San Rafael, Calif., May 27, 2014 – BioMarin Pharmaceutical Inc. announced today that it has dosed the first patient with BMN 701 (GILT-tagged Recombinant Human GAA) in the Phase 3 INSPIRE trial for Pompe disease. BMN 701 is a novel fusion protein of insulin-like growth factor 2 and acid alpha glucosidase (IGF2-GAA), designed to target delivery to the lysosomes where the enzyme is most needed. The INSPIRE trial focuses on late onset Pompe disease – an inherited condition caused by a deficiency in the lysosomal enzyme acid alpha glucosidase, which can lead to progressive weakening of the muscles of the body, including the diaphragm, a crucial respiratory muscle. Respiratory impairment is the leading cause of morbidity and mortality in late onset Pompe disease.
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