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FDA Quietly Consults Patients To Understand Vimizim's Six-Minute Walk Significance

 
• By Derrick Gingery

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.

FDA consulted a Morquio A patient and several caregivers as it struggled to determine the clinical relevance of Vimizim’s effect on the six-minute walk test, apparently unbeknownst to the product’s sponsor, BioMarin Pharmaceutical Inc.

The consultation during FDA’s active BLA review process shows that patient groups can engage with FDA outside the context of...

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More from North America

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• By Sarah Karlin-Smith

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• By Derrick Gingery and Sue Sutter

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• By Sue Sutter

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