uniQure (NASDAQ: QURE) announced Wednesday that the U.S. Food and Drug Administration (FDA) has agreed to accept three-year data from its early-stage Phase I/II trial of AMT-130 as the primary evidence for an accelerated approval submission. The decision marks a significant reversal in the regulatory trajectory for the gene therapy candidate, which had previously faced skepticism from the agency. Shares of uniQure soared as much as 78% in premarket trading following the news, reflecting renewed investor optimism about the therapy's potential to become the first disease-modifying treatment for Huntington's disease.
The company plans to submit a Biologics License Application (BLA) in the third quarter of 2026, setting the stage for a potential near-term U.S. approval. The FDA had earlier indicated that the overall data might be insufficient, with a senior regulator reportedly describing the therapy as a "failed product" late last year. However, after a Type B meeting, the agency changed its stance, allowing uniQure to use the 36-month Phase I/II results as the main support for the filing.
Huntington's disease is a rare, inherited neurodegenerative disorder characterized by progressive motor, cognitive, and psychiatric symptoms. There are currently no approved therapies that can delay or slow the progression of the disease, which affects approximately 75,000 patients in the U.S., EU, and UK. AMT-130 is designed to reduce the activity of the huntingtin gene, which produces the mutant huntingtin protein responsible for the disease. The one-time treatment is delivered via MRI-guided stereotactic neurosurgery directly into the striatum, a brain region critical for movement control.
According to uniQure's September data update, the high dose of AMT-130 slowed disease progression by 75% as measured by the composite Unified Huntington's Disease Rating Scale (cUHDRS), which assesses movement, cognition, and daily living functions, compared to a matched external control group. The therapy also demonstrated a 60% slowing in the decline of Total Functional Capacity, a measure of daily independence. Sarah Tabrizi, a clinical neurology professor at University College London and director of its Huntington's Disease Center, previously called the data "the most convincing in the field to date."
The FDA has requested that uniQure finalize the design of a confirmatory trial before the BLA submission, potentially using standard-of-care patients as controls rather than a sham surgery group. This step is crucial for the accelerated approval pathway, which allows earlier access to therapies for serious conditions with unmet medical needs but requires subsequent verification of clinical benefit. UniQure expects to receive the final meeting minutes from the FDA within the next 30 days and acknowledges that the agency could still determine that the Phase I/II data are insufficient or that post-approval requirements could be costly.
The regulatory shift has broader implications for the Huntington's disease drug development landscape. Competitors such as Novartis, which licensed PTC Therapeutics' oral HTT mRNA splice modulator PTC518 in a deal worth up to $2.9 billion, and Wave Life Sciences, which is developing the allele-selective silencing therapy WVE-003, are also advancing their programs. AMT-130's potential accelerated approval could reshape the competitive dynamics in the space, offering a gene therapy option that targets the root cause of the disease.
CEO Matt Kapusta expressed optimism about the development, stating that the FDA's agreement validates the company's clinical data and opens a clear path forward for the BLA submission. However, he cautioned that the approval process is not guaranteed and that the company must still navigate the confirmatory trial requirements and potential regulatory hurdles.
Market reaction has been overwhelmingly positive, with shares more than doubling from their pre-halt levels. The stock's surge underscores the high stakes for uniQure and the potential for AMT-130 to address a significant unmet medical need in Huntington's disease. Investors will be closely watching the final FDA meeting minutes and the company's progress in finalizing the confirmatory trial design in the coming weeks.
