UniQure prepares for FDA advisory committee on Huntington’s gene therapy amid rare-disease regulatory jitters

UniQure said it is ready to present its Huntington's disease gene therapy, AMT-130, to an FDA advisory committee, as discussed in its Q2 earnings call.

The company expects the FDA may schedule the meeting and notes the therapy already holds Breakthrough Therapy and Regenerative Medicine Advanced Therapy designations, which could allow priority review.

The announcement comes as other rare-disease developers such as Capricor and Replimune have recently faced intensive advisory committee scrutiny and setbacks, creating broader regulatory uncertainty in the sector.