FDA Sets May 1 PDUFA Action Date for uniQure’s Huntington’s Disease Gene Therapy
uniQure has aligned with the FDA on what the company calls key elements of the confirmatory study design: The study is being designed as a randomized, standard-of-care trial enrolling approximately 200 patients in a 2:1 ratio of ifezuntirgene inilparvovec to standard of care, with Total Functional Capacity (TFC) at 36 months serving as the primary endpoint.
- Published by
- GEN — Genetic Engineering & Biotechnology News
- Evidence type
- Regulatory
uniQure has aligned with the FDA on what the company calls key elements of the confirmatory study design: The study is being designed as a randomized, standard-of-care trial enrolling approximately 200 patients in a 2:1 ratio of ifezuntirgene inilparvovec to standard of care, with Total Functional Capacity (TFC) at 36 months serving as the primary endpoint.
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