Drug Information
| Drug General Information | Top | |||
|---|---|---|---|---|
| Drug ID |
D7HLI1
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| Drug Name |
Valoctocogene roxaparvovec
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| Synonyms |
BMN 270
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|
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| Drug Type |
Gene therapy
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| Indication | Haemophilia A [ICD-11: 3B10.0; ICD-9: 286] | Phase 3 | [1] | |
| Company |
BioMarin Pharmaceutical
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| Target and Pathway | Top | |||
|---|---|---|---|---|
| Target(s) | Coagulation factor VIII (F8) | Target Info | Replacement | [2] |
| KEGG Pathway | Complement and coagulation cascades | |||
| Panther Pathway | Blood coagulation | |||
| Pathwhiz Pathway | Coagulation | |||
| Reactome | Platelet degranulation | |||
| Intrinsic Pathway of Fibrin Clot Formation | ||||
| Common Pathway of Fibrin Clot Formation | ||||
| COPII (Coat Protein 2) Mediated Vesicle Transport | ||||
| Cargo concentration in the ER | ||||
| WikiPathways | Complement and Coagulation Cascades | |||
| Blood Clotting Cascade | ||||
| Formation of Fibrin Clot (Clotting Cascade) | ||||
| References | Top | |||
|---|---|---|---|---|
| REF 1 | ClinicalTrials.gov (NCT04323098) Study to Evaluate the Efficacy and Safety of Valoctocogene Roxaparvovec, With Prophylactic Steroids in Hemophilia A (GENEr8-3). U.S. National Institutes of Health. | |||
| REF 2 | Clinical pipeline report, company report or official report of BioMarin Pharmaceutical. | |||
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