Review Of Reviews: Drug Review Profiles 2014
Pharmaceutical Approvals Monthly’s Drug Review Profile stories from 2014.
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Meeting with Morquio A patient and caregivers appears to have helped to assuage FDA concerns about clinical relevance of the Vimizim trial’s primary endpoint – and illustrates FDA’s openness to patient input even during specific reviews of new product applications.
Vimizim post-marketing requirements could help FDA better understand the clinical significance of anti-drug antibodies commonly seen with enzyme replacement therapies for lysosomal storage diseases and the risks of tolerance induction therapy to manage immunogenicity.
BioMarin showed that the Vimizim BLA did not have enough data to prove that clinical and commercial sites are different. That’s not the same as concluding there is no difference between the sites, FDA said.