NITYR (nitisinone)
NITYR helps adults and children who have hereditary tyrosinemia type 1 (HT-1). It is used in combination with a restricted diet that limits the intake of tyrosine and phenylalanine. This medication helps prevent the serious liver and kidney damage often associated with this condition by managing how the body processes certain amino acids.
How NITYR Works
This medication works by blocking an enzyme called 4-hydroxyphenyl-pyruvate dioxygenase, which is involved in the breakdown of tyrosine. By stopping this process early, the drug prevents the accumulation of toxic byproducts that otherwise cause liver toxicity, kidney damage, and neurological crises.
Development Insights
Details
- Status
- Prescription
- First Approved
- 2017-07-26
- Patent Cliff
- 2035
- Routes
- ORAL
- Dosage Forms
- TABLET
NITYR Approval History
What NITYR Treats
1 indicationsNITYR is approved for 1 conditions since its original approval in 2017. 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.
NITYR FDA Label Details
Indications & Usage
FDA Label (PDF)NITYR is indicated for the treatment of Hereditary Tyrosinemia Type 1.
NITYR Patents & Exclusivity
Patents (1 active)
Pro Intelligence Preview
Deep insights for NITYR
Revenue Insights
- • Quarterly revenue tracking
- • Historical trend analysis
Patent Timeline
- • Cliff: 2035
- • 9 active patents
Trial Analysis
- • 8 total trials
- • Stage: Declining
Competitive Landscape
- • 2 similar drugs
- • Same target/indication analysis
Full approval history • All patents • Revenue trends • Competitor analysis
Data Sources
Data sourced from official FDA and NIH databases. Click links to verify on original sources.
How We Calculate These Metrics
Trial Activity Stage
Measures the current development activity pattern based on trial phases, status, and trends. Important: This measures R&D activity, not commercial lifecycle.
Trial statuses: "Active" means recruiting or ongoing. "Completed" means reached planned endpoint. "Terminated" means stopped early—often due to safety, efficacy, or business reasons.
- Growth: High proportion of early-phase trials (Phase 1/2), active development
- Expansion: Significant Phase 3 activity, approaching or pursuing approvals
- Mature: Substantial Phase 4 post-marketing studies
- Stable: Mixed phase distribution, steady development
- Declining: Low active trial ratio, reduced R&D investment