uniQure Soars After FDA's U-Turn Opens Accelerated Approval Pathway for Huntington's Therapy

Shares of uniQure N.V. (NASDAQ:QURE) pumped around 78% on Wednesday after the FDA cleared the path for a uniQure AMT-130 FDA approval filing under the accelerated pathway — a dramatic reversal from the agency’s prior stance that had sent the stock to a 52-week low of $8.73.

What Happened: The AMT-130 FDA Approval Path Opens

During a recent Type B meeting with the FDA, regulators communicated that the 3-year analysis from uniQure's Phase I/II clinical study of AMT-130 is acceptable as the primary basis for a Biologics License Application under the accelerated approval pathway. The company intends to submit the BLA in the third quarter of 2026.

These are the same AMT-130 data that were previously concluded as insufficient to support a marketing application. That shock had sent QURE shares to a 52-week low of $8.73. Wednesday's announcement means the regulatory overhang that has haunted the stock for months has been fully lifted.

The FDA also asked to finalize the design of the required confirmatory study before the BLA is filed, including the potential use of concurrent controls on standard-of-care therapy instead of a sham procedure — a notable shift in trial expectations for this rare neurodegenerative disorder. 

"Today's announcement reflects the outcome we have worked toward throughout our continued regulatory engagement with FDA, and we are deeply grateful for FDA's genuine commitment to addressing the unmet need of Americans living with Huntington's disease".
— Matt Kapusta, CEO, uniQure

What Is uniQure AMT-130 and Why Does FDA Approval Matter?

AMT-130 has received RMAT, Breakthrough Therapy, and Fast Track designations — the first RMAT designation ever granted for Huntington's disease — reflecting the therapy's potential effect for an unmet condition.

The 3-year data showed a statistically 75% slowing of disease progression at the high dose and was generally well-tolerated, with a manageable safety profile.

A Broader FDA Tailwind For The Whole Sector

The catalyst behind the uniQure AMT-130 FDA approval clearance is a broader shift in the regulatory backdrop. The FDA issued draft guidance aimed at accelerating cell and gene therapies. The guidance shows how sponsors can build on existing science, reducing redundant testing and accelerating treatments for patients with rare and life-threatening diseases

Wall Street Responds: Cantor Fitzgerald Triples Its Price Target

Cantor Fitzgerald upgraded QURE from Neutral to Overweight, raising the price target to $61 from $18 per share. Barclays restated a Positive rating on shares of uniQure on the same day. The consensus analyst price target now stands at approximately $47.17, with a 12-to-1 Buy-to-Hold ratio among covering firms.

Shares of QURE traded up $20.62 during mid-day trading on Wednesday, reaching $47.61. The stock had a trading volume of 11,513,314 shares, compared to its average volume of 2,641,930 — a 4.4x surge suggesting a combination of institutional buying and short-seller capitulation.

Earnings:

UniQure last announced its quarterly earnings on May 5, 2026. The company reported ($0.85) EPS for the quarter, beating estimates of $0.88 by $0.03. The firm had revenue of $3.56 million during the quarter, compared to analyst estimates of $5.21 million. Analysts expect uniQure N.V. will post -$3.64 EPS for the current fiscal year.

Risks

Accelerated approval is not unconditional. The FDA requires a confirmatory study to validate clinical benefit, and any future disagreement over design — or failure to demonstrate benefit — could result in withdrawal. Insiders sold 85,817 shares worth approximately $2.2M over the past 90 days, a relatively huge figure given the company’s post-spike market cap, but worth monitoring given that two director sales occurred just days before the FDA announcement.

Key Milestones To Check

uniQure N.V. also has a parallel UK regulatory track, having held a Pre-Submission Meeting with the MHRA and planning a Marketing Authorization Application for AMT-130 for the treatment of Huntington's disease in the third quarter of 2026.

This article is for informational purposes only and does not constitute investment advice.

Benzinga Disclaimer: This article is from an unpaid external contributor. It does not represent Benzinga’s reporting and has not been edited for content or accuracy.