Hank Fuchs (BioMarin)

How durable is durable? Bio­Marin gears up to bring he­mo­phil­ia A gene ther­a­py to FDA again with 2-year da­ta

A year and a half af­ter a stun­ning re­jec­tion, Bio­Marin says it has the dura­bil­i­ty da­ta the FDA pre­vi­ous­ly re­quest­ed, paving the way for a new BLA in the sec­ond quar­ter of this year.

Tout­ing “sta­ble and durable” bleed con­trol in what it calls the largest ever glob­al study of a gene ther­a­py in­volv­ing pa­tients with se­vere he­mo­phil­ia A, Bio­Marin notes its drug, val­oc­toco­gene rox­a­parvovec (bet­ter known as val­rox or Roc­ta­vian), met all pri­ma­ry and sec­ondary ef­fi­ca­cy end­points at the two-year analy­sis.

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