UniQure's AMT-130 Gets FDA RMAT Designation in Huntington's Disease
By Colin Kellaher
UniQure has won a key U.S. Food and Drug Administration designation for its AMT-130 gene-therapy product candidate for the brain disorder Huntington's disease.
UniQure on Monday said the FDA granted regenerative-medicine-advanced-therapy, or RMAT, designation to AMT-130, marking the agency's first-ever RMAT designation for a gene-therapy product candidate in Huntington's, a rare, inherited neurodegenerative disorder.
The FDA's RMAT designation offers eligibility for expedited development and regulatory review of product candidates, including earlier and more frequent consultation with the agency, and the potential for accelerated approval.
Amsterdam-based UniQure said there are currently no approved therapies to delay the onset of Huntington's or to slow the disease's progression.
UniQure said it expects to provide updated interim data from ongoing Phase I/II studies of AMT-130 in the middle of 2024.
Write to Colin Kellaher at colin.kellaher@wsj.com
(END) Dow Jones Newswires
June 03, 2024 07:54 ET (11:54 GMT)
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