uniQure N.V. held a pre‑submission meeting with the UK Medicines and Healthcare products Regulatory Agency (MHRA) on April 30, 2026, to review the data package and manufacturing requirements for its Huntington’s disease gene therapy, AMT‑130. The discussion focused on the three‑year Phase I/II data that show a 75 % slowing of disease progression at the high dose (p = 0.003) and a 60 % reduction in the decline of patients’ ability to function independently, as well as the manufacturing plan needed to support a UK marketing authorization application (MAA).
Following the meeting, uniQure confirmed that it will submit the MAA in the third quarter of 2026. The company also secured a Type B meeting with the U.S. Food and Drug Administration (FDA) for the second quarter of 2026, where it will discuss a prospective, randomized, sham‑surgery‑controlled Phase III trial design and the statistical analysis plan for a four‑year data set. This follows the FDA’s March 2026 indication that Phase I/II data might not be sufficient for approval and that a more robust Phase III study is required.
The UK regulatory engagement represents a significant parallel pathway that could accelerate patient access outside the United States. While the FDA remains cautious, the MHRA’s constructive feedback signals that the current data package meets UK regulatory expectations, providing a tangible step toward global approval. The planned Q3 2026 MAA submission underscores uniQure’s confidence that the Phase I/II data are robust enough for the UK market, even as the company continues to refine its U.S. strategy.
"We are encouraged by the constructive feedback from the MHRA as we continue to work towards a regulatory submission for AMT‑130 in Huntington’s disease," said CEO Matt Kapusta. The announcement was well received by investors, reflecting optimism about the company’s progress toward regulatory approval in a major market and the potential for accelerated patient access.
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