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Orfadin(nitisinone)
Nityr, Orfadin (nitisinone) is a small molecule pharmaceutical. Nitisinone was first approved as Orfadin on 2002-01-18. It is used to treat tyrosinemias in the USA. It has been approved in Europe to treat tyrosinemias. The pharmaceutical is active against 4-hydroxyphenylpyruvate dioxygenase.
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Commercial
Therapeutic Areas
Therapeutic Area
MeSH
nervous system diseasesD009422
hereditary congenital and neonatal diseases and abnormalitiesD009358
nutritional and metabolic diseasesD009750
Trade Name
FDA
EMA
Nityr, Orfadin (generic drugs available since 2019-08-26)
Drug Products
FDA
EMA
New Drug Application (NDA)
New Drug Application (NDA)
Abbreviated New Drug Application (ANDA)
Abbreviated New Drug Application (ANDA)
Nitisinone
Tradename
Company
Number
Date
Products
NITYRCycle PharmaceuticalsN-209449 RX2017-07-26
3 products, RLD, RS
ORFADINOrphan MedicalN-021232 RX2002-01-18
4 products, RLD
ORFADINOrphan MedicalN-206356 RX2016-04-22
1 products, RLD, RS
Labels
FDA
EMA
Brand Name
Status
Last Update
nitisinoneANDA2023-04-27
nityrNew Drug Application2021-02-28
orfadinNew Drug Application2022-06-20
Indications
FDA
EMA
Indication
Ontology
MeSH
ICD-10
tyrosinemias—D020176E70.21
Agency Specific
FDA
EMA
No data
Patent Expiration
Patent
Expires
Flag
FDA Information
Nitisinone, Nityr, Cycle
103280292035-01-05DPU-1836
Nitisinone, Orfadin, Swedish Orphan
93019322033-02-28DPU-1836
ATC Codes
A: Alimentary tract and metabolism drugs
— A16: Other alimentary tract and metabolism products in atc
— A16A: Other alimentary tract and metabolism products in atc
— A16AX: Various alimentary tract and metabolism products
— A16AX04: Nitisinone
HCPCS
No data
Clinical
Clinical Trials
16 clinical trials
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Indications Phases 4
No data
Indications Phases 3
Indication
MeSH
Ontology
ICD-10
Ph 1
Ph 2
Ph 3
Ph 4
Other
Total
TyrosinemiasD020176E70.214—1—27
AlkaptonuriaD000474E70.29—32——4
Indications Phases 2
Indication
MeSH
Ontology
ICD-10
Ph 1
Ph 2
Ph 3
Ph 4
Other
Total
AlbinismD000417E70.311———1
Indications Phases 1
Indication
MeSH
Ontology
ICD-10
Ph 1
Ph 2
Ph 3
Ph 4
Other
Total
Healthy volunteers/patients—2————2
Drug interactionsD0043471————1
Indications Without Phase
Indication
MeSH
Ontology
ICD-10
Ph 1
Ph 2
Ph 3
Ph 4
Other
Total
Chronic diseaseD002908————11
Lactic acidosisD000140EFO_1000036E87.20————11
Epidemiology
Epidemiological information for investigational and approved indications
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Drug
General
Drug common nameNITISINONE
INNnitisinone
Description
Nitisinone is a cyclohexanone that is cyclohexane-1,3-dione substituted at position 2 by a 2-nitro-4-(trifluoromethyl)benzoyl group. It is used in the treatment of hereditary tyrosinemia type 1. It has a role as an EC 1.13.11.27 (4-hydroxyphenylpyruvate dioxygenase) inhibitor. It is a member of cyclohexanones, a C-nitro compound, a member of (trifluoromethyl)benzenes and a mesotrione.
Classification
Small molecule
Drug class—
Image (chem structure or protein)
Structure (InChI/SMILES or Protein Sequence)
O=C1CCCC(=O)C1C(=O)c1ccc(C(F)(F)F)cc1[N+](=O)[O-]
Identifiers
PDB—
CAS-ID104206-65-7
RxCUI61805
ChEMBL IDCHEMBL1337
ChEBI ID50378
PubChem CID115355
DrugBankDB00348
UNII IDK5BN214699 (ChemIDplus, GSRS)
Target
Agency Approved
HPD
HPD
Organism
Homo sapiens
Gene name
HPD
Gene synonyms
PPD
NCBI Gene ID
3242
Protein name
4-hydroxyphenylpyruvate dioxygenase
Protein synonyms
4-hydroxyphenylpyruvic acid oxidase, 4HPPD, glyoxalase domain containing 3, HPD, HPPDase
Uniprot ID
P32754
Mouse ortholog
Hpd (15445)
4-hydroxyphenylpyruvate dioxygenase (Q91WV9)
Alternate
No data
Variants
Clinical Variant
No data
Financial
No data
Trends
PubMed Central
Top Terms for Disease or Syndrome:
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Additional graphs summarizing 584 documents
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Safety
Black-box Warning
No Black-box warning
Adverse Events
Top Adverse Reactions
Mock data
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13,778 adverse events reported
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