Nitisinone

Nitisinone
Adult Indications & Dosage
Pediatric Indications & Dosage
Contraindications
Warnings & Precautions
Adverse Reactions
Drug Interactions
Use in Specific Populations
Administration & Monitoring
Overdosage
Pharmacology
Clinical Studies
How Supplied
Images
Patient Counseling Information
Precautions with Alcohol
Brand Names
Look-Alike Names

Editor-In-Chief: C. Michael Gibson, M.S., M.D. [1];

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Overview

Nitisinone is a Gastrointestinal Agent that is FDA approved for the treatment of hereditary tyrosinemia type 1 (HT-1). Common adverse reactions include hepatic neoplasm, liver failure, thrombocytopenia, leucopenia, visual system complaints including conjunctivitis, corneal opacity, keratitis, and photophobia.

Adult Indications and Dosage

FDA-Labeled Indications and Dosage (Adult)

Indications

• Orfadin(R) capsules (nitisinone) are indicated as an adjunct to dietary restriction of tyrosine and phenylalanine in the treatment of hereditary tyrosinemia type 1

Dosage

• The recommended dose of Orfadin is 1 to 2 mg/kg divided into two daily doses. The initial dose is 1 mg/kg/day divided for morning and evening administration. Treatment with Orfadin should be initiated by a physician experienced in the treatment of HT-1.

The dose of Orfadin may be adjusted in each patient. In patients whose erythrocyte PBG-synthase activity and urine 5-ALA and urine succinylacetone are not normalized within one month after the start of Orfadin treatment, the Orfadin dose may be increased to 1.5 mg/kg/day. In patients receiving 1.5 mg/kg/day, whose erythrocyte PBG-synthase activity and urine 5-ALA and urine succinylacetone remain elevated and whose plasma succinylacetone is not normalized after three months, the dose may be increased to up a maximum dose of 2 mg/kg/day.

If plasma nitisinone concentration, plasma succinylacetone, urine 5-ALA and erythrocyte PBG-synthase activity are not available, clinical laboratory assessments should include urine succinyl acetone, liver function tests, alpha fetoprotein, and serum tyrosine and phenylalanine level. During initiation of therapy and during acute exacerbations, it may be necessary to follow more closely all available biochemical parameters

3 DOSAGE FORMS AND STRENGTHS Orfadin capsules (nitisinone) are available as white capsules imprinted with ”NTBC” followed by "2 mg", "5 mg", or "10 mg", indicating the actual amount of nitisinone in each capsule.

Off-Label Use and Dosage (Adult)

Guideline-Supported Use

There is limited information regarding Off-Label Guideline-Supported Use of Nitisinone in adult patients.

Non–Guideline-Supported Use

There is limited information regarding Off-Label Non–Guideline-Supported Use of Nitisinone in adult patients.

Pediatric Indications and Dosage

FDA-Labeled Indications and Dosage (Pediatric)

Indications

• Orfadin(R) capsules (nitisinone) are indicated as an adjunct to dietary restriction of tyrosine and phenylalanine in the treatment of hereditary tyrosinemia type 1 (HT-1)

Off-Label Use and Dosage (Pediatric)

Guideline-Supported Use

There is limited information regarding Off-Label Guideline-Supported Use of Nitisinone in pediatric patients.

Non–Guideline-Supported Use

There is limited information regarding Off-Label Non–Guideline-Supported Use of Nitisinone in pediatric patients.

Contraindications

• Condition1

Warnings

• Description

Precautions

• Description

Adverse Reactions

Clinical Trials Experience

There is limited information regarding Clinical Trial Experience of Nitisinone in the drug label.

Body as a Whole

Cardiovascular

Digestive

Endocrine

Hematologic and Lymphatic

Metabolic and Nutritional

Musculoskeletal

Neurologic

Respiratory

Skin and Hypersensitivy Reactions

Special Senses

Urogenital

Miscellaneous

Postmarketing Experience

There is limited information regarding Postmarketing Experience of Nitisinone in the drug label.

Body as a Whole

Cardiovascular

Digestive

Endocrine

Hematologic and Lymphatic

Metabolic and Nutritional

Musculoskeletal

Neurologic

Respiratory

Skin and Hypersensitivy Reactions

Special Senses

Urogenital

Miscellaneous

Drug Interactions

• Drug

• Description

Use in Specific Populations

Pregnancy

Pregnancy Category (FDA):

• Pregnancy Category

Pregnancy Category (AUS):

• Australian Drug Evaluation Committee (ADEC) Pregnancy Category

There is no Australian Drug Evaluation Committee (ADEC) guidance on usage of Nitisinone in women who are pregnant.

Labor and Delivery

There is no FDA guidance on use of Nitisinone during labor and delivery.

Nursing Mothers

There is no FDA guidance on the use of Nitisinone with respect to nursing mothers.

Pediatric Use

There is no FDA guidance on the use of Nitisinone with respect to pediatric patients.

Geriatic Use

There is no FDA guidance on the use of Nitisinone with respect to geriatric patients.

Gender

There is no FDA guidance on the use of Nitisinone with respect to specific gender populations.

Race

There is no FDA guidance on the use of Nitisinone with respect to specific racial populations.

Renal Impairment

There is no FDA guidance on the use of Nitisinone in patients with renal impairment.

Hepatic Impairment

There is no FDA guidance on the use of Nitisinone in patients with hepatic impairment.

Females of Reproductive Potential and Males

There is no FDA guidance on the use of Nitisinone in women of reproductive potentials and males.

Immunocompromised Patients

There is no FDA guidance one the use of Nitisinone in patients who are immunocompromised.

Administration and Monitoring

Administration

Orfadin should be taken at least one hour before or at least two hours after a meal, since food effect is unknown. For young children, Orfadin capsules may be opened and the contents suspended in a small amount of water immediately before use.

Physicians should counsel patients and their parents or caregivers of the need to maintain dietary restriction of tyrosine and phenylalanine when taking Orfadin to treat hereditary tyrosinemia type 1.

Monitoring

There is limited information regarding Monitoring of Nitisinone in the drug label.

• Description

IV Compatibility

There is limited information regarding IV Compatibility of Nitisinone in the drug label.

Overdosage

Acute Overdose

Signs and Symptoms

• Description

Management

• Description

Chronic Overdose

There is limited information regarding Chronic Overdose of Nitisinone in the drug label.

Pharmacology

There is limited information regarding Nitisinone Pharmacology in the drug label.

Mechanism of Action

Structure

File:Nitisinone01.png

This image is provided by the National Library of Medicine.

Pharmacodynamics

There is limited information regarding Pharmacodynamics of Nitisinone in the drug label.

Pharmacokinetics

There is limited information regarding Pharmacokinetics of Nitisinone in the drug label.

Nonclinical Toxicology

There is limited information regarding Nonclinical Toxicology of Nitisinone in the drug label.

Clinical Studies

There is limited information regarding Clinical Studies of Nitisinone in the drug label.

How Supplied

Storage

There is limited information regarding Nitisinone Storage in the drug label.

Images

Drug Images

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Package and Label Display Panel

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Patient Counseling Information

There is limited information regarding Patient Counseling Information of Nitisinone in the drug label.

Precautions with Alcohol

• Alcohol-Nitisinone interaction has not been established. Talk to your doctor about the effects of taking alcohol with this medication.

Brand Names

• ®

Look-Alike Drug Names

• A® — B®

Drug Shortage Status

Price

References

The contents of this FDA label are provided by the National Library of Medicine.

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