NDAORALTABLET
Approved
Jul 2017
Lifecycle
Peak
Competitive Pressure
0/100
Clinical Trials
9
Data completeness
6/8 — Strong
NITYR (nitisinone) by Medunik is hydroxyphenylpyruvate dioxygenase inhibitors [moa]. Approved for hereditary tyrosinemia type i. First approved in 2017.
Drug data last refreshed 20h ago · AI intelligence enriched 2w ago
AI summarySynthesized from FDA + DailyMed + ClinicalTrials.gov data
NITYR (nitisinone) is an oral small-molecule inhibitor of hydroxyphenylpyruvate dioxygenase approved in 2017 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 tyrosine metabolism, preventing accumulation of toxic metabolites that cause organ damage. The drug addresses a critical unmet need in ultra-rare genetic diseases with limited treatment options.
$0.03BPart D
Peak8.7 years to LOENITYR is in peak commercial phase with modest spending ($30K Part D in 2023), indicating a niche orphan drug with limited team size and stable, predictable market dynamics.
Mechanism of Action
Hydroxyphenylpyruvate Dioxygenase Inhibitors
Pharmacologic Class:
4-Hydroxyphenyl-Pyruvate Dioxygenase Inhibitor
Indications (1)
Clinical Trials (9)
CommunityRx-Kidney Health
Started Mar 2026
634 enrolled
Chronic Kidney Disease (Stages 1-4)
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
Worked on NITYR at Medunik? Share your interview experience or compensation data (+7 days Pro)
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.