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Whatcha Say on Pompe, FDA? Biomarin Posts Endpoint Plan

By Randy Osborne
Staff Writer

Thursday, August 22, 2013
Biomarin Pharmaceutical Inc. has made clearer its Phase III plans for the Pompe disease drug candidate BMN-701, a fusion protein of insulin-like growth factor 2 and acid alpha glucosidase (GAA), but the study has not yet begun to enroll patients and talks are still under way about an endpoint that could help better distinguish the compound from Sanofi SA's competitor Lumizyme.

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