General Information of Drug (ID: DMM4FXA)

Drug Name
Asfotase alfa Drug Info
Synonyms
SPHEX, BioMep; SPHEX, Enobia; PHEX-based enzyme replacement therapy, BioMep; PHEX-based enzyme replacement therapy, Enobia; X-linked hypophosphatemic rickets therapy, BioMep; X-linked hypophosphatemic rickets therapy, Enobia
Indication
Disease Entry ICD 11 Status REF
Genetic disease 8E02 Approved [1]
Cross-matching ID
TTD Drug ID
DMM4FXA

Molecule(s) Related to This Drug


Drug Therapeutic Target (DTT)
DTT Name DTT ID UniProt ID MOA REF
Alkaline phosphatase tissue-nonspecific (ALPL) TTMR5UV PPBT_HUMAN Modulator [2]

The Expression Level of Molecule(s) in Normal Tissue of Major ADME-Related Organs

Molecule Molecule Type Gene Name Liver Colon Kidney Small Intestine
Alkaline phosphatase tissue-nonspecific (ALPL) DTT ALPL 6.35 4.07 5.7 3.609
Molecule Expression Atlas in Normal Tissue of Major ADME-related organs

The Expression Level of Molecule(s) between Disease Section and Healthy Individual Tissue

The Studied Disease Genetic disease
ICD Disease Classification 8E02
Molecule Name Molecule Type Gene Name p-value Fold-Change Z-score
Alkaline phosphatase tissue-nonspecific (ALPL) DTT ALPL 4.67E-13 -0.15 -0.79
Molecular Expression Atlas between Disease Section and Healthy Individual Tissue

References

1 ClinicalTrials.gov (NCT01176266) Open-Label Study of Asfotase Alfa in Infants and Children 5 Years of Age With Hypophosphatasia (HPP). U.S. National Institutes of Health.
2 Enzyme-replacement therapy in life-threatening hypophosphatasia. N Engl J Med. 2012 Mar 8;366(10):904-13.