Publication date: Apr 09, 2019
uniQure Receives FDA Fast Track Designation for AMT-130 Gene Therapy for the Treatment of Huntington’s Disease ~ On Track to Treat First Patient in Phase I/II Study of AMT-130 in 2H19 ~ LEXINGTON, Mass. and AMSTERDAM, the Netherlands, April 08, 2019 (GLOBE NEWSWIRE) — uniQure N. V. (NASDAQ:QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that the U. S. Food and Drug Administration (FDA) has granted Fast Track designation for AMT-130, the Company’s gene therapy candidate for the treatment of Huntington’s disease.
“Achieving Fast Track Designation from the FDA underscores the high unmet medical need for patients suffering from Huntington’s disease, for which there are currently no approved, disease-modifying treatments,” stated Matt Kapusta, chief executive officer of uniQure.
“We are nearing the initiation of a Phase I/II study of AMT-130, the first one-time administered AAV gene therapy to enter clinical testing for Huntington’s disease, and are on track to treat the first patient in the second half of 2019. “
Our actual results could differ materially from those anticipated in these forward-looking statements for many reasons, including, without limitation, risks associated with our and our collaborators’ clinical development activities, clinical results, collaboration arrangements, corporate reorganizations and strategic shifts, regulatory oversight, product commercialization and intellectual property claims, as well as the risks, uncertainties and other factors described under the heading “Risk Factors” in uniQure’s Annual Report on Form 10-K filed on February 28, 2019.