A Second Path: uniQure Plans Regulatory Filing for AMT-130 in the UK

On April 30, 2026, uniQure announced that it held a Pre-Submission Meeting with the United Kingdom’s Medicines and Healthcare products Regulatory Agency (MHRA) and plans to file a formal application for approval of AMT-130 for the treatment of Huntington’s disease (HD) in the UK. While the US regulatory path has hit significant bumps this year, this update reflects uniQure’s broader strategy to bring AMT-130 to people living with HD through multiple regulatory routes internationally.

What is the MHRA, and what is a Pre-Submission Meeting?

The MHRA is the regulatory agency responsible for assessing and approving medicines in the UK, roughly the equivalent of the US Food and Drug Administration (FDA). Like the FDA, the MHRA evaluates whether a drug is safe and effective before it can be prescribed to people.

A Pre-Submission Meeting is an early conversation between a drug company and a regulatory agency, held before a formal application is submitted. It allows the company to confirm that their data package and manufacturing information meet the agency’s requirements, essentially making sure everything is in order before going through the formal process. 

uniQure describes the feedback from this meeting as constructive, and says they are now planning to submit a Marketing Authorization Application (MAA), the formal application to market a drug in the UK, in the third quarter of 2026.

What data will support the application?

The planned UK filing will be based on the three-year data from uniQure’s ongoing Phase 1/2 clinical trials in the US and Europe. At the three-year mark, uniQure’s data analysis showed that people receiving the high dose of AMT-130 showed approximately 75% slowing of disease progression compared to a natural history control group, as measured by the composite Unified Huntington’s Disease Rating Scale (cUHDRS).

Some astute readers may be wondering whether this would be a standard approval or something more like an “accelerated” or “conditional” approval, the kind of earlier access pathway that was previously on the table in the US. The honest answer is that uniQure’s press release only refers to a standard MAA, and they haven’t specified whether they’ll seek a conditional authorization from the MHRA. 

Different regulatory agencies can (and do!) reach different conclusions from the same dataset.

The MHRA does have a conditional approval pathway for medicines that show promising early evidence in conditions with serious unmet need, and HD would clearly qualify on that front. But we don’t want to get ahead of what’s actually been announced. What we know right now is that uniQure plans to submit an application, and the MHRA will evaluate it on its own terms.

What’s happening in the US?

The US situation remains unresolved. The FDA told uniQure earlier this year that the existing Phase 1/2 data are not sufficient to support approval in the US and recommended a new randomized, sham-controlled trial. 

uniQure has since been granted a Type B meeting with the FDA in the second quarter of 2026, where they expect to discuss potential Phase 3 trial designs and get feedback on the statistical analysis plan for four-year AMT-130 data, expected in the third quarter of 2026.

Adding another layer of uncertainty, the leadership has just changed hands again at the Center for Biologics Evaluation and Research (CBER), which is the FDA division responsible for evaluating gene therapies like AMT-130. 

Dr. Vinay Prasad, who directed CBER during the period when the agency pushed back hardest on AMT-130 and called for a sham-controlled trial, departed the FDA on April 30, 2026, returning to his faculty position at the University of California, San Francisco. 

Dr. Katherine Szarama, who had served as Prasad’s deputy, has been named acting CBER director. Szarama is relatively new to the role and her views on gene therapy evaluation standards, including where she lands on AMT-130 specifically, are not yet publicly known. 

A permanent replacement for Prasad has not yet been officially named. How the incoming leadership approaches the AMT-130 question remains to be seen, and it’s one more thread we’ll be watching closely as the US path continues to take shape. 

Why does this matter, and what should we expect?

Different regulatory agencies can (and do!) reach different conclusions from the same dataset. The MHRA operates under its own standards, its own frameworks, and its own assessment of benefit and risk. So it’s genuinely possible that the UK path could look different from the US one. 

That said, submitting an application is not the same as receiving approval. The MHRA will evaluate the data and make its own determination.

For the HD community, particularly families in the UK and internationally, this is a meaningful step forward. uniQure has also stated it is actively pursuing regulatory pathways in additional international markets, with further updates expected in the second half of 2026. 

For families in the US, the picture is more complicated right now, but the Type B FDA meeting expected this quarter and the four-year data readout on the horizon mean that the US story is far from over. We’ll be watching closely and will keep you updated as the process develops.

Summary

• On April 30, 2026, uniQure announced a successful Pre-Submission Meeting with the UK’s MHRA
• uniQure plans to submit a formal Marketing Authorization Application (MAA) in the UK for AMT-130 in Q3 2026
• The filing will be based on three-year Phase 1/2 trial data from uniQure showing ~75% slowing of disease progression at the high dose
• uniQure has not specified whether they are seeking conditional or standard approval, but we’ll share more when that becomes clear
• In the US, the FDA has requested more data in a Phase 3 trial and a Type B meeting is planned for Q2 2026 to discuss potential trial designs
• uniQure is also pursuing regulatory pathways in additional international markets