BioMarin execs make their case for hemophilia gene therapy as fretful analysts start to hedge their bets
BioMarin isn’t letting the perfect get in the way of the good.
In their Q2 update Thursday night, the executive crew led by J.J. Bienaimé told analysts they were dumping a lower dose of the hemophilia A gene therapy Valrox and argued that the early data in hand supports their case for heading straight to regulators before the end of the year with their pitch for a new drug approval in 2020.
“We’re really thrilled with the durability of Valrox three years down and counting,” says Bienaimé, touting the “huge” economic value of the gene therapy. “What shouldn’t get lost here is the value of patients and we’ve heard such transforming stories from patients about the impact that this has in their lives. I would be pleased to talk about that all day, every day.”
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