Orfadin

Orfadin is a hydroxy-phenylpyruvate dioxygenase inhibitor used to treat patients with hereditary tyrosinemia type 1 (HT-1). HT-1 is a disorder that occurs when the body does not produce the required enzymes to break down the proteins. Orfadin works by breaking down amino acid to prevent toxic substances from building up and damaging the liver or kidneys.

• Orfadin
• Nityr

• Aldurazyme
• Ammonul
• Betaine
• Brineura
• Buphenyl
• Calcijex
• Calcitriol
• Carbaglu
• Carglumic Acid
• Carnitor
• Carnitor SF
• Cinacalcet HCl
• Citrulline Easy
• Crysvita
• Cystadane
• Doxercalciferol
• Elaprase
• Elfabrio
• Fabrazyme
• Galafold
• Hectorol
• Javygtor
• Kanuma
• Kuvan
• Lamzede
• levOCARNitine
• levOCARNitine SF
• Lumizyme
• McCarnitine
• Mepsevii
• Myalept
• Myozyme
• Naglazyme
• Nexviazyme
• Nitisinone
• Nityr
• Nulibry
• Olpruva (2 GM Dose)
• Olpruva (3 GM Dose)
• Olpruva (4 GM Dose)
• Olpruva (5 GM Dose)
• Olpruva (6 GM Dose)
• Olpruva (6.67 GM Dose)
• Opfolda
• Orfadin
• Palynziq
• Paricalcitol
• Parsabiv
• Pheburane
• Pombiliti
• Ravicti
• Rayaldee
• Revcovi
• Rocaltrol
• Sapropterin Dihydrochloride
• Sensipar
• Sod Benz-Sod Phenylacet
• Sodium Phenylbutyrate
• Strensiq
• Vimizim
• Xenpozyme
• Xphozah
• Xuriden
• Zemplar

• Tablet
• Capsule
• Liquid

• By mouth

Take your medicine as directed. Your dose may need to be changed several times to find what works best for you.

Carefully follow your doctor's instructions about a special diet.

Capsule: Take this medicine on an empty stomach at least 1 hour before or 2 hours after a meal.If you cannot swallow the capsule, you may open it and pour the contents into a small amount of water, formula, or applesauce. Stir the mixture well and swallow it without chewing.

Oral liquid: Allow the bottle to warm to room temperature for 30 to 60 minutes.When used the first time, shake the bottle for at least 20 seconds until the solid cake at the bottom of the bottle is completely dissolved.Measure the medicine with an oral syringe and use the bottle adapter.Shake the bottle for at least 5 seconds before measuring each dose to remove any particles at the bottom of the bottle.

Tablet: If you are unable to swallow the tablet whole, you can mix the tablet with water in an oral syringe or crush the tablet and mix with applesauce. Do not mix it with any other food or liquid.To prepare for an applesauce mixture: Wash your hands well before preparing the medicine.Measure about 1 teaspoon of applesauce and put it into a clean container (eg, glass or bowl).Place the tablet onto a spoon. Crush one tablet at a time between 2 spoons until the tablet is crushed to a fine powder. Carefully place the powder into the clean container of applesauce. Make sure that no tablet powder remains on the teaspoon. If more than 1 tablet is needed, repeat the previous steps. Stir the mixture well.Swallow all of the mixture right away or within 2 hours of mixing. Repeat this step until no mixture is left in the container. Measure another 1 teaspoon of applesauce and place it in the container. Stir the fresh applesauce with any remaining tablet powder until mixed well. Swallow this mixture right away. This will make sure any remaining medicine in the container is administered.To prepare for an oral syringe: Wash your hands well before preparing the medicine.Use only 1 or 2 tablets in an oral syringe at a time.For 1 tablet: place the tablet in the oral syringe. Add 2.6 milliliters (mL) of room temperature water and pull the plunger back to the 3 mL mark. For 2 tablets: place the 2 tablets in the oral syringe. Add 5 mL of room temperature water.Replace the cap on the syringe and let it sit for at least 15 minutes for 2 milligrams (mg) tablets and at least 60 minutes for 5 mg or 10 mg tablets.Turn the syringe up and down for at least 30 seconds.Check the mixture in the syringe. The tablet should have broken down into small pieces and are evenly spread in the water (suspension). Do not take the suspension if there are any large pieces of tablet in it or if there are any tablet pieces that are not evenly spread through the water.If the tablet is not fully dissolved, let it sit for another 10 minutes. Then, turn the syringe up and down for at least 30 seconds.Place the tip of the syringe in the mouth. If you are giving it to a child, place the tip of the syringe along the inner cheek of the child's mouth.Slowly push the plunger down until a small amount of air is left in the syringe, between the plunger and the tip. Do not press the plunger all the way down to the end of the syringe. Draw up an additional 2 mL of water into the syringe. Replace the cap on the syringe. Shake well to spread the remaining tablet pieces evenly through the water. If particles are still present, repeat this step one more time.

Read and follow the patient instructions that come with this medicine. Talk to your doctor or pharmacist if you have any questions.

Missed dose: Take a dose as soon as you remember. If it is almost time for your next dose, wait until then and take a regular dose. Do not take extra medicine to make up for a missed dose.

Capsules: Store the medicine in the refrigerator.Oral liquid: Store the medicine in the refrigerator. Do not freeze. Once opened, you may store it at room temperature for up to 60 days. Throw away any unused medicine after 60 days.Tablets: Store the medicine in its original container at room temperature. Keep the tablets and the medicine prepared in an oral syringe and with applesauce away from direct light. Throw away unused mixture within 2 hours after mixing with applesauce or within 24 hours after mixing in the oral syringe.

This medicine is not right for everyone. Do not use it if you had an allergic reaction to nitisinone.

Tell your doctor if you are pregnant or breastfeeding, or if you have liver disease or eye problems.

This medicine may cause the following problems:Eye or vision problemsIntellectual disability and developmental delayHyperkeratotic plaques (skin condition) on the soles and palms

This medicine may make you bleed, bruise, or get infections more easily. Take precautions to prevent illness and injury. Wash your hands often.

Your doctor will do lab tests at regular visits to check on the effects of this medicine. Keep all appointments. You may also need to have regular eye examinations.

Keep all medicine out of the reach of children. Never share your medicine with anyone.

Unusual bleeding, bruising, or weakness

Some medicines can affect how nitisinone works. Tell your doctor if you are using adefovir, celecoxib, ganciclovir, methotrexate, phenytoin, tolbutamide, or warfarin.

This medicine may make you bleed, bruise, or get infections more easily. Take precautions to prevent illness and injury. Wash your hands often.

Your doctor will do lab tests at regular visits to check on the effects of this medicine. Keep all appointments. You may also need to have regular eye examinations.

Keep all medicine out of the reach of children. Never share your medicine with anyone.

• Raised or thickened skin areas on the palms of your hands or soles of your feet
• Problems in intelligence, short-term memory, learning ability, and attention
• Allergic reaction: Itching or hives, swelling in your face or hands, swelling or tingling in your mouth or throat, chest tightness, trouble breathing
• Blurred vision or changes in vision, difficulty seeing at night, increased sensitivity of the eyes to sunlight, pain, redness, swelling, or burning of the eyes
• Fever, chills, cough, sore throat, body aches
• Unusual bleeding, bruising, or weakness, nosebleed
• Headache
• Diarrhea, stomach upset