'The head­lines are the head­lines, but': Bio­Marin talks up po­ten­tial sav­ings as he­mo­phil­ia gene ther­a­py launch looms

Bio­Marin ex­ecs are still stay­ing tight-lipped about their pric­ing plans for what is poised to be the world’s first he­mo­phil­ia gene ther­a­py. But as the com­pa­ny en­ters the fi­nal reg­u­la­to­ry stretch and ap­proach­es a po­ten­tial launch this sum­mer, they are al­so drop­ping more hints to get in­vestors ready.

First thing to know: They re­al­ly, re­al­ly don’t ex­pect an ad­vi­so­ry com­mit­tee to be con­vened for val­rox, which is un­der pri­or­i­ty re­view, to pop up be­fore its PDU­FA date on Au­gust 21.

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