FDA Supports uniQure Plan to Seek Accelerated Approval for Huntington’s Gene Therapy Using 3-Year Data
UniQure said the US Food and Drug Administration supports using three-year Phase I/II data from AMT-130 as the primary basis for a BLA seeking accelerated approval in Huntington’s disease. The Dutch drugmaker said it plans to submit the filing in the third quarter of 2026.
The feedback gives UniQure a clearer regulatory path for the gene therapy and also lifted ClearPoint Neuro, whose navigation system and SmartFlow cannula are used to deliver AMT-130 directly into the brain. ClearPoint shares rose 34% after the update.
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@stocksavvyshay$CLPT is up 34% after the FDA allowed $QURE to use three-year Phase I/II AMT-130 data as primary basis for an accelerated approval BLA filing planned for Q3 2026. ClearPoint is moving because AMT-130 is delivered directly into the brain us
@wallstengineuniQure $QURE says FDA feedback supports using 3-year Phase I/II AMT-130 data as the primary basis for a BLA seeking accelerated approval in Huntington’s disease. BLA planned for Q3 2026. ClearPoint $CLPT is also moving as AMT-130 is deli
@reuterslegalDutch drugmaker UniQure said on Wednesday the U.S. Food and Drug Administration has agreed that existing clinical trial data could support an accelerated approval filing for its gene therapy to treat Huntington's disease.
@uniqure_nvToday we provided a regulatory update on a path forward for AMT-130 for #huntingtonsdisease. Read the full update: