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BioWorld - Friday, May 15, 2026
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Home » Biomarin sees durability in hemophilia A gene therapy, gives phase III data peek
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Biomarin sees durability in hemophilia A gene therapy, gives phase III data peek

May 29, 2019
By Michael Fitzhugh
New data on Biomarin Pharmaceutical Inc.'s investigational treatment for severe hemophilia A, valoctocogene roxaparvovec (valrox), sparked mild debate among analysts who expressed appreciation for the gene therapy's apparent durability but uncertainty regarding interim data from a phase III test of the medicine. But overall, it "at least met – and in some cases exceeded – the majority of investors' expectations," J.P. Morgan analyst Cory Kasimov said. Though the market rendered its own verdict, driving company shares (NASDAQ:BMRN) 5.1% lower to $84.50 by Tuesday's close, the program seemed poised to retain significant interest going forward.
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