FDA reverses stance, allows UniQure to file for accelerated approval of Huntington’s gene therapy AMT-130
The Food and Drug Administration has changed its earlier position and now permits UniQure to submit a marketing application for its experimental Huntington’s disease gene therapy, AMT-130.
The agency indicated that a three-year analysis of early-stage study data, which showed benefit for patients, is acceptable to support an accelerated approval filing.
UniQure said it will file the application in the third quarter, seeking accelerated approval based on the presented evidence.
The move clears a regulatory obstacle for a therapy that could become the first genetic treatment for Huntington’s disease, a development closely watched by the biotech sector.
This writeup was produced by pharmadog from original reporting by STAT.
Original headline: “STAT+: Following dispute with FDA, UniQure is cleared to submit Huntington’s treatment for approval”
read at STAT ↗
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