Drug Information
| Drug General Information | Top | |||
|---|---|---|---|---|
| Drug ID |
D05JEG
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| Former ID |
DIB008154
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| Drug Name |
BMN-701
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| Synonyms |
IGF2-GAA; XC-701; ZC-701; Enzyme replacement therapy (GILT, Pompe disease), BioMarin/ZyStor
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| Indication | Pompe disease [ICD-11: 5C51.3; ICD-10: E74.0] | Phase 3 | [1] | |
| Type 2 glycogen storage disease [ICD-11: 5C51.3; ICD-10: E74.0; ICD-9: 271] | Phase 3 | [2] | ||
| Company |
Biomarin pharmaceutical
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| Target and Pathway | Top | |||
|---|---|---|---|---|
| Target(s) | Lysosomal alpha-glucosidase (GAA) | Target Info | Modulator | [3], [4] |
| KEGG Pathway | Galactose metabolism | |||
| Starch and sucrose metabolism | ||||
| Metabolic pathways | ||||
| Lysosome | ||||
| Pathwhiz Pathway | Galactose Metabolism | |||
| Pathway Interaction Database | Notch-mediated HES/HEY network | |||
| References | Top | |||
|---|---|---|---|---|
| REF 1 | ClinicalTrials.gov (NCT01924845) BMN 701 Phase 3 in rhGAA Exposed Subjects With Late Onset Pompe Disease (INSPIRE Study). U.S. National Institutes of Health. | |||
| REF 2 | Clinical pipeline report, company report or official report of the Pharmaceutical Research and Manufacturers of America (PhRMA) | |||
| REF 3 | Clinical pipeline report, company report or official report of BioMarin Pharma. | |||
| REF 4 | ClinicalTrials.gov (NCT01898364) Safety and Efficacy Evaluation of Repeat neoGAA Dosing in Late Onset Pompe Disease Patients.. U.S. National Institutes of Health. | |||
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