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

This article was originally published in Pharmaceutical Approvals Monthly

Executive Summary

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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FDA Quietly Consults Patients To Understand Six-Minute Walk Significance

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 Awaits Data On Clinical Significance Of Vimizim Neutralizing Antibodies

Vimizim post-marketing requirements could help FDA better understand the clinical significance of anti-drug antibodies commonly seen with enzyme replacement therapies for lysosomal storage diseases and the risks of tolerance induction therapy to manage immunogenicity.

Vimizim Panel’s Desire For Quality-Of-Life Data Runs Into Regulatory Reality

FDA advisory committee overwhelmingly endorses BioMarin’s elosulfase alfa for Morquio A syndrome based on a walking endpoint but says future studies in the rare disease should include QoL assessments, prompting an agency rep to caution that such instruments need to be fully validated for regulatory purposes.


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