Details of the Drug

General Information of Drug (ID: DMMKE2H)

Drug Name

Valoctocogene roxaparvovec

Synonyms

BMN 270

Indication

Disease Entry	ICD 11	Status	REF
Haemophilia A	3B10.0	Phase 3	[1]

Affected Organisms

Humans and other mammals

Drug Type

Gene therapy

Cross-matching ID

Molecular Interaction Atlas of This Drug

Drug Therapeutic Target (DTT)

DTT Name	DTT ID	UniProt ID	MOA	REF
Coagulation factor VIII (F8)	TT1290U	FA8_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 A

ICD Disease Classification

3B10.0

Molecule Name	Molecule Type	Gene Name	p-value	Fold-Change	Z-score
Coagulation factor VIII (F8)	DTT	F8	1.08E-22	0.89	1.82

Molecular Expression Atlas (MEA)

Jump to Detail Molecular Expression Atlas of This Drug

References

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.
2	Clinical pipeline report, company report or official report of BioMarin Pharmaceutical.