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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