NITISINONE
NITISINONE is indicated for the treatment of Hereditary Tyrosinemia Type 1.
How NITISINONE Works
This drug works by blocking an enzyme called 4-hydroxyphenyl-pyruvate dioxygenase, which is part of the process the body uses to break down tyrosine. By stopping this process early, it prevents the formation of harmful metabolic byproducts that cause organ damage and neurological issues. This helps protect the liver and kidneys while also preventing the porphyric crises associated with the disease.
Details
- Status
- Prescription
- First Approved
- 2019-08-26
- Routes
- ORAL
- Dosage Forms
- CAPSULE
NITISINONE Approval History
What NITISINONE Treats
1 indicationsNITISINONE is approved for 1 conditions since its original approval in 2019. These indications span multiple therapeutic areas including oncology, immunology, and more.
- Hereditary Tyrosinemia Type 1
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Clinical Trial Registry
8 trials| Trial | Sponsor ID | Phase | Status | Title |
|---|---|---|---|---|
| NCT01390077 results posted | WLN02 | Ph 2, Ph 3 | completed | Nitisinone (NTBC) In Different Age Groups Of Patients With Alkaptonuria |
| NCT01838655 results posted | 130124 13-EI-0124 | Ph 1, Ph 2 | completed | Nitisinone for Type 1B Oculocutaneous Albinism |
| NCT02750345 results posted | CT-003 PXL227430 | Ph 1 | completed | Bioequivalence Study of Two Oral Nitisinone Formulations to Treat Hereditary Tyrosinemia (HT-1) |
| NCT02750709 results posted | CT-001 PXL225418 | Ph 1 | completed | Bioequivalence Study of Two Nitisinone Formulations Compared to Orfadin |
| NCT02750332 results posted | CT-002 PXL225421 | Ph 1 | completed | Bioavailability Food-Effect Study of an Oral Nitisinone Formulation to Treat Hereditary Tyrosinemia (HT-1) |
| NCT02323529 HT-1 | Sobi.NTBC-003 | Ph 3 | completed | Efficacy and Safety of Once Daily Dosing Compared to Twice Daily Dosing of Nitisinone in HT-1 |
| NCT01734889 results posted | Sobi.NTBC-002 | Ph 1 | completed | Taste and Palatability of Orfadin Suspension |
| NCT01828463 SONIA1 | UoL000928 | Ph 2 | completed | Dose Response Study of Nitisinone in Alkaptonuria |
Active Pipeline
Ongoing clinical trials by development phase
Key Completed Trials
Completed studies with published results, ranked by significance
Trial Timeline
Full development history with FDA approval milestones
Understanding FDA Approval Types
| Count | Type | What it means |
|---|---|---|
| - | ORIG | Original approval - drug first enters market |
| - | SUPPL - Efficacy | New indication (new disease/condition approved) |
| - | SUPPL - Labeling | Label text changes (warnings, dosing updates) |
| - | SUPPL - Manufacturing | Production changes (new facility) |
| - | SUPPL - Chemistry | Formulation changes (new dosage strength) |
Green lines in the timeline show ORIG and Efficacy approvals - the clinically meaningful milestones.
NITISINONE FDA Label Details
Indications & Usage
NITISINONE is indicated for the treatment of Hereditary Tyrosinemia Type 1.
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ORFADIN
Full clinical data, patents, trials, and competitive landscape for nitisinone.
Data Sources
Data sourced from official FDA and NIH databases. Click links to verify on original sources.