Dutch gene therapy company uniQure (Nasdaq: QURE) announced that the US Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to AMT-130, sending the firm’s shares leaping more than 38% to $13.00 by close of trading on Thursday.
The investigational drug is for the treatment of Huntington’s disease, a rare, inherited neurodegenerative disorder for which there are currently no disease-modifying therapies available.
This designation is in addition to Regenerative Medicine Advanced Therapy (RMAT) designation, Orphan Drug designation and Fast Track designation, all previously granted by the FDA to AMT-130.
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