Bio­Marin buffs up da­ta on he­mo­phil­ia A gene ther­a­py as FDA de­ci­sion dead­line ap­proach­es

Bio­Marin says its Phase III study for a he­mo­phil­ia A gene ther­a­py met its pri­ma­ry and sec­ondary end­points at year three, and it has added more da­ta per the FDA’s re­quest. The da­ta come ahead of a March 31 FDA de­ci­sion dead­line.

Last Au­gust, the San Rafael, CA biotech’s gene ther­a­py — known as val­oc­toco­gene rox­a­parvovec, or val­rox for short — won con­di­tion­al ap­proval in the EU, where it is mar­ket­ed as Roc­ta­vian and costs about $1.5 mil­lion. In the US, Bio­Marin ex­pects to price the drug be­tween $2 mil­lion and $3 mil­lion, CEO Jean-Jacques Bi­en­aimé has pre­vi­ous­ly said, which a spokesper­son re­it­er­at­ed in an email to End­points News.

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