Hereditary Tyrosinemia, Type I
Pipeline by Development Stage
Competitive Landscape
2 companies ranked by most advanced pipeline stage
Trial Timeline
Clinical trial activity over time
Clinical Trials (7)
Total enrollment: 419 patients across 7 trials
Efficacy and Safety of Once Daily Dosing Compared to Twice Daily Dosing of Nitisinone in HT-1
Bioequivalence Study of Two Oral Nitisinone Formulations to Treat Hereditary Tyrosinemia (HT-1)
Bioavailability Food-Effect Study of an Oral Nitisinone Formulation to Treat Hereditary Tyrosinemia (HT-1)
Bioequivalence Study of Two Nitisinone Formulations Compared to Orfadin
Taste and Palatability of Orfadin Suspension
A Non-interventional, Post-Marketing Study to Describe Outcome of Nitisinone Treatment in HT-1 Patients
Long Term Safety Study of Orfadin Treatment in HT-1 Patients in Standard Clinical Care
Phase Legend
Key Insights
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.