Medicines and Healthcare products Regulatory Agency

Stories

uniQure files Huntington's gene therapy for FDA and UK approval

Reviewing the AMT-130 Marketing Authorisation Application

uniQure filed its Huntington's disease gene therapy, called AMT-130, for approval in the United States and the United Kingdom on September 2, 2026. If approved, it would be the first disease-modifying treatment for a disease that today has none.

Updated Sep 4

United Kingdom enacts largest clinical trial reforms in 20 years

Rule Changes

Co-author and primary enforcer of the reformed framework

Running a clinical trial in the United Kingdom required navigating a 2004 framework for more than two decades, but on 28 April 2026, it was retired. New regulations from the Medicines and Healthcare products Regulatory Agency (MHRA) and Health Research Authority (HRA) introduce a fast-track route for lower-risk studies and a seven-day approval pathway for trial modifications. Registration and publication of summary results become legal requirements for the first time.

Updated May 31