The FDA approved the gene therapy valoctocogene roxaparvovec for adults with severe hemophilia A.
, uptake in Europe has been delayed due to reimbursement issues, given the cost of treatment and clinical uncertainties.
More than 80% of participants had no bleeding events requiring treatment, and there was a 98% reduction from baseline in mean use of exogenous factor VIII. Trial investigators estimated that the typical half-life of the transgene-derived factor VIII production system is 123 weeks.
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