FDA Takes Long, Skeptical View Of Vimizim’s Six-Minute Walk Endpoint
This article was originally published in Pharmaceutical Approvals Monthly
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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