Details of the Drug

General Information of Drug (ID: DMM4FXA)

Drug Name

Asfotase alfa

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]
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Sequence

LVPEKEKDPKYWRDQAQETLKYALELQKLNTNVAKNVIMFLGDGMGVSTVTAARILKGQL
HHNPGEETRLEMDKFPFVALSKTYNTNAQVPDSAGTATAYLCGVKANEGTVGVSAATERS
RCNTTQGNEVTSILRWAKDAGKSVGIVTTTRVNHATPSAAYAHSADRDWYSDNEMPPEAL
SQGCKDIAYQLMHNIRDIDVIMGGGRKYMYPKNKTDVEYESDEKARGTRLDGLDLVDTWK
SFKPRYKHSHFIWNRTELLTLDPHNVDYLLGLFEPGDMQYELNRNNVTDPSLSEMVVVAI
QILRKNPKGFFLLVEGGRIDHGHHEGKAKQALHEAVEMDRAIGQAGSLTSSEDTLTVVTA
DHSHVFTFGGYTPRGNSIFGLAPMLSDTDKKPFTAILYGNGPGYKVVGGERENVSMVDYA
HNNYQAQSAVPLRHETHGGEDVAVFSKGPMAHLLHGVHEQNYVPHVMAYAACIGANLGHC
APASSLKDKTHTCPPCPAPELLGGPSVFLFPPKPKDTLMISRTPEVTCVVVDVSHEDPEV
KFNWYVDGVEVHNAKTKPREEQYNSTYRVVSVLTVLHQDWLNGKEYKCKVSNKALPAPIE
KTISKAKGQPREPQVYTLPPSREEMTKNQVSLTCLVKGFYPSDIAVEWESNGQPENNYKT
TPPVLDSDGSFFLYSKLTVDKSRWQQGNVFSCSVMHEALHNHYTQKSLSLSPGKDIDDDD
DDDDDD

ADMET Property

Half-life: The concentration or amount of drug in body reduced by one-half in 5 days [2]

Cross-matching ID

DrugBank ID: DB09105
TTD ID: D05XSJ

Molecular Interaction Atlas of This Drug

Drug Therapeutic Target (DTT)

DTT Name	DTT ID	UniProt ID	MOA	REF
Alkaline phosphatase tissue-nonspecific (ALPL)	TTMR5UV	PPBT_HUMAN	Modulator	[3]

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Molecular Interaction Atlas (MIA)

MIA Detail

Jump to Detail Molecular Interaction Atlas of This Drug

Molecular Expression Atlas of This Drug

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 (MEA)

MEA Detail

Jump to Detail Molecular Expression Atlas of This Drug

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	Trend Analysis of a Database of Intravenous Pharmacokinetic Parameters in Humans for 1352 Drug Compounds
3	Enzyme-replacement therapy in life-threatening hypophosphatasia. N Engl J Med. 2012 Mar 8;366(10):904-13.