Still reeling from rejection, BioMarin reports a glimpse of those 2-year valrox data FDA was looking for — but is it enough to change regulators' minds?
BioMarin now has the Phase III data it needs to refile valrox, its gene therapy for hemophilia A, at the EMA — and possibly convince the FDA to reconsider its requirement.
Pulling from a total of 134 patients who had been followed up for a mean of 71.6 weeks, the biotech zeroed in on a subgroup that was rolled over from a non-interventional study. Among those 112 patients, the annualized bleeding rate was reduced by 84% compared to prophylactic Factor VIII replacement. The one-time injection of valrox also cut mean annualized Factor VIII infusion by 99% (p <0.0001).
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