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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Review Of Reviews: Drug Review Profiles 2014
Pharmaceutical Approvals Monthly’s Drug Review Profile stories from 2014.
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.