uniQure Moves Closer to Potential Huntington's Therapy Approval With FDA Type A Meeting on AMT-130 BLA Submission

uniQure N.V. (QURE) has secured a significant regulatory milestone, with the FDA scheduling a Type A meeting to review the Biologics License Application data for AMT-130, the company’s experimental one-time gene therapy targeting Huntington’s disease. This consultation marks a critical step toward potential accelerated approval of a treatment addressing a devastating neurodegenerative condition for which no disease-modifying options currently exist.

Understanding the Disease and Unmet Need

Huntington’s disease remains one of the most challenging neurodegenerative disorders, defined by progressive motor dysfunction, cognitive deterioration, and psychiatric symptoms that culminate in early mortality. The absence of approved treatments that can modify disease progression underscores the profound unmet need in the patient population affected by this genetic condition.

How AMT-130 Works and Clinical Promise

AMT-130 represents an innovative approach utilizing one-time adeno-associated virus (AAV) delivery to introduce microRNA sequences directly into neural tissue. The therapy is engineered to silence the huntingtin gene (HTT), the genetic driver behind disease progression. Preliminary clinical evidence indicates that AMT-130 can reduce levels of mutant huntingtin protein, suggesting the potential to decelerate or arrest disease advancement.

Regulatory Momentum Building

The development trajectory has accelerated significantly in recent months. uniQure secured Breakthrough Therapy designation from the FDA in April 2025 following Phase 1/2 data analysis against external comparators. Prior to this, the company had already obtained Regenerative Medicines Advanced Therapy (RMAT) designation in May 2024, both designations reflecting recognition of the therapy’s capacity to address serious therapeutic gaps.

Financial Position and Market Response

The Type A meeting announcement has energized market sentiment around the biotech company. QURE shares have demonstrated volatility over the trailing twelve-month period, trading within a range of $7.76 to $71.50. In pre-market activity following the announcement, stock price reached $26.88, representing a 16.11% gain. The company maintains a solid financial foundation with approximately $694.2 million in cash, cash equivalents, and investment holdings as reported in the most recent quarterly filing, positioning uniQure to advance AMT-130 through the regulatory pathway ahead.

The Type A meeting represents a collaborative discussion between the company and regulators to finalize the BLA package contents before formal submission, a process that could accelerate the timeline toward a potential approval decision for this novel gene therapy approach.