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
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.