UniQure Wins First FDA RMAT in Huntington’s Disease After Strong Phase I/II Data

1. First FDA RMAT Designation: UniQure's investigational gene therapy, AMT-130, has received the first-ever Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA for the treatment of Huntington's disease.
2. Phase I/II Clinical Trials: The RMAT designation is based on interim data from ongoing U.S. and European Phase I/II clinical trials, which have shown promising results in slowing disease progression and reducing neurofilament light (NfL) levels in patients with Huntington's disease.
3. Dose-Dependent Clinical Benefit: The trials have demonstrated a dose-dependent clinical benefit, with high-dose AMT-130 resulting in significant slowing of disease progression and lowering of NfL levels compared to a propensity-weighted external control group.
4. Safety Profile: The gene therapy has been found to be generally well-tolerated, with a manageable safety profile and no new serious adverse events reported.
5. Future Development: UniQure plans to hold a Type B, multi-disciplinary meeting with the FDA to discuss potential expedited clinical development pathways and accelerated approval for AMT-130.