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Nitisinone Side Effects

The most commonly reported side effects of nitisinone include amino acid level increased, liver transplant, and hepatocellular carcinoma, based on 969 FDA adverse event reports from 2004 to 2025.

Nitisinone side effects

Percentages show how often each reaction appears relative to total reports for nitisinone.

1
Amino Acid Level Increased13.7%133
2
Liver Transplant8.8%85
3
Hepatocellular Carcinoma6.5%63
4
Cataract4.9%47
5
Off Label Use4.6%45
6
Liver Transplant4.4%43
7
Alpha 1 Foetoprotein Increased4.2%41
8
Treatment Noncompliance4.1%40
9
Death3.6%35
10
Vomiting3.6%35
11
Lenticular Opacities3.5%34
12
Succinylacetone Increased3.4%33
13
Hepatic Failure3.2%31
14
Abdominal Pain3.0%29
15
Hepatic Cirrhosis2.5%24

These are voluntary reports and do not establish that nitisinone caused these reactions.

Report severity

73.5%Serious712 reports
31.1%Hospitalizations301 reports
16.5%Fatal160 reports

Seriousness is determined by the reporter, not by OpenClaim.

Nitisinone drug interactions

Other drugs that appear in adverse event reports alongside nitisinone. Drugs flagged as a suspected cause appear in the first column. Drugs taken at the same time but not suspected appear in the second.

Also suspected

1
Omeprazole0.7%7
2
Tacrolimus0.4%4
3
Oxycodone0.3%3
4
Esomeprazole0.3%3
5
Calcium0.3%3
6
Alendronate-sodium0.3%3
7
Acetaminophen0.2%2
8
Fluticasone0.2%2
9
Lamotrigine0.2%2
10
Rufinamide0.2%2
11
Clobazam0.2%2
12
Topiramate0.2%2
13
Levetiracetam0.2%2
14
Lansoprazole0.2%2
15
Ergocalciferol0.2%2

Taken alongside

1
Ergocalciferol3.6%35
2
Omeprazole3.0%29
3
Acetaminophen2.9%28
4
Albuterol2.2%21
5
Aspirin1.8%17
6
Ibuprofen1.8%17
7
Ascorbic-acid1.5%15
8
Tramadol-hydrochloride1.4%14
9
Cetirizine-hydrochloride1.4%14
10
Spironolactone1.3%13
11
Bisoprolol-fumarate1.3%13
12
Furosemide1.2%12
13
Gabapentin1.2%12
14
Escitalopram-oxalate1.2%12
15
Cholecalciferol1.1%11

Co-occurrence in adverse event reports does not establish a drug interaction. Patients often take multiple medications, and these lists reflect prescribing patterns rather than causal relationships. Consult a healthcare provider about potential drug interactions.

Who reports nitisinone side effects

38.2% of nitisinone adverse event reports involve female patients and 42.8% involve male patients. The largest age group is child at 39%. These figures reflect who reports side effects, not underlying risk.

Sex

Female38.2%
Male42.8%
Unknown19.0%

Age group

< 223.2%
2–1138.7%
12–1712.7%
18–6421.4%
65+4.0%

What is nitisinone used for

Conditions and purposes for which patients were taking nitisinone when the adverse event was reported.

AlbinismAlkaptonuriaAmino Acid Metabolism DisorderAlkaptonuriaAmino Acid Metabolism DisorderBlood Amino Acid Level IncreasedChronic Kidney DiseaseGlioblastoma MultiformeInborn Error Of Amino Acid MetabolismLiver DisorderMetabolic DisorderMetabolic DisorderNeoplasm MalignantNeuroblastomaOcular Albinism

Showing 15 of 24 indications

Nitisinone brand names and reporting trend

Nitisinone is sold under the brand name Nityr.

Brand names

Nityr219

Quarterly reports (20042025)

20052010201520202025

An upward trend reflects increased FAERS reporting overall, not necessarily increased risk.

Taking nitisinone with another medication?

Ask OpenClaim about your specific drug combination. Get a sourced report using FDA data, drug labels, and medical literature.