UniQure seeks FDA and UK approval for Huntington's gene therapy AMT-130

UniQure has submitted a biologics license application for AMT-130, a gene therapy for Huntington's disease, to the FDA and UK regulators. If the FDA grants priority review, approval could come as early as the second quarter of 2027.

UniQure's regulatory submission positions AMT-130 as a potential first-in-class treatment for Huntington's disease, an inherited condition that currently has no disease-modifying options. The therapy employs a viral vector to deliver genetic instructions that may suppress production of the toxic huntingtin protein responsible for neuronal damage. A priority review designation from the FDA would compress the review timeline, potentially yielding a decision by mid-2027.

The filing arrives amid notable momentum and setbacks in rare neurodegenerative drug development. Ionis recently secured approval for an antisense therapy targeting Alexander disease, while Ultragenyx's gene therapy candidate for Angelman syndrome failed in late-stage testing, underscoring the technical and clinical risks inherent to this therapeutic area. UniQure's path forward will hinge on durability of effect and long-term safety data from its ongoing trials.