Medicines and Healthcare products Regulatory Agency

Stories

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

As of October 2026: Reviewing the AMT-130 Marketing Authorisation Application

uniQure reported four-year data from its Huntington's gene therapy trial on September 29. The results were mixed: the main composite measure showed 44% slower decline, short of statistical significance, while a functional-capacity measure showed 61% slower decline.

Updated Oct 1

United Kingdom enacts largest clinical trial reforms in 20 years

Rule Changes

As of May 2026: 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