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Shares of gene therapy developer uniQure N.V. (QURE) shot up 41% on Thursday afternoon after it announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to its AMT-130 gene therapy for the treatment of Huntington’s disease.
The new designation is in addition to the existing Regenerative Medicine Advanced Therapy (RMAT), Orphan Drug, and Fast Track designations granted to AMT-130.
uniQure CEO Walid Abi-Saab said that the designation underscores both the urgent need for effective treatments for Huntington’s disease and the encouraging interim data demonstrating that AMT-130 has the potential to slow disease progression.
He added that the company looks to commercialize the drug as quickly as possible.
Forty-five patients have received AMT-130 to date as part of trials, the company stated, while adding that an additional regulatory update regarding its Biologics License Application submission to the FDA requesting marketing approval is expected in the second quarter.
Huntington’s disease is a rare, inherited neurodegenerative disorder. According to a 2021 study in Neuroepidemiology, approximately 70,000 people have been diagnosed with Huntington’s disease in the U.S. and Europe, with hundreds of thousands of others at risk of inheriting it.
FDA’s Breakthrough Therapy designation is intended to expedite the development and review of investigational therapeutic candidates that are intended to treat a serious condition, and if preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over available therapy.
On Stocktwits, retail sentiment around uniQure flipped from ‘bearish’ to ‘extremely bullish’ over the past 24 hours while message volume jumped from ‘low’ to ‘extremely high’.
QURE stock is down approximately 27% year-to-date but up about 183% over the past 12 months.
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