Class Name(s):

• 92:92 Other Miscellaneous Therapeutic Agents

Pronunciation:

• nye tis' i none

Brand Name(s):

• Harliku^®
• Nityr ^®
• Orfadin^®

WHY is this medicine prescribed?

Nitisinone is used to treat hereditary tyrosinemia type 1 (HT-1; rare genetic disorder where body cannot break down amino acid tyrosine causing build-up that can harm kidneys and liver) and reduction of urine homogentisic acid (HGA) in patients with alkaptonuria (AKU; rare genetic disorder where body can't breakdown aminoacids tyrosine and phenylalanine leading to builld up of HGA causing deposits in connective tissue causing severe arthritis symptoms). Nitisinone is in a class of medications called hydroxyphenyl-pyruvate dioxygenase inhibitors. It works by by helping the body eliminate the toxic substances that build-up during HT-1 and AKU.

HOW should this medicine be used?

Nitisinone comes as a tablet, capsule and suspension (liquid) to take by mouth. Take tablets and suspension with or without food 1-2 times a day depending on the product prescribed. Take capsules capsule one hours before or two hours after a meal 2 times a day. Take nitisinone at around the same time(s) every day.

Take nitisinone exactly as directed. Do not take more or less of it or take it more often than prescribed by your doctor.

If unable to swallow the tablets, they may be dissolved in water and given with an oral syringe or crushed and mixed with applesauce.

To give tablet with an oral syringe, follow these steps:

1. Take the plunger out of an oral syringe and place a 1-2 tablets in the syringe (based on prescription).
2. Replace the plunger and draw up 2.6 mL (for 1 tablet) or 5 mL (for 2 tablets) of room teperature water.
3. Cap the syringe and leave for 15 to 60 minutes depending on the tablet strength (15 minutes for 2 mg tablet and 60 minutes for 5 or 10 mg tablets).
4. After the right amount of time has passed, turn the syringe up and down for 30 seconds to mix. Then look at syringe closely to make sure tablet dissolved. If not dissolved, leave syringe for 10 more minutes and repeat mixing.
5. Remove syringe cap and give contents of syringe by mouth leaving a small gap at the end of the syringe. Draw up 2 mL more of room temperature water, mix again and take by mouth pushing the plunger all the way to the end.
6. Once mixed in the syringe, the suspension can be stored at room tempature for 24 hours. If the mixture has not been taken by 24 hours, then it must be discrded

To give tablet with applesauce, follow these steps:

• Crush 1 tablet at a time between two teaspoons to form a fine powder.
• Place fine powder of crushed tablets into 1 teaspoon of applesauce placed in a clean container and mix well.
• Take the entire applesauce mixture within 2 hours of mixing.
• After giving to the patient, place another teaspoon of applesauce into the container and mix and take the entire mixture.

Shake the suspension well before each use to mix the medication evenly.

To give the suspension, read and follow the instructions for use provided with the prescription closely. Ask your doctor or pharmacist for help if there are any parts you do not understand.

Your doctor may start you on a low dose of nitisinone and gradually increase your dose.

Nitisinone controls HT-1 and AKU but does not cure it. Continue to take nitisinone even if you feel well. Do not stop taking nitisinone without talking to your doctor.

Ask your pharmacist or doctor for a copy of the manufacturer's information for the patient.

Are there OTHER USES for this medication?

This medication may be prescribed for other uses; ask your doctor or pharmacist for more information.

What SPECIAL PRECAUTIONS should I follow?

Before taking nitisinone,

• tell your doctor or pharmacist if you are allergic to this medication, any part of this medication, or any other medications, foods or substances. Tell your doctor or pharmacist about the allergy and what symptoms you had.
• tell your doctor and pharmacist what prescription and nonprescription medications, vitamins, nutritional supplements, and herbal products you are taking or plan to take while taking nitisinone. Your doctor may need to change the doses of your medications or monitor you carefully for side effects.
• tell your doctor if you have or have ever had eye conditions, bleeding disorders, or a chronic infection.
• tell your doctor if you are pregnant, plan to become pregnant, or are breast-feeding. If you become pregnant while taking nitisinone, call your doctor.
• if you are having surgery, including dental surgery, tell the doctor or dentist that you are taking nitisinone.

What SPECIAL DIETARY instructions should I follow?

Your doctor may tell you to maintain a diet low in tyrosine and phenylalaninine containing foods (avoid high protein foods such as meat, poultry, fish, eggs, dairy, beans and legumes, nuts and seeds, and soy) while takiing nitisinone. Follow this diet very closely.

Unless your doctor tells you otherwise, continue your normal diet.

What should I do IF I FORGET to take a dose?

Take the missed dose as soon as you remember it. However, if it is almost time for the next dose, skip the missed dose and continue your regular dosing schedule. Do not take a double dose to make up for a missed one.

What SIDE EFFECTS can this medicine cause?

Nitisinone may cause side effects. Tell your doctor if any of these symptoms are severe or do not go away:

• nose bleeds
• itching or dry skin
• thin or loss of hair

Some side effects can be serious. If you experience any of these symptoms, call your doctor immediately:

• sensitivity to light; eye pain; red, swollen or burning or itchy eyes; blurry vision; excessive tears or eye discharge or crusty eyes
• painful thickened, raised patches of skin on soles or palms of sports
• fever and/or chills; cough; shortness of breath; painful or difficulty urinating; sore throat; weakness or fatigue
• easy bruising; bleeding gums or nose bleeds; heavy periods; taking longer to stop bleeding after cuts or scrapes; blood in urine or stool

Nitisinone may cause other side effects. Call your doctor if you have any unusual problems while taking this medication.

If you experience a serious side effect, you or your doctor may send a report to the Food and Drug Administration's (FDA) MedWatch Adverse Event Reporting program online (http://www.fda.gov/Safety/MedWatch) or by phone (1-800-332-1088).

What should I do in case of OVERDOSE?

In case of overdose, call the poison control helpline at 1-800-222-1222. Information is also available online at http://www.poisonhelp.org/help. If the victim has collapsed, had a seizure, has trouble breathing, or can't be awakened, immediately call emergency services at 911.

What should I know about STORAGE and DISPOSAL of this medication?

Keep this medication in the container it came in, tightly closed, and out of reach of children. Store tabletsat room temperature and away from excess heat and moisture (not in the bathroom). Store capsules in refrigerator (not in freezer) or can be stored at room temperature, away frome xcess heat and moisture, for 45 days. Supension should be stored in refrigerator until opened for use. Once opened for use, store at room temperature, away from excess heat and moisture, for up to 60 days.

Keep all medication out of sight and reach of children as many containers are not child-resistant. Always lock safety caps. Place the medication in a safe location - one that is up and away and out of their sight and reach. http://www.upandaway.org

Dispose of unneeded medications in a way so that pets, children, and other people cannot take them. Do not flush this medication down the toilet. Use a medicine take-back program. Talk to your pharmacist about take-back programs in your community. Visit the FDA's Safe Disposal of Medicines website http://goo.gl/c4Rm4p for more information.

What OTHER INFORMATION should I know?

Keep all appointments with your doctor and the laboratory. Your doctor will order certain lab tests to check your body's response to nitisinone.

Do not let anyone else take your medication. Ask your pharmacist any questions you have about refilling your prescription.

Keep a written list of all of the prescription and nonprescription (over-the-counter) medicines, vitamins, minerals, and dietary supplements you are taking. Bring this list with you each time you visit a doctor or if you are admitted to the hospital. You should carry the list with you in case of emergencies.

This report on medications is for your information only, and is not considered individual patient advice. Because of the changing nature of drug information, please consult your physician or pharmacist about specific clinical use.

The American Society of Health-System Pharmacists, Inc. represents that the information provided hereunder was formulated with a reasonable standard of care, and in conformity with professional standards in the field. The American Society of Health-System Pharmacists, Inc. makes no representations or warranties, express or implied, including, but not limited to, any implied warranty of merchantability and/or fitness for a particular purpose, with respect to such information and specifically disclaims all such warranties. Users are advised that decisions regarding drug therapy are complex medical decisions requiring the independent, informed decision of an appropriate health care professional, and the information is provided for informational purposes only. The entire monograph for a drug should be reviewed for a thorough understanding of the drug's actions, uses and side effects. The American Society of Health-System Pharmacists, Inc. does not endorse or recommend the use of any drug. The information is not a substitute for medical care.

AHFS^® Patient Medication Information™ Copyright, 2025. The American Society of Health-System Pharmacists^®, 4500 East-West Highway, Suite 900, Bethesda, Maryland. All Rights Reserved. Duplication for commercial use must be authorized by ASHP.

Selected Revisions: September 15, 2025.