ORFADIN

LOE Approaching

nitisinone

NDAORALCAPSULEPriority Review
Approved
Jan 2002
Lifecycle
LOE Approaching
Competitive Pressure
30/100
Clinical Trials
7

Mechanism of Action

Hydroxyphenylpyruvate Dioxygenase Inhibitors

Pharmacologic Class:

4-Hydroxyphenyl-Pyruvate Dioxygenase Inhibitor

Clinical Trials (5)

NCT06227429N/AWithdrawn

A Non-interventional, Post-Marketing Study to Describe Outcome of Nitisinone Treatment in HT-1 Patients

Started Sep 2025
0
Hereditary Tyrosinemia, Type I
NCT03103568Phase 1Completed

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
NCT02323529Phase 3Completed

Efficacy and Safety of Once Daily Dosing Compared to Twice Daily Dosing of Nitisinone in HT-1

Started Dec 2014
18 enrolled
Hereditary Tyrosinemia, Type I
NCT02320084N/ACompleted

Long Term Safety Study of Orfadin Treatment in HT-1 Patients in Standard Clinical Care

Started Sep 2013
315 enrolled
Hereditary Tyrosinemia, Type I
NCT01857362Phase 1Completed

Bioequivalence of Orfadin 20 mg Compared to Orfadin 10 mg Capsules.

Started May 2013
12 enrolled
Healthy