uniQure gains FDA Priority Review for Huntington's disease gene therapy
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Puntos clave:
- The US FDA has accepted uniQure's BLA and granted Priority Review for its gene therapy ifezuntirgene inilparvovec for Huntington's disease.
- The action date is May 1, 2027.
- The company said that the agency is "tentatively planning" for an advisory committee meeting to discuss the application.
- The BLA is backed by a 36-month data analysis from a phase I/II clinical study of ifezuntirgene inilparvovec, compared to a propensity score-matched external control.
- The FDA and uniQure are also on the same page on crucial parts of the confirmatory study design. It is a randomized, standard-of-care trial enrolling about 200 patients in a 2:1 ratio of ifezuntirgene inilparvovec to standard of care, with total functional capacity as the primary endpoint at three years.
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