In an­oth­er shock­er, FDA re­jects Bio­Marin’s he­mo­phil­ia A gene ther­a­py, de­mand­ing more da­ta on dura­bil­i­ty

It turns out that Bio­Marin’s ri­vals aren’t the on­ly ones with doubts about the dura­bil­i­ty of Bio­Marin’s he­mo­phil­ia A gene ther­a­py. Reg­u­la­tors at the FDA have some prob­lems with it as well.

In the sec­ond ma­jor CRL in­side of 24 hours, the FDA has slapped down Bio­Marin’s ap­pli­ca­tion for val­oc­toco­gene rox­a­parvovec (val­rox) — their top pro­gram wide­ly tapped as a cinch at the agency. Ac­cord­ing to the biotech, they were com­plete­ly blind­sided by a de­mand to see 2 years of fol­low-on da­ta from their Phase III tri­al, rather than the lat­est up­date from Bio­Marin’s in­ves­ti­ga­tors.

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