Nitisinone

Nitisinone
Clinical data
Trade names	Orfadin, others
Other names	NTBC
AHFS/Drugs.com	Monograph
License data	US DailyMed: Nitisinone;
Routes of; administration	By mouth
ATC code	A16AX04 (WHO) ;
Legal status
Legal status	CA: ℞-only; US: ℞-only; EU: Rx-only;
Pharmacokinetic data
Elimination half-life	Approximately 54 h (Range: 39 to 86 hours)
Identifiers
	IUPAC name 2-[2-nitro-4-(trifluoromethyl)benzoyl]; cyclohexane-1,3-dione;
CAS Number	104206-65-7 ;
PubChem CID	115355;
IUPHAR/BPS	6834;
DrugBank	DB00348 ;
ChemSpider	103195 ;
UNII	K5BN214699;
KEGG	D05177 ;
ChEBI	CHEBI:50378 ;
ChEMBL	ChEMBL1337 ;
CompTox Dashboard (EPA)	DTXSID9042673 ;
ECHA InfoCard	100.218.521
Chemical and physical data
Formula	C14H10F3NO5
Molar mass	329.231 g·mol−1
3D model (JSmol)	Interactive image;
	SMILES O=C(c1ccc(cc1[N+]([O-])=O)C(F)(F)F)C2C(=O)CCCC2=O;
	InChI InChI=1S/C14H10F3NO5/c15-14(16,17)7-4-5-8(9(6-7)18(22)23)13(21)12-10(19)2-1-3-11(12)20/h4-6,12H,1-3H2 ; Key:OUBCNLGXQFSTLU-UHFFFAOYSA-N ;

Nitisinone, sold under the brand name Orfadin among others, is a medication used for the treatment of hereditary tyrosinemia type 1;^[2]^[4] or for the reduction of urine homogentisic acid in adults with alkaptonuria.^[5] Nitisinone is a hydroxyphenyl-pyruvate dioxygenase inhibitor.^[2]^[4]

It is available as a generic medication.^[6]

^ "Health Canada New Drug Authorizations: 2016 Highlights". Health Canada. 14 March 2017. Retrieved 7 April 2024.
^ ^a ^b ^c "Orfadin- nitisinone capsule". DailyMed. 30 November 2021. Retrieved 5 July 2025.
^ "Orfadin- nitisinone suspension". DailyMed. 20 June 2022. Retrieved 5 July 2025.
^ ^a ^b ^c "Nityr- nitisinone tablet". DailyMed. 24 May 2024. Retrieved 5 July 2025.
^ ^a ^b https://www.accessdata.fda.gov/drugsatfda_docs/label/2025/209449s018lbl.pdf
^ "2023 First Generic Drug Approvals". U.S. Food and Drug Administration (FDA). 29 June 2023. Archived from the original on 29 June 2023. Retrieved 29 June 2023.

[1] "Health Canada New Drug Authorizations: 2016 Highlights". Health Canada. 14 March 2017. Retrieved 7 April 2024.

[Orfadin_FDA_label-2] "Orfadin- nitisinone capsule". DailyMed. 30 November 2021. Retrieved 5 July 2025.

[3] "Orfadin- nitisinone suspension". DailyMed. 20 June 2022. Retrieved 5 July 2025.

[Nityr_FDA_label-4] "Nityr- nitisinone tablet". DailyMed. 24 May 2024. Retrieved 5 July 2025.

[Harliku_FDA_label-5] ttps://www.accessdata.fda.gov/drugsatfda_docs/label/2025/209449s018lbl.pdf

[6] "2023 First Generic Drug Approvals". U.S. Food and Drug Administration (FDA). 29 June 2023. Archived from the original on 29 June 2023. Retrieved 29 June 2023.

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