TheraRadar
Data updated: May 26, 2026

HARLIKU (nitisinone)

Hydroxyphenylpyruvate Dioxygenase Inhibitors
Rare Disease Approved 2017-07-26

Harliku helps adult patients with alkaptonuria by lowering the levels of homogentisic acid found in their urine. This medication is used to manage the metabolic buildup associated with the condition. It is prescribed specifically for adults to address the accumulation of this acid within the tyrosine breakdown process.

Source: FDA Label • CYCLE • 4-Hydroxyphenyl-Pyruvate Dioxygenase Inhibitor

How HARLIKU Works

This drug works by blocking an enzyme called hydroxyphenyl-pyruvate dioxygenase, which is located upstream in the tyrosine catabolic pathway. By inhibiting this enzyme, the medication prevents the production of homogentisic acid that would otherwise accumulate in patients.

3
Indications
--
Phase 3 Trials
1
Priority Reviews
8
Years on Market

Details

Status
Prescription
First Approved
2017-07-26
Patent Cliff
2035

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Routes
ORAL
Dosage Forms
TABLET

Companies

Active Ingredient: NITISINONE

HARLIKU Approval History

2018
2019
2020
2021
2022
2023
2024
2025
2026
Original
New Indication
New Form
Label Update
5 FDA actions from 2017 to 2025 · 2 indication expansions
Jun 2025 SUPPL Priority
Efficacy
Jan 2024 SUPPL
Label · Labeling
Sep 2020 SUPPL
Efficacy

What HARLIKU Treats

1 indications

HARLIKU is approved for 1 conditions since its original approval in 2017. These indications span multiple therapeutic areas including oncology, immunology, and more.

  • Alkaptonuria
Source: FDA Label
📋

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
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Active Pipeline

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Key Completed Trials

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Completed studies with published results, ranked by significance

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📊

Trial Timeline

Full development history with FDA approval milestones

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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.

HARLIKU FDA Label Details

Indications & Usage

FDA Label (PDF)

HARLIKU is indicated for the treatment of Alkaptonuria.

View full patent landscape →
1 OB patents · 1 families · 19 international docs across 16 countries

HARLIKU Patents & Exclusivity

Latest Patent: Jan 2035

Patents (1 active)

US10328029 Expires Jan 5, 2035
Source: FDA Orange Book

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Patent Timeline

  • Cliff: 2035
  • 3 active patents

Trial Analysis

  • Clinical trial tracking
  • Development stage analysis

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Data Sources

Data sourced from official FDA and NIH databases. Click links to verify on original sources.