AMT-130, uniQure’s investigational treatment for Huntington’s Disease, reaches an important regulatory milestone

Regulatory applications have been submitted in the United States of America and the United Kingdom

UniQure announced on the 2nd of September, 2026 that it has submitted applications seeking regulatory approval for AMT-130, its investigational gene therapy for Huntington’s disease, to both the U.S. Food and Drug Administration (FDA) and the UK Medicines and Healthcare products Regulatory Agency (MHRA).

The submissions are supported by three-year data from the Phase I/II clinical program, in which uniQure reported a slowing of disease progression compared with a matched external control group from Enroll-HD. AMT-130 remains an investigational therapy, and these submissions do not mean that the treatment has yet been approved.

Reaching the regulatory review stage is nevertheless an important milestone after years of research, clinical trial participation and commitment from researchers, clinicians, people with HD and their families. We now await the assessments and decisions of both the FDA and MHRA.

At this time, uniQure’s announcement relates specifically to regulatory submissions in the United States and the United Kingdom. The company has not announced regulatory submissions or timelines for other regions in this update. As an international organization, IHA recognizes that regulatory approval is only one part of meaningful access. Future access may also depend on affordability, health-system readiness, and the specialized clinical expertise and infrastructure needed to deliver treatments such as AMT-130. IHA will continue working toward a future in which advances in Huntington’s disease treatment and care can benefit families regardless of where in the world they live.

The International Huntington Association President, Svein Olaf Olsen, issued a statement in response:

“This is truly fantastic news and an important milestone for the entire Huntington’s disease community. After so many years of research, commitment and hope, it is extremely encouraging to see AMT-130 reach the regulatory authorities in both the United States and the United Kingdom.
The next step will be particularly exciting as we await the assessments and decisions from the FDA and MHRA. Whatever the outcome, reaching this stage represents significant progress and gives renewed hope to people and families affected by Huntington’s disease around the world.
Congratulations to the entire uniQure team and everyone who has contributed to bringing AMT-130 this far!”

For further details, read uniQure’s full press release.