Treats hereditary tyrosinemia type 1 (HT-1). Sold as Orfadin, Nityr, Harliku. Every FDA application for this active ingredient: 3 branded and 4 generic.
As of Nov 14, 2026, FDA lists 7 applications for Nitisinone (sold as Orfadin, Nityr, Harliku), first approved Jan 18, 2002, with 4 new uses approved and 0 published complete response letters.
| Orfadin | Part D | $784K | +223% | 0 |
| Nitisinone | Part D | $649K | +46% | 0 |
| Harliku | Part D | $637K | New | 0 |
| Nityr | Part D | $35K | -13% | 0 |
| Jan 18, 2002 | OrfadinNDA 21232 · New molecular entity | Swedish Orphan | 12 | Marketed |
| Apr 22, 2016 | OrfadinNDA 206356 · New dosage form | Swedish Orphan | 4 | Marketed |
| Jul 26, 2017 | Nityr, HarlikuNDA 209449 · New formulation or manufacturer | Cycle | 4 | Marketed |
| Aug 26, 2019 | NitisinoneANDA 211041 | Novitium Pharma | 1 | Marketed |
| May 26, 2022 | NitisinoneANDA 212390 | Medunik | 0 | Marketed |
| Jan 9, 2023 | NitisinoneANDA 215908 | Torrent | 0 | Discontinued |
| May 25, 2023 | NitisinoneANDA 216201 | Eton | 0 | Marketed |
Applications, approvals and products come from FDA's Drugs@FDA data files, refreshed weekly, grouped by active ingredient as FDA spells it. Complete response letters are FDA's published letters. Medicare spending is CMS's Part D and Part B dashboard, matched to this ingredient by generic name; shortages are FDA's shortage list, matched by application number; clinical trials are ClinicalTrials.gov studies naming the ingredient as an intervention, refreshed daily; safety programs are FDA's REMS list, matched by application number or, for shared programs, by ingredient. Drugs@FDA
Novitium Pharma