NDAORALSUSPENSION
Approved
Apr 2016
Lifecycle
Peak
Competitive Pressure
0/100
Clinical Trials
12
Data completeness
4/8 — Partial
ORFADIN (nitisinone) by Swedish Orphan Biovitrum is hydroxyphenylpyruvate dioxygenase inhibitors [moa]. First approved in 2016.
Drug data last refreshed 3h ago · AI intelligence enriched 1w ago
AI summarySynthesized from FDA + DailyMed + ClinicalTrials.gov data
ORFADIN (nitisinone) is an oral suspension hydroxyphenylpyruvate dioxygenase inhibitor approved in 2016 for the treatment of hereditary tyrosinemia type 1 (HT-1), a rare genetic disorder affecting amino acid metabolism. The drug works by blocking a key enzyme in the tyrosine degradation pathway, reducing toxic metabolite accumulation and preventing liver and kidney damage.
Peak6.4 years to LOE
Peak-stage rare disease product with established market presence; commercial team is stable and focused on patient access and market penetration in HT-1 population.
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.