uniQure plans UK regulatory filing for Huntington's disease gene therapy

uniQure N.V. (NASDAQ: QURE) announced it held a pre-submission meeting with the UK's Medicines and Healthcare products Regulatory Agency and plans to submit a Marketing Authorization Application for AMT-130 for Huntington's disease treatment in the third quarter of 2026.

The gene therapy company discussed data package and manufacturing requirements with the MHRA to support the application. uniQure expects to base its submission on three-year analysis data from ongoing U.S. and European Phase I/II clinical trials.

The three-year data showed a 75% slowing of disease progression at the high dose as measured by the composite Unified Huntington's Disease Rating Scale compared to a propensity score-matched external control, with statistical significance of p=.003. The treatment was generally well-tolerated with a manageable safety profile, according to the company.

uniQure has also been granted a Type B meeting with the U.S. Food and Drug Administration in the second quarter of 2026. The company expects to discuss potential Phase III trial design elements and receive feedback on the proposed statistical analysis plan for four-year analysis data expected in the third quarter of 2026.

"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 Matt Kapusta, chief executive officer of uniQure.

The company is pursuing additional regulatory pathways in international markets for potential AMT-130 registration and expects to provide updates in the second half of 2026.

The information is based on a company press release.

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