UniQure Faces FDA Approval Uncertainty for Huntington’s Gene Therapy Amid Data Concerns

UniQure's gene therapy AMT-130 for Huntington's disease faces regulatory uncertainty after the FDA indicated that data from Phase 1/2 studies using an external control group may no longer suffice for a Biologics License Application (BLA) submission. This represents a significant shift from prior FDA guidance over the past year, which had previously suggested such data could support accelerated approval. As a result, the timeline for AMT-130's approval filing is now unclear, prompting UniQure to plan urgent discussions with the FDA and regulatory bodies in Europe and the UK to explore alternative approval pathways. The FDA had granted AMT-130 Breakthrough Therapy and Regenerative Medicines Advanced Therapy designations based on the earlier data, highlighting the impact of this regulatory change. UniQure's CEO expressed surprise and disappointment, emphasizing the need to continue validating the therapy for patients lacking effective treatments. The news caused a sharp decline in UniQure's stock price, reflecting market concerns about the therapy's future prospects.