UniQure's Huntington's Gene Therapy Faces FDA Hurdles, Casting Doubt on Approval Timeline

UniQure, a Netherlands-based biopharmaceutical company, has encountered significant regulatory challenges in its pursuit of approval for AMT-130, a potentially groundbreaking gene therapy for Huntington's disease. Recent communications with the U.S. Food and Drug Administration (FDA) have cast doubt on the therapy's path to market, leading to a sharp decline in investor confidence and the company's stock price.

FDA Feedback Signals Longer Regulatory Road

In a surprising turn of events, UniQure revealed that the FDA no longer considers the data from its Phase I/II studies of AMT-130 sufficient to support a Biologics License Application (BLA). This revelation came during a pre-BLA meeting with the agency in late October, with final minutes confirming the challenging outlook.

Matt Kapusta, CEO of UniQure, stated, "We are committed to collaborating with the FDA to advance AMT-130 to patients and their families as rapidly as possible." The company plans to urgently request a follow-up meeting with the FDA in the first quarter of 2026 to address the agency's concerns and chart a path forward.

Clinical Data and Market Implications

Despite the regulatory setback, AMT-130 has shown promising results in clinical trials. Data from a pivotal Phase I/II trial demonstrated that the gene therapy slowed disease progression by 75% over three years, surpassing analyst expectations. Sarah Tabrizi, director of the University College London Huntington's Disease Center, emphasized the significance of these findings, stating, "That means for one year of disease progression, they will have four years longer in terms of disease-free lives. It's a huge effect size, and will have massive effects on patients' lives."

The therapy's mechanism of action involves delivering genetic material to brain cells to inhibit the production of the mutated protein responsible for Huntington's disease. If approved, AMT-130 would represent the first genetic treatment for this devastating neurodegenerative condition.

Market Response and Analyst Perspectives

UniQure's stock has experienced significant volatility in response to these developments. Following the initial disclosure of the FDA's feedback in November, the company's share price plummeted by 65%. The latest update caused a further decline, with shares falling over 16% at market open on December 4, 2025, before recovering slightly to around $23 by mid-morning.

Analysts remain cautiously optimistic about AMT-130's future, despite the setbacks. Paul Matteis of Stifel noted that multiple scenarios are still possible, ranging from an FDA reversal to a compromise involving additional patient data. Joseph Schwartz of Leerink Partners expressed hope, stating, "We remain cautiously optimistic that AMT-130 can find its way to market on the basis of the existing data to date in some form."

The pharmaceutical industry and Huntington's disease community are closely watching UniQure's next moves, as the outcome of this regulatory process could have far-reaching implications for gene therapy development and approval pathways for rare diseases.