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Efficacy Endpoint For BioMarin’s Vimizim Faces FDA Panel Scrutiny

This article was originally published in The Pink Sheet Daily

Executive Summary

Review documents released ahead of a Nov. 19 advisory committee meeting question whether the six-minute walk test is an appropriate endpoint for the Morquio A syndrome treatment, and whether the findings are clinically meaningful.

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BioMarin’s Vimizim Secures Broad Label In Morquio A Syndrome

No limits on age or disease severity of patients appear in elosulfase alfa labeling; agency reviewers had raised the issue of baseline function at a November advisory committee meeting.

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.

FDA Panel Backs Efficacy Of BioMarin’s Rare Disease Drug Vimizim

Majority of the Endocrinologic and Metabolic Drugs Advisory Committee says effectiveness of elosulfase alfa has been demonstrated and the drug should be approved for all patients with Morquio A syndrome regardless of baseline disease severity.

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