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Drug

Nitisinone

Approved · EMA
Emerging researchLate-stage development
Also known as Harliku, Nityr, Orfadin, Nitisinona+3 more

Harliku, Nityr, Orfadin, Nitisinona, 2(2N-4tri-fl-m-benz)1,3-cyclohexanedione, 2-(alpha,alpha,alpha-Trifluoro-2-nitro-p-tuluoyl)-1,3-cyclohexanedione, Nitisinonum.

RxNorm61805ChEMBLCHEMBL1337UNIIK5BN214699
2
Research papers
2
Regulatory events
Latest activity
beta

Recent clinical, regulatory, research and industry developments relating to this drug.

A Study to Evaluate the Bioequivalence of Orfadin Capsules 20 mg Compared to Orfadin Capsules 10 mg. An Open-label, Randomized, Cross-over, Single-dose Study in Healthy Volunteers.

Clinical trial2021-10-12Results posted · ClinicalTrials.gov

Long-Term Clinical Trial of Nitisinone in Alkaptonuria

Clinical trial2021-08-26Results posted · ClinicalTrials.gov

Nitisinone (NTBC) In Different Age Groups Of Patients With Alkaptonuria

Clinical trial2021-04-22Results posted · ClinicalTrials.gov

Orfadin and Nitinosine Study

Clinical trial2020-12-31Primary completion · ClinicalTrials.gov

A Pilot Study of Nitisinone in the Treatment of Oculocutaneous Albinism, Type 1B

Clinical trial2019-02-26Results posted · ClinicalTrials.gov

One-year pilot study on the effects of nitisinone on melanin in patients with OCA-1B.

Research2019-01-24JCI insight

Approval: Nityr (EMA)

Regulatory2018-07-26EMA

A Three-Period Crossover Study to Determine the Bioequivalence of Two Oral Formulations Containing Nitisinone 10 mg Compared to Reference Formulation Orfadin In Healthy Subjects Under Fasting Conditions

Clinical trial2017-06-14Results posted · ClinicalTrials.gov

A Single Center, Single-Dose, Open-Label, Laboratory-Blind, Randomized, Three-Period Crossover Study to Determine the Bioequivalence of Two Oral Formulations Containing of Nitisinone 10 mg Compared to the Reference Formulation Orfadin 10 mg in at Least 18 Healthy Male and Female Subjects Under Fasting Conditions

Clinical trial2017-03-15Results posted · ClinicalTrials.gov

A Single Center, Single-Dose, Open-Label, Randomized Study to Compare the Bioavailability of an Oral Test Formulation Containing Nitisinone 10 mg in at Least 16 Healthy Male and Female Subjects Under Fasting and Fed Conditions

Clinical trial2017-03-15Results posted · ClinicalTrials.gov

Metabolic Effects of Increasing Doses of Nitisinone in the Treatment of Alkaptonuria.

Research2015-02-10JIMD reports

Approval: Orfadin (EMA)

Regulatory2005-02-21EMA

Profile

Identifiers & mechanism

Canonical identifiers, marketed brand names and mechanism, resolved across RxNorm, ChEMBL and ATC.

Canonical name
Nitisinone
Aliases & brands
HarlikuNityrOrfadinNitisinona2(2N-4tri-fl-m-benz)1,3-cyclohexanedione2-(alpha,alpha,alpha-Trifluoro-2-nitro-p-tuluoyl)-1,3-cyclohexanedioneNitisinonum
RxNorm CUI
61805
ChEMBL ID
CHEMBL1337
UNII
K5BN214699
Regulatory jurisdictions
ema

Regulatory timeline

2 events

The complete regulatory record, grouped by authority — approvals, safety advisories and label changes. Each authority shows its most recent events; expand one to read its full history.

Earliest approval
2005-02-21
Latest approval
2018-07-26
Authorities
EMA
Total events
2
emaEuropean Medicines Agency· 2 events
2018-07-26Approval
Approval: Nityr (EMA)

Indication: Treatment of adult and paediatric patients with confirmed diagnosis of hereditary tyrosinemia type 1 (HT-1) in combination with dietary restriction of tyrosine and phenylalanine.

Evidence ↗
2005-02-21Approval
Approval: Orfadin (EMA)
Indication: Hereditary tyrosinemia type 1 (HT 1)Orfadin is indicated for the treatment of adult and paediatric (in any age range) patients with confirmed diagnosis of hereditary tyrosinemia… Show full indication

Hereditary tyrosinemia type 1 (HT 1)Orfadin is indicated for the treatment of adult and paediatric (in any age range) patients with confirmed diagnosis of hereditary tyrosinemia type 1 (HT 1) in combination with dietary restriction of tyrosine and phenylalanine. Alkaptonuria (AKU)Orfadin is indicated for the treatment of adult patients with alkaptonuria (AKU).

Evidence ↗

Contains information from the European Medicines Agency (European Medicines Agency), © EMA, reused under CC BY 4.0.

Clinical trials

14 trials

The current development programme across all trial phases — status mix, phase distribution and the late-stage studies shaping the evidence base.

Development programme
CLINICALTRIALS.GOV · LIVE REGISTRY
14
registered trials across all phases
LATEST COMPLETION 2017
5
Late-stage (III+)
12
Completed
2
Discontinued
PHASE DISTRIBUTIONn = 14
Phase 16Phase 1 / 21Phase 22Phase 2 / 31Phase 32Phase N / A2

Late-stage studies

Phase III+ trials still open or recently active — where late-stage evidence is being generated.

An International, Multicentre, Randomised, Evaluator-blind, No-treatment Controlled, Parallel-group Study to Assess the Efficacy and Safety of Once Daily Nitisinone in Patients With Alkaptonuria After 12 Months of Treatment, Followed by an Additional 36 Month Treatment Period.

Unknown statusPH IIIUniversity of Liverpooln=1402020

Nitisinone (NTBC) In Different Age Groups Of Patients With Alkaptonuria

CompletedPH II/IIIUniversity of California, San Diegon=82016

Open-label, Multicentre, Multiple-dose Trial to Evaluate Pharmacokinetics, Efficacy and Safety of Once Daily Dosing Compared to Twice Daily Dosing of Orfadin in Patients Diagnosed With Hereditary Tyrosinemia Type 1

CompletedPH IIISwedish Orphan Biovitrumn=182015

Recent completions

Trials that read out recently, adding to the completed evidence base.

A Pilot Study of Nitisinone in the Treatment of Oculocutaneous Albinism, Type 1B

CompletedPH I/IINational Eye Institute (NEI)n=52017

Nitisinone (NTBC) In Different Age Groups Of Patients With Alkaptonuria

CompletedPH II/IIIUniversity of California, San Diegon=82016

A Three-Period Crossover Study to Determine the Bioequivalence of Two Oral Formulations Containing Nitisinone 10 mg Compared to Reference Formulation Orfadin In Healthy Subjects Under Fasting Conditions

CompletedPH ICycle Pharmaceuticals Ltd.n=242016

A Single Center, Single-Dose, Open-Label, Randomized Study to Compare the Bioavailability of an Oral Test Formulation Containing Nitisinone 10 mg in at Least 16 Healthy Male and Female Subjects Under Fasting and Fed Conditions

CompletedPH ICycle Pharmaceuticals Ltd.n=202016

A Single Center, Single-Dose, Open-Label, Laboratory-Blind, Randomized, Three-Period Crossover Study to Determine the Bioequivalence of Two Oral Formulations Containing of Nitisinone 10 mg Compared to the Reference Formulation Orfadin 10 mg in at Least 18 Healthy Male and Female Subjects Under Fasting Conditions

CompletedPH ICycle Pharmaceuticals Ltd.n=242016

Open-label, Multicentre, Multiple-dose Trial to Evaluate Pharmacokinetics, Efficacy and Safety of Once Daily Dosing Compared to Twice Daily Dosing of Orfadin in Patients Diagnosed With Hereditary Tyrosinemia Type 1

CompletedPH IIISwedish Orphan Biovitrumn=182015

Research activity

2 papers

Key research shaping understanding of this drug, combining the latest publications with the most influential evidence.

Publications over time
20152019
Journals, researchers & institutions
Top journals
  • JCI insight1
  • JIMD reports1
Leading researchers
  • Abraham M1
  • Adams DR1
  • Alur RP1
  • Barshop BA1
  • Bernstein S1
  • Brooks BP1
  • Chiang PW1
  • Cunningham D1
Leading institutions
free-text, unnormalised
  • Biochemical Genetics and Metabolomics Laboratory1
  • Fujita Health University School of Health Sciences1
  • Molecular Vision Laboratory1
  • National Eye Institute and.1
  • National Human Genome Research Institute1
  • National Institute of Neurological Disease and Stroke1
References & data sources
  • RxNorm (U.S. National Library of Medicine) — drug identity
  • ChEMBL (EMBL-EBI) & UniProt — pharmacology and targets
  • Europe PMC — research literature
  • ClinicalTrials.gov — clinical trials
  • Regulatory event sources are credited in the Regulatory Timeline above.