Details of the Drug

General Information of Drug (ID: DMFZ81T)

Drug Name
Fidanacogene elaparvovec
Synonyms AAV8-hFIX19
Indication
Disease Entry ICD 11 Status REF
Haemophilia B 3B11.0 Phase 3 [1]
Drug Type
Gene therapy
Cross-matching ID
UNII
413EU9081Y
DrugBank ID
DB16783
TTD ID
DN87BL

Molecular Interaction Atlas of This Drug


Drug Therapeutic Target (DTT)
DTT Name DTT ID UniProt ID MOA REF
Coagulation factor IX (F9) TTFEZ5Q FA9_HUMAN Replacement [2]
Molecular Interaction Atlas (MIA) Jump to Detail Molecular Interaction Atlas of This Drug

Molecular Expression Atlas of This Drug

The Studied Disease Haemophilia B
ICD Disease Classification 3B11.0
Molecule Name Molecule Type Gene Name p-value Fold-Change Z-score
Coagulation factor IX (F9) DTT F9 5.69E-01 -0.13 -0.45
Molecular Expression Atlas (MEA) Jump to Detail Molecular Expression Atlas of This Drug

References

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.
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.