FDA rejects potentially game-changing hemophilia A gene therapy BioMarin over concerns it might not really be a one-and-done lifetime treatment
The U.S. Food and Drug Administration's rejection late Tuesday means the San Rafael, California-based company will have to complete an ongoing late-stage patient study, likely delaying possible approval till late in 2022.
The company added that FDA concluded differences between the results of a small, early-stage study and interim data from the late-stage study left unclear how long the therapy's effect would last. The gene therapy works by using an inactivated virus, created in a lab, to deliver to liver cells a working gene via a one-time IV infusion meant to enable the body to produce FVIII on its own.
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