Prescription medicine
Nitisinone Brand names: Orfadin, Nityr, Harliku
Nitisinone (Harliku) is a prescription medicine with an FDA approval on record since 2002. FDA's NDC Directory lists it from 4 generic labelers.
- Generic listed
Check it at the source: the nitisinone FDA label on DailyMed (effective November 30, 2021, SWEDISH ORPHAN BIOVITRUM AB (PUBL)); the NDA 21232 record on Drugs@FDA; every nitisinone label on DailyMed.
Highlights of the record
FDA label of November 30, 2021; NDC Directory, Drugs@FDA and Orange Book, October 8, 2026
Used for
From the label: indications and usageORFADIN ® is indicated for the treatment of adult and pediatric patients with hereditary tyrosinemia type 1 (HT-1) in combination with dietary restriction of tyrosine and phenylalanine.
Forms and strengths
- 2 mg
- 5 mg
- 10 mg
- 20 mg
- 4 mg/ml
Capsule; tablet; suspension.
On the market
- Earliest approval on file
- January 18, 2002 Orfadin, Swedish Orphan; Drugs@FDA’s file, where older or withdrawn approvals can be missing
- Generic approvals
- 4 first August 26, 2019; 3 with a marketed status in Drugs@FDA
- Labelers listed (NDC)
- 6 of which 4 under generic applications, 2 under brand applications, repackagers included
- Last listed patent
- January 5, 2035 Nityr tablets; not a generic launch date
Supply and safety
- Pharmacy cost (NADAC)
- Not in the survey
- Shortage (FDA)
- No current entry
- Recalls by its makers (FDA)
- No recall recorded
- FAERS reports
- 948 through July 30, 2026
What is nitisinone used for?
From the label: indications and usageORFADIN ® is indicated for the treatment of adult and pediatric patients with hereditary tyrosinemia type 1 (HT-1) in combination with dietary restriction of tyrosine and phenylalanine. ORFADIN is a hydroxy-phenylpyruvate dioxygenase inhibitor indicated for the treatment of adult and pediatric patients with hereditary tyrosinemia type 1 (HT-1) in combination with dietary restriction of tyrosine and phenylalanine.
FDA pharmacologic class: 4-Hydroxyphenyl-Pyruvate Dioxygenase Inhibitor.
Source: FDA drug label (Orfadin), SWEDISH ORPHAN BIOVITRUM AB (PUBL), effective November 30, 2021 (SPL set 5d449b73-d503-4132-b978-d890491975df), via openFDA; the full label on DailyMed
Is there a generic for nitisinone (Harliku)?
The FDA approved the first generic nitisinone on August 26, 2019. 4 generic applications are approved; 3 of them carry a current (not discontinued) marketing status in Drugs@FDA; that status is not evidence that the generic is on sale.
FDA's NDC Directory lists nitisinone from 4 labelers under a generic (ANDA) application and from 2 brand labelers. A listing means the company has registered the product for sale; it does not show that every pharmacy stocks it.
The Orange Book lists 1 patent for Nityr tablets, the last expiring on January 5, 2035. A patent or exclusivity date is not the date a generic goes on sale: court cases and settlements move it either way.
- 2002 earliest approval on file
- 2019 first generic approved
- 2035 last listed patent
Source: Drugs@FDA, FDA NDC Directory and the Orange Book (openFDA bulk files, October 8, 2026)
What does the nitisinone label say about other medicines?
The nitisinone label names 5 other medicines in its interaction, contraindication or warning sections. The sentences are the labels' own; a label lists what its studies and reports found, not every interaction.
| Medicine named | Section | What the label says |
|---|---|---|
| Celecoxib | drug interactions | Clinically Relevant Interactions Affecting Co-Administered Drugs Sensitive CYP2C9 Substrates (e.g., celecoxib, tolbutamide) or CYP2C9 Substrates with a Narrow Therapeutic Index (e.g., phenytoin, warfarin) Clinical Impact Increased exposure of the co-administered drugs metabolized by CYP2C9. … |
| Ganciclovir | drug interactions | OAT1/OAT3 Substrates (e.g., adefovir, ganciclovir, methotrexate) Clinical Impact Increased exposure of the interacting drug [see Clinical Pharmacology ( 12.3 )] Intervention Monitor for potential adverse reactions related to the co-administered drug. … |
| Methotrexate | drug interactions | OAT1/OAT3 Substrates (e.g., adefovir, ganciclovir, methotrexate) Clinical Impact Increased exposure of the interacting drug [see Clinical Pharmacology ( 12.3 )] Intervention Monitor for potential adverse reactions related to the co-administered drug. … |
| Phenytoin | drug interactions | Clinically Relevant Interactions Affecting Co-Administered Drugs Sensitive CYP2C9 Substrates (e.g., celecoxib, tolbutamide) or CYP2C9 Substrates with a Narrow Therapeutic Index (e.g., phenytoin, warfarin) Clinical Impact Increased exposure of the co-administered drugs metabolized by CYP2C9. … |
| Warfarin | drug interactions | Clinically Relevant Interactions Affecting Co-Administered Drugs Sensitive CYP2C9 Substrates (e.g., celecoxib, tolbutamide) or CYP2C9 Substrates with a Narrow Therapeutic Index (e.g., phenytoin, warfarin) Clinical Impact Increased exposure of the co-administered drugs metabolized by CYP2C9. … |
Source: FDA drug label (Orfadin), SWEDISH ORPHAN BIOVITRUM AB (PUBL), effective November 30, 2021 (SPL set 5d449b73-d503-4132-b978-d890491975df), via openFDA; the full label on DailyMed
What do FAERS reports show for nitisinone?
The FDA Adverse Event Reporting System holds 948 reports that list nitisinone among the medicines taken, through July 30, 2026, each a report of something that happened, not proof the medicine caused it; 153 are coded as a death and 294 as a hospital stay. Counts follow how widely a medicine is used and watched, and they are not a measure of how risky it is.
Reports received each year
The most reported reactions
Source: FDA Adverse Event Reporting System (FAERS) via openFDA, reports through July 30, 2026
What next?
- Nitisinone side effects
- the label's list beside FAERS reports
- Check an interaction
- what two or more labels say about each other