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FDA Quietly Consults Patients To Understand Six-Minute Walk Significance

This article was originally published in Pharmaceutical Approvals Monthly

Executive Summary

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.

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FDA Takes Long, Skeptical View Of Vimizim’s Six-Minute Walk Endpoint

FDA questioned the clinical meaningfulness of BioMarin’s endpoint for its Morquio A syndrome treatment, but the company declined to conduct FDA’s suggested trial. Unmet need convinced the agency to approve Vimizim on available data while it still seeks more suitable measures of efficacy.

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