BRIEF—uniQure receives FDA RMAT designation for AMT-130 in Huntington’s

Netherlands-based gene therapy company uniQure announced that the US Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation for its AMT-130 gene therapy in development for Huntington’s disease.

It says that AMT-130 is the first ever investigational therapy for Huntington’s disease to receive RMAT designation.

The designation is given to gene therapies that show potential clinical benefit for patients.

With AMT-130, the designation follows the FDA’s review of interim Phase I/II clinical data announced in  December 2023 and is based on an analysis comparing the 24-month data to a non-concurrent criteria-matched natural history cohort.

uniQure expects to provide updated interim data from the ongoing Phase I/II US and European studies of AMT-130 in mid-2024.

The data will include up to three years of follow-up on 29 treated patients, 21 of which will have been followed for at least two years.