What FDA’s Newest Gene Therapy Approval Tells Us About Durability: How Long is Long Enough?
FDA Law Blog: Drug Discovery
DECEMBER 7, 2022
This product was developed by uniQure prior to being licensed to CSL Behring. The FDA-approved Package Insert describes the “main efficacy outcome” as being “a non-inferiority test of annualized bleeding rate (ABR) during Months 7 to 18” as compared to the ABR during the lead-in period. That brings us to the product’s approval.
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