Bio­Marin re­turns to FDA with a new pitch for he­mo­phil­ia gene ther­a­py — and a promise to get to the bot­tom of dura­bil­i­ty

Two years ago, the FDA slapped a sur­prise re­jec­tion on Bio­Marin’s BLA for its gene ther­a­py for he­mo­phil­ia A, de­mand­ing two-year da­ta from a Phase III tri­al to an­swer ques­tions about dura­bil­i­ty.

Bio­Marin is now back with a re­sub­mis­sion — with the da­ta that the FDA asked for and a promise for even more.

The new BLA fil­ing comes a month af­ter the Eu­ro­pean Com­mis­sion grant­ed con­di­tion­al ap­proval to val­oc­toco­gene rox­a­parvovec, more col­lo­qui­al­ly known as val­rox, un­der the brand name Roc­ta­vian.

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