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.

Unlock this article instantly by becoming a free subscriber.

You’ll get access to free articles each month, plus you can customize what newsletters get delivered to your inbox each week, including breaking news.

SIGN UP LOG IN BECOME A PREMIUM SUBSCRIBER