Still reel­ing from re­jec­tion, Bio­Marin re­ports a glimpse of those 2-year val­rox da­ta FDA was look­ing for — but is it enough to change reg­u­la­tors' minds?

Bio­Marin now has the Phase III da­ta it needs to re­file val­rox, its gene ther­a­py for he­mo­phil­ia A, at the EMA — and pos­si­bly con­vince the FDA to re­con­sid­er its re­quire­ment.

Pulling from a to­tal of 134 pa­tients who had been fol­lowed up for a mean of 71.6 weeks, the biotech ze­roed in on a sub­group that was rolled over from a non-in­ter­ven­tion­al study. Among those 112 pa­tients, the an­nu­al­ized bleed­ing rate was re­duced by 84% com­pared to pro­phy­lac­tic Fac­tor VI­II re­place­ment. The one-time in­jec­tion of val­rox al­so cut mean an­nu­al­ized Fac­tor VI­II in­fu­sion by 99% (p <0.0001).

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