The makers of the treatment AMT 130, which saw such promising results announced in September 2025 have outlined what happens next in the development of the drug.
uniQure previously announced positive results of a new gene therapy treatment for Huntington's disease. The phase I/II study has shown that the treatment - AMT-130 - causes a statistically significant slowing (75%) of disease progression when measured at 36 months.
In April, an announcement was made of a Pre-Submission Meeting with the United Kingdom’s (UK) Medicines and Healthcare products Regulatory Agency (MHRA) and plans to submit a Marketing Authorization Application (MAA) for AMT-130 for the treatment of Huntington’s disease in the third quarter of 2026.
You can read more about the MHRA role in drug development on the website.
uniQure has now made progress with the FDA ( in America). The FDA had previously said they would not accept the data from the trial; however, they have now announced that the trial data would be acceptable as the primary basis of a Biologics License Application (BLA) for the accelerated approval of AMT-130 in Huntington’s disease.
You can read what this means in the article by HDBuzz
Cath Stanley BEM, Chief Executive Huntington’s Disease Association said,
Any news that the drug is being seriously considered for licensing is progress. However, as always, there has to be some caution; this process will take time. However, it seems both the UK and USA are open to the next stages. As a charity, we will continue to advocate for anyone affected by Huntington’s disease and will do all we can to support the community in gaining access to any potential treatments.