Jean-Jacques Bienaimé (BioMarin via Youtube)

Once again, the FDA is de­lay­ing Bio­Marin’s he­mo­phil­ia A quest as gene ther­a­py frets sim­mer

Two years af­ter the FDA turned back Bio­Marin’s push for a land­mark ac­cel­er­at­ed ap­proval of their he­mo­phil­ia A gene ther­a­py val­rox, reg­u­la­tors are ask­ing for still more in­fo on the ther­a­py — and once again de­lay­ing any prospec­tive launch, this time in­to next year.

Buried un­der the lat­est set of ex­tend­ed da­ta from their Phase I/II tri­al of val­rox (val­oc­toco­gene rox­a­parvovec), which con­tin­ues to demon­strate its ef­fec­tive­ness in pre­vent­ing bleeds up to 6 years af­ter ini­tial treat­ment, the biotech spelled out the rea­sons why they’re shov­ing back an ex­pect­ed re­sub­mis­sion from June to Sep­tem­ber. The agency wants more da­ta and analy­sis, says the biotech, but isn’t ask­ing for any new stud­ies. And the news will like­ly en­hance some deep-seat­ed con­cerns that the FDA ap­pears to be in no rush to ap­prove any gene ther­a­py over­shad­owed by ques­tions of dura­bil­i­ty and safe­ty.

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