Drugs / Nitisinone
Nitisinone
also known as
NTBC · Nitisinona · Nitisinone mdk (previously nitisinone mendelikabs) · Nityr · Orfadin · SC-0735
Small molecule targets CYP2C9, CYP2D6, CYP2E1, HPD via inhibition
Regulatory milestones approvals, filings & regulatory actions · 5 recorded
| Milestone | Jurisdiction | Brand | Indication | Date | Sentence it was read from |
|---|---|---|---|---|---|
| Label expansions (2) | US (FDA) | HARLIKU | — | 2020-09-02 – 2025-06-10 | fda.gov ↗ |
| Label expansion | US (FDA) | ORFADIN | — | 2017-09-01 | fda.gov ↗ |
| Approved | EU (EMA) | Orfadin | — | 2005-02-21 | europa.eu ↗ |
| Approved | US (FDA) | ORFADIN | — | 2002-01-18 | fda.gov ↗ |
Trials 12 · a red edge is where a trial was stopped
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 13 trials | ||||||
| Phase 1 | NCT03103568 | Mar → Jun 2017 | — | Swedish Orphan Biovitrum | Completed | No outcome recorded |
| Phase 1 | NCT02750332 | Nov → Dec 2015 | tyrosinemia type I | Cycle Pharmaceuticals Ltd. | Completed | No outcome recorded |
| Phase 1 | NCT01734889 | Oct 2012 → Mar 2013 | tyrosinemia type I | Swedish Orphan Biovitrum | Completed | No outcome recorded |
| Phase 1/21 trial | ||||||
| Phase 1/2 | NCT01838655 | Apr 2013 → Jul 2016 | oculocutaneous albinism, vision disorder | National Eye Institute (NEI) | Completed | No outcome recorded |
| Phase 22 trials | ||||||
| Phase 2 | NCT01828463 | May → Oct 2013 | alkaptonuria | University of Liverpool | Completed | No outcome recorded |
| Phase 2 | NCT00107783 | Jan 2005 → Apr 2009 | alkaptonuria | National Human Genome Research Institute (NHGRI) | Completed | No outcome recorded |
| Phase 2/31 trial | ||||||
| Phase 2/3 | NCT01390077 | Jan 2011 → Jun 2016 | alkaptonuria | University of California, San Diego | Completed | No outcome recorded |
| Phase 32 trials | ||||||
| Phase 3 | NCT02323529 | Dec 2014 → Sep 2015 | tyrosinemia type I | Swedish Orphan Biovitrum | Completed | No outcome recorded |
| Phase 3 | NCT01916382 | Apr 2014 → Feb 2016 | alkaptonuria | University of Liverpool | Unknown | No outcome recorded |
| Phase not applicable3 trials | ||||||
| — | NCT04113772 | Nov 2019 → Dec 2020 | tyrosinemia type I | Sutphin Drugs | Unknown | No outcome recorded |
| — | NCT00031161 | Sep 2001 → Sep 2005 | lactic acidosis | University of Florida | Completed | No outcome recorded |
| — | NCT00004443 | Oct 1998 → Sep 2006 | tyrosinemia type I | University of Washington | Completed | No outcome recorded |
Evidence & citations 14 cited values
Every value below carries the sentence it was read from. 13 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | Nitisinone | ClinicalTrials.gov intervention name — accepted as the source's own label NCT01828463 ↗ |
| Known as | Nitisinona | ChEMBL registry synonym — accepted as the source's own label CHEMBL1337 ↗ |
| Known as | Nitisinone mdk (previously nitisinone mendelikabs) | ChEMBL registry synonym — accepted as the source's own label CHEMBL1337 ↗ |
| Known as | Nityr | ChEMBL registry synonym — accepted as the source's own label CHEMBL1337 ↗ |
| Known as | NTBC | “a compound named 2-(2-nitro-4-trifluoromethylbenzoyl) - 1, 3-cyclohexanedione (nitisinone, NTBC, Orfadin) inhibits 4-hydroxyphenylpyruvate dioxygenase” NCT00107783 ↗ |
| Known as | Orfadin | “Nitisinone 10 mg Tablets (Test Product are bioequivalent to the reference product Orfadin 10 mg” NCT04113772 ↗ |
| Known as | SC-0735 | ChEMBL registry synonym — accepted as the source's own label CHEMBL1337 ↗ |
| Action | Inhibit | “4-hydroxyphenylpyruvate dioxygenase inhibitor” CHEMBL1337 ↗1“inhibits 4-hydroxyphenylpyruvate dioxygenase” NCT00107783 ↗ |
| Modality | Small molecule | — CHEMBL1337 ↗ |
| Route | Oral | “Patients receive oral NTBC twice a day.” NCT00004443 ↗ |
| Target | CYP2C9 | “potential inhibition of nitisinone on CYP2C9, CYP2D6, and CYP2E1” NCT03103568 ↗ |
| Target | CYP2D6 | “potential inhibition of nitisinone on CYP2C9, CYP2D6, and CYP2E1” NCT03103568 ↗ |
| Target | CYP2E1 | “potential inhibition of nitisinone on CYP2C9, CYP2D6, and CYP2E1” NCT03103568 ↗ |
| Target | HPD | “4-hydroxyphenylpyruvate dioxygenase inhibitor” CHEMBL1337 ↗ |