHRA in the third quarter of 2026 following positive regulatory discussions. The filing will be supported by three-year trial data showing a 75% slowing of disease progression.

FDA Alignment After Leadership Shakeup

After the FDA’s March rejection, Wells Fargo suggested that AMT-130’s regulatory prospects could improve following Vinay Prasad’s departure from the agency. Prasad was a controversial figure who faced criticism over several high-profile drug review decisions.

Following a leadership reshuffle at the FDA last month, Karim Mikhail was named Acting Director of the Center for Biologics Evaluation and Research (CBER).

QURE Stock: Retail Turns Bullish

Retail sentiment surrounding QURE on Stocktwits flipped to ‘bullish’ from ‘bearish’ a day earlier.

Following the FDA update, one user said the stock could be worth $120.

However, another user called it an “overreaction,” stating that the drug has not been approved yet.

The stock has gained nearly 10% so far this year.

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