Details of the Drug

General Information of Drug (ID: DMBXR23)

Drug Name
BMN-110
Synonyms
Enzyme replacement therapy (iv infusion, MPS IVA), BioMarin; Enzyme replacement therapy (iv infusion, Morquio A syndrome), BioMarin; Enzyme replacement therapy (iv infusion, mucopolysaccharidosis IVA), BioMarin; N-acetylgalactosamine-6-sulfatase (GALNS) (Morquio A syndrome), BioMarin
Indication
Disease Entry ICD 11 Status REF
Morquio syndrome 5C56.32 Phase 3 [1]
Cross-matching ID
TTD ID
D0OQ9V

Molecular Interaction Atlas of This Drug


Drug Therapeutic Target (DTT)
DTT Name DTT ID UniProt ID MOA REF
N-acetylgalactosamine 6 sulfatase (GALNS) TTT9YPO GALNS_HUMAN Modulator [2]
Molecular Interaction Atlas (MIA) Jump to Detail Molecular Interaction Atlas of This Drug

References

1 ClinicalTrials.gov (NCT01415427) Long-Term Efficacy and Safety Extension Study of BMN 110 in Patients With Mucopolysaccharidosis IVA (Morquio A Syndrome). U.S. National Institutes of Health.
2 Efficacy and safety of enzyme replacement therapy with BMN 110 (elosulfase alfa) for Morquio A syndrome (mucopolysaccharidosis IVA): a phase 3 randomised placebo-controlled study. J Inherit Metab Dis. 2014 Nov;37(6):979-90.