An­oth­er road­block looms for Bio­Mar­in's he­mo­phil­ia A gene ther­a­py — but it might not take too long

More than two years ago, Bio­Marin went in­to a ver­sion of cor­po­rate shock when the FDA de­mand­ed to see two years of fol­low-up da­ta from its Phase III tri­al ahead of a mar­ket­ing de­ci­sion for its he­mo­phil­ia A gene ther­a­py val­oc­toco­gene rox­a­parvovec (val­rox). And Mon­day evening, ex­ecs put out word that the FDA has upped the ante one more time — putting a damper on any lin­ger­ing hopes for a Q1 ap­proval by the PDU­FA date next year.

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