FDA Wants Additional Study from UniQure for Huntington’s Disease Gene Therapy

Rare Daily Staff

The U.S. Food and Drug Administration advised gene therapy developer UniQure  to run an additional study of its experimental Huntington’s disease treatment, AMT-130, before seeking marketing approval.

The agnecy told UniQure that data from its phase 1/2 studies, which used an external control group for comparison, are not sufficient to demonstrate the therapy’s effectiveness. Instead, the agency recommended a prospective, randomized, double-blind, sham surgery–controlled trial.

UniQure said it plans to request a meeting with the FDA in the second quarter to discuss potential phase 3 study designs and broader development plans for AMT-130.

“While we did not reach alignment on a submission pathway based on the phase 1/2 data, we believe the totality and durability of our data warrant continued substantive dialogue regarding how the FDA’s stated commitment to regulatory flexibility may be appropriately applied in this setting,” CEO Matt Kapusta said.

He said the company remains committed to working with the FDA to define a “clear, scientifically grounded, and efficient path forward” for AMT-130.

Huntington’s disease is a rare, inherited neurodegenerative disorder caused by a mutation in the HTT gene. It typically appears in mid-adulthood and leads to progressive loss of motor control, cognitive decline, and psychiatric symptoms. There are no approved treatments that can halt or reverse the underlying course of the disease, and patients often face a gradual loss of independence over 10 to 20 years.

AMT-130 is designed as a one-time gene therapy delivered directly to the brain using an adeno-associated virus vector. The treatment aims to silence the mutant huntingtin gene that drives the disease process, with the goal of slowing or preventing neuronal damage.

Early clinical data have suggested durable lowering of the target protein and signs of clinical stabilization, but UniQure acknowledged that the small sample size and reliance on external controls limit the conclusions regulators can draw from the current studies.

Photo: Matt Kapusta, CEO of UniQure