NDAORALCAPSULEPriority Review
Approved
Jan 2002
Lifecycle
LOE Approaching
Competitive Pressure
30/100
Clinical Trials
12
Data completeness
5/8 — Partial
Priority Review
ORFADIN (nitisinone) by Swedish Orphan Biovitrum is hydroxyphenylpyruvate dioxygenase inhibitors [moa]. Approved for hereditary tyrosinemia type i. First approved in 2002.
Drug data last refreshed 20h ago · AI intelligence enriched 2w ago
AI summarySynthesized from FDA + DailyMed + ClinicalTrials.gov data
ORFADIN (nitisinone) is an oral small-molecule hydroxyphenylpyruvate dioxygenase inhibitor approved in 2002 for hereditary tyrosinemia type I and other rare metabolic disorders including alkaptonuria and albinism-related vision loss. It works by blocking a key enzyme in the tyrosine degradation pathway, preventing accumulation of toxic metabolites. This mechanism addresses the root cause of these ultra-rare genetic conditions.
$0.317MPart D
LOE ApproachingStable
Product approaching loss of exclusivity with limited Part D volume (37 claims, $317K in 2023) suggests small specialized patient population and stable but declining career opportunities on the brand team.
Mechanism of Action
Hydroxyphenylpyruvate Dioxygenase Inhibitors
Pharmacologic Class:
4-Hydroxyphenyl-Pyruvate Dioxygenase Inhibitor
Indications (1)
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.