uniQure N.V. plans a UK MAA submission in Q3 2026 for AMT-130 in Huntington’s disease following MHRA discussions, supported by Phase I/II data showing 75% slowing of progression.
Written By: Chikkula Pavan Kumar, PharmD
Reviewed By: Pharmacally Editorial Team
uniQure N.V. announced that it has held a pre‑submission meeting with the UK’s 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 United Kingdom in the third quarter of 2026.
During the meeting, the company and the MHRA discussed the clinical and non‑clinical data package, as well as the manufacturing and quality‑system requirements needed to support the planned MAA. Following these discussions, uniQure intends to base its submission on the three‑year analysis from the ongoing Phase I/II clinical trials conducted in the United States and Europe.
At the three‑year time point, AMT‑130 demonstrated a statistically significant 75% slowing of disease progression at the high dose, as measured by the composite Unified Huntington’s Disease Rating Scale (cUHDRS), compared with a propensity‑score‑matched external control (p = 0.003). The therapy was generally well tolerated, with a manageable safety profile reported in these studies.
Matt Kapusta, chief executive officer of uniQure, stated that the feedback from the MHRA was constructive and supports the company’s plan to proceed with the MAA submission in the third quarter of 2026. He emphasized that this milestone is important for the Huntington’s disease community and reaffirmed the company’s intent to engage with global regulators to advance the therapy.
In parallel, uniQure has secured a Type B meeting with the U.S. Food and Drug Administration (FDA) in the second quarter of 2026. The company expects to use this meeting to discuss key aspects of a potential Phase III trial design and to obtain feedback on the statistical analysis plan for a four‑year dataset, which is anticipated to be available in the third quarter of 2026.
AMT-130 is an investigational gene therapy designed to reduce the production of mutant huntingtin protein, which drives disease progression.
The company is also exploring additional regulatory pathways in international markets for AMT-130 and expects to provide further updates in the second half of 2026.
Reference
uniQure Announces Successful UK MHRA Pre-Submission Meeting for AMT-130 in Huntington’s Disease, 30 April 2026, Press Releases | Investors & Media | uniQure
About the Writer
Chikkula Pavan Kumar, PharmD is a Doctor of Pharmacy with a keen interest in clinical pharmacy, pharmacovigilance, and evidence-based practice. In his words, he is passionate about patient safety and translating complex medical information into clear, research-driven communication.