Drug Information
| Drug General Information | Top | |||
|---|---|---|---|---|
| Drug ID |
DN87BL
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| Drug Name |
Fidanacogene elaparvovec
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| Synonyms |
AAV8-hFIX19
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|
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| Drug Type |
Gene therapy
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| Indication | Haemophilia B [ICD-11: 3B11.0; ICD-9: 286.1] | Phase 3 | [1] | |
| Company |
Pfizer
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| Target and Pathway | Top | |||
|---|---|---|---|---|
| Target(s) | Coagulation factor IX (F9) | Target Info | Replacement | [2] |
| KEGG Pathway | Complement and coagulation cascades | |||
| Panther Pathway | Blood coagulation | |||
| Pathwhiz Pathway | Coagulation | |||
| Reactome | Extrinsic Pathway of Fibrin Clot Formation | |||
| Intrinsic Pathway of Fibrin Clot Formation | ||||
| Gamma-carboxylation of protein precursors | ||||
| Transport of gamma-carboxylated protein precursors from the endoplasmic reticulum to the Golgi apparatus | ||||
| Removal of aminoterminal propeptides from gamma-carboxylated proteins | ||||
| WikiPathways | Complement and Coagulation Cascades | |||
| PTM: gamma carboxylation, hypusine formation and arylsulfatase activation | ||||
| Blood Clotting Cascade | ||||
| Formation of Fibrin Clot (Clotting Cascade) | ||||
| References | Top | |||
|---|---|---|---|---|
| REF 1 | ClinicalTrials.gov (NCT03861273) A Study to Evaluate the Efficacy and Safety of Factor IX Gene Therapy With PF-06838435 in Adult Males With Moderately Severe to Severe Hemophilia B (BENEGENE-2). U.S. National Institutes of Health. | |||
| REF 2 | Factor IX assay discrepancies in the setting of liver gene therapy using a hyperfunctional variant factor IX-Padua. J Thromb Haemost. 2021 May;19(5):1212-1218. | |||
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