NDAORALCAPSULEPriority Review
Approved
Jan 2002
Lifecycle
LOE Approaching
Competitive Pressure
30/100
Clinical Trials
12
Data completeness
4/8 — Partial
Priority Review
ORFADIN (nitisinone) by Swedish Orphan Biovitrum is hydroxyphenylpyruvate dioxygenase inhibitors [moa]. First approved in 2002.
Drug data last refreshed 23h ago · AI intelligence enriched 1mo ago
AI summarySynthesized from FDA + DailyMed + ClinicalTrials.gov data
ORFADIN (nitisinone) is an oral small-molecule hydroxyphenylpyruvate dioxygenase inhibitor approved in 2002 for the treatment of hereditary tyrosinemia type 1 (HT-1), a rare genetic disorder affecting amino acid metabolism. It works by blocking an enzyme in the tyrosine degradation pathway, preventing accumulation of toxic metabolites that damage the liver and kidneys. The drug is indicated for patients with HT-1 who require management of the disease's progressive hepatorenal complications.
$0.317MPart D
LOE ApproachingStable
Product approaching loss of exclusivity with modest Part D utilization (37 claims, $317K annual spending), indicating a small but stable patient population typical of rare disease orphan products.
Mechanism of Action
Hydroxyphenylpyruvate Dioxygenase Inhibitors
Pharmacologic Class:
4-Hydroxyphenyl-Pyruvate Dioxygenase Inhibitor
Indications
Indication data is being enriched from DailyMed and FDA labeling. Check back soon for approved therapeutic uses.
Clinical Trials (12)
A Non-interventional, Post-Marketing Study to Describe Outcome of Nitisinone Treatment in HT-1 Patients
Started Sep 2025
0Hereditary Tyrosinemia, Type I
A Study to Investigate the Potential Influence of Nitisinone on the Metabolism and the Transport of Other Drugs in Healthy Volunteers
Started Mar 2017
36 enrolled
Drug Drug Interaction
Bioequivalence Study of Two Oral Nitisinone Formulations to Treat Hereditary Tyrosinemia (HT-1)
Started Mar 2016
24 enrolled
Hereditary Tyrosinemia, Type I
Bioavailability Food-Effect Study of an Oral Nitisinone Formulation to Treat Hereditary Tyrosinemia (HT-1)
Started Nov 2015
20 enrolled
Hereditary Tyrosinemia, Type I
Bioequivalence Study of Two Nitisinone Formulations Compared to Orfadin
Started Oct 2015
24 enrolled
Hereditary Tyrosinemia, Type I
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Data Sources
- Drug label: DailyMed / FDA
- Clinical trials: ClinicalTrials.gov
- Patent data: FDA Orange Book
- Spending data: CMS Medicare
The information on this page is for informational purposes only and should not be used as a substitute for professional medical advice. Drug information is sourced from FDA, DailyMed, and other government databases. Adverse event data from FAERS does not establish causation. Always consult a healthcare professional for medical decisions.