STAT+: Following dispute with FDA, UniQure is cleared to submit Huntington’s treatment for approval
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- The FDA reversed its opposition to UniQure's experimental treatment AMT-130 for Huntington's disease, clearing a path for the company to file for U.S. approval.
- UniQure announced it will submit a marketing application in the third quarter, seeking accelerated approval for AMT-130.
- The FDA agreed at a recent meeting that three-year data from an early-stage study showing a benefit for Huntington's patients was "acceptable" to support a marketing application.
- The newly accepted data are the same AMT-130 results that Marty Makary, the FDA Commissioner, and Vinay Prasad, the agency's top regulator of cell and gene therapies, previously concluded were insufficient to support a filing.
- AMT-130 is a closely watched gene therapy for Huntington's disease, a fatal neurodegenerative condition with no approved disease-modifying treatments.
Why it matters: Huntington's patients gain a potential path to the first disease-modifying therapy for a fatal neurodegenerative disease, but the marketing application rests on early-stage data that FDA leadership itself once called insufficient — and Prasad, the gatekeeper of cell and gene therapy decisions who originally rejected the data, remains in his post to review the very same application.
