FDA Panel Backs Efficacy Of BioMarin’s Rare Disease Drug Vimizim
This article was originally published in The Pink Sheet Daily
Executive Summary
Majority of the Endocrinologic and Metabolic Drugs Advisory Committee says effectiveness of elosulfase alfa has been demonstrated and the drug should be approved for all patients with Morquio A syndrome regardless of baseline disease severity.
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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.