General Information of Drug (ID: DM4XMZW)

Drug Name
BH4 Drug Info
Indication
Disease Entry ICD 11 Status REF
Discovery agent N.A. Investigative [1]
Cross-matching ID
PubChem CID
135398654
ChEBI ID
CHEBI:59560
CAS Number
CAS 62989-33-7
TTD Drug ID
DM4XMZW

Molecule-Related Drug Atlas

Molecule-Related Drug Atlas
Molecule Type:
DTT
Drug Status:
Approved Drug(s)
Clinical Trial Drug(s)
Investigative Drug(s)
Drug Name Drug ID Indication ICD 11 Highest Status REF
Sapropterin hydrochloride DM90TJP Hyperphenylalaninemia 5C50 Approved [2]
Pegvaliase DM6NZ2P Phenylketonuria 5C50.0 Approved [3]
Phenylalanine hydroxylase DMRP3CV Autoimmune diabetes 5A10 Phase 2 [4]
HepaStem DM8QGPE Liver failure DB99.7 Phase 2 [5]
HMI-102 DM8B85Y Phenylketonuria 5C50.0 Phase 1/2 [6]
BMN-307 DMY0SKM Phenylketonuria 5C50.0 Phase 1/2 [7]
fenclonine DMUBO3E Discovery agent N.A. Investigative [5]
BMN-168 DM0VMZ4 Phenylketonuria 5C50.0 Investigative [5]
ALTU-236 DMDLN9S Genetic disease 8E02 Investigative [5]
alpha-methylphenylalanine DMAUH96 Discovery agent N.A. Investigative [8]
⏷ Show the Full List of 10 Drug(s)

Molecular Interaction Atlas of This Drug

Molecular Interaction Atlas

Drug Therapeutic Target (DTT)
DTT Name DTT ID UniProt ID MOA REF
Phenylalanine hydroxylase (PAH) TTGSVH2 PH4H_HUMAN Modulator [1]

References

1 Company report of BioMarin
2 2007 FDA drug approvals: a year of flux. Nat Rev Drug Discov. 2008 Feb;7(2):107-9.
3 Induction, titration, and maintenance dosing regimen in a phase 2 study of pegvaliase for control of blood phenylalanine in adults with phenylketonuria.Mol Genet Metab. 2018 Nov;125(3):217-227.
4 Single-dose, subcutaneous recombinant phenylalanine ammonia lyase conjugated with polyethylene glycol in adult patients with phenylketonuria: an open-label, multicentre, phase 1 dose-escalation trial. Lancet. 2014 Jul 5;384(9937):37-44.
5 URL: http://www.guidetopharmacology.org Nucleic Acids Res. 2015 Oct 12. pii: gkv1037. The IUPHAR/BPS Guide to PHARMACOLOGY in 2016: towards curated quantitative interactions between 1300 protein targets and 6000 ligands. (Target id: 1240).
6 Clinical pipeline report, company report or official report of Homology Medicines
7 ClinicalTrials.gov (NCT04480567) A Phase 1/2 Open-Label, Dose Escalation Study to Determine the Safety and Efficacy of BMN 307, an Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Phenylalanine Hydroxylase in Subjects With Phenylketonuria. U.S.National Institutes of Health.
8 Alpha-methylphenylalanine, a new inducer of chronic hyperphenylalaninemia in sucling rats. Science. 1976 Jun 4;192(4243):1007-8.