Drug Information

Drug General Information
Drug ID
D05JEG
Former ID
DIB008154
Drug Name
BMN-701
Synonyms
IGF2-GAA; XC-701; ZC-701; Enzyme replacement therapy (GILT, Pompe disease), BioMarin/ZyStor
Indication Pompe's disease [ICD10:E74.02] Phase 3 [524401]
Company
Biomarin pharmaceutical
Target and Pathway
Target(s) Lysosomal alpha-glucosidase Target Info Modulator [524362], [551880]
KEGG Pathway Galactose metabolism
Starch and sucrose metabolism
Metabolic pathways
Lysosome
Pathway Interaction Database Notch-mediated HES/HEY network
PathWhiz Pathway Galactose Metabolism
References
Ref 524401ClinicalTrials.gov (NCT01924845) BMN 701 Phase 3 in rhGAA Exposed Subjects With Late Onset Pompe Disease (INSPIRE Study). U.S. National Institutes of Health.
Ref 524362ClinicalTrials.gov (NCT01898364) Safety and Efficacy Evaluation of Repeat neoGAA Dosing in Late Onset Pompe Disease Patients.. U.S. National Institutes of Health.
Ref 551880Clinical pipeline report, company report or official report of BioMarin Pharma.

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