Substance / Medication

Nitisinone

Overview

Active Ingredient
nitisinone
RxNorm CUI
61805

Indications

® NITYRis indicated for the treatment of adult and pediatric patients with hereditary tyrosinemia type 1 (HT-1) in combination with dietary restriction of tyrosine and phenylalanine.

Labeler: Cycle Pharmaceuticals Ltd.Updated: 2025-12-22T00:00:00.000ZFull label on DailyMed

Contraindications

When this intervention should not be used

None.

Indications & Related Conditions

Conditions associated via SNOMED clinical relationships

Administration & Protocol

Dosing, route, and treatment protocol

Detailed dosage and administration information is available in the full FDA drug label.

View full prescribing information on DailyMed

Monitoring & Follow-Up

Biomarkers relevant to this intervention via related conditions

No monitoring biomarkers have been mapped yet. Biomarker-intervention linkages are derived through related conditions and will expand as the knowledge graph grows.

Research Evidence

Published studies and systematic reviews

Sort:
Efficacy and safety of Nitisinone for patients with alkaptonuria: A systematic review with metanalysis.
Mayrink Flávia Diniz, Dorneles Gilson, da Silva Igor Martins et al. · Mol Genet Metab · 2025
PMID: 40157162Meta-Analysis
Evaluation of pre-symptomatic nitisinone treatment on long-term outcomes in Tyrosinemia type 1 patients: a systematic review.
Geppert Julia, Stinton Chris, Freeman Karoline et al. · Orphanet J Rare Dis · 2017
PMID: 28893311Meta-AnalysisFull text (PMC)
Nitisinone Treatment Affects Biomarkers of Bone and Cartilage Remodelling in Alkaptonuria Patients.
Genovese Federica, Frederiksen Peder, Bay-Jensen Anne-Christine et al. · Int J Mol Sci · 2023
PMID: 37446173RCTFull text (PMC)
Radiological evolution of spinal disease in alkaptonuria and the effect of nitisinone.
Imrich Richard, Sedláková Jana, Úlehlová Mária et al. · RMD Open · 2022
PMID: 36270742RCTFull text (PMC)
A 3-year randomized therapeutic trial of nitisinone in alkaptonuria.
Introne Wendy J, Perry Monique B, Troendle James et al. · Mol Genet Metab · 2011
PMID: 21620748RCTFull text (PMC)
Dietary restriction of tyrosine and phenylalanine lowers tyrosinemia associated with nitisinone therapy of alkaptonuria.
Hughes Juliette H, Wilson Peter J M, Sutherland Hazel et al. · J Inherit Metab Dis · 2020
PMID: 31503358ObservationalFull text (PMC)
Efficacy and safety of once-daily nitisinone for patients with alkaptonuria (SONIA 2): an international, multicentre, open-label, randomised controlled trial.
Ranganath Lakshminarayan R, Psarelli Eftychia Eirini, Arnoux Jean-Baptiste et al. · Lancet Diabetes Endocrinol · 2020
PMID: 32822600Observational
Evolution of tyrosinemia type 1 disease in patients treated with nitisinone in Spain.
Couce María Luz, Sánchez-Pintos Paula, Aldámiz-Echevarría Luís et al. · Medicine (Baltimore) · 2019
PMID: 31574857ObservationalFull text (PMC)

Research data from MEDLINE/PubMed

Benefits & Expected Outcomes

Benefits, expected outcomes, efficacy data, and NNT (Number Needed to Treat) are pending physician authorship and evidence review.

Risks & Side Effects

Adverse reaction and safety data for this drug is sourced from the FDA-approved label.

View adverse reactions & drug interactions on DailyMed

Related Symptoms

Symptoms associated with conditions this intervention addresses

No related symptoms have been mapped yet. Symptom linkages are derived through associated conditions.

Alternatives & Comparisons

Alternative treatments, comparison data, and clinical decision support are pending physician authorship.

Medical Disclaimer

This information is for educational purposes only and does not constitute medical advice, diagnosis, or treatment. Always consult with a qualified healthcare provider regarding any medical condition or treatment plan.

Do not start, stop, or change any treatment without consulting your healthcare provider.

Quick Facts

Type
Substance / Medication
Fully Specified Name
Nitisinone (substance)
SNOMED CT
385996000
UMLS CUI
C0173083
RxNorm CUI
61805
Labeler
Cycle Pharmaceuticals Ltd.

Clinical Data

This intervention maps to 1 entities in the Healos knowledge graph.

1
Conditions
0
Biomarkers
0
Specialists
0
Symptoms

Data is sourced from SNOMED CT, UMLS, and the Healos clinical knowledge graph. Content sections marked as pending require physician authorship. Consult a healthcare provider before starting any treatment.