Orfadin

DRUG DESCRIPTION

Orfadin contains nitisinone, which is a hydroxyphenyl-pyruvate dioxygenase inhibitor indicated as an adjunct to dietary restriction of tyrosine and phenylalanine in the treatment of hereditary tyrosinemia type 1 (HT-1).

Nitisinone occurs as white to yellowish-white, crystalline powder. It is practically insoluble in water, soluble in 2M sodium hydroxide and in methanol, and sparingly soluble in alcohol.

Chemically, nitisinone is 2-(2-nitro-4-trifluoromethylbenzoyl) cyclohexane-1,3-dione, and the structural formula is:

Figure 1. The molecular formula is C14H10F3NO5 with a relative mass of 329.23

ORFADIN®
  (nitisinone) capsules  Structural Formula Illustration

Orfadin (nitisinone) is a hard white-opaque capsule, marked as 2 mg, 5 mg or 10 mg strengths of nitisinone and is intended for oral administration. Each capsule contains 2 mg, 5 mg or 10 mg nitisinone, plus pre-gelatinized starch. The capsule shell is gelatin and titanium dioxide and the imprint is an iron oxide.

What are the possible side effects of nitisinone (Orfadin)?

Stop using nitisinone and get emergency medical help if you have any of these serious side side effects. Some of these side effects can be caused by the medication or by your diet:

  • an allergic reaction (difficulty breathing; closing of your throat; swelling of your lips, tongue, or face; or hives);
  • jaundice (yellowing of the skin or eyes);
  • eye redness, pain, burning, or sensitivity to light;
  • vision changes;
  • bloody or black stools; or
  • unusual bruising or...

Read All Potential Side Effects and See Pictures of Orfadin »

What are the precautions when taking nitisinone (Orfadin)?

Before giving nitisinone, tell the doctor or pharmacist if your child is allergic to it; or if your child has any other allergies. This product may contain inactive ingredients, which can cause allergic reactions or other problems. Talk to your pharmacist for more details.

Before using this medication, tell the doctor or pharmacist your child's medical history, especially of: eye problems (such as cataracts, corneal ulcers).

Patients taking this medication are at risk for developing high levels of tyrosine in the blood, possibly leading to eye, skin, and nervous system problems. (See Side effects section.) To decrease the risk of high tyrosine levels, patients should follow a diet low in tyrosine and phenylalanine. Avoid foods and beverages that contain aspartame or are...

Read All Potential Precautions of Orfadin »


Orfadin Consumer (continued)

SIDE EFFECTS: Remember that the doctor has prescribed this medication because he or she has judged that the benefit is greater than the risk of side effects. Many people using this medication do not have serious side effects.

This medication may rarely cause a low number of blood cells such as white cells and platelets. This effect can decrease your body's ability to fight an infection or cause easy bruising/bleeding. Tell your doctor immediately if you develop any of the following unlikely symptoms: signs of infection (such as fever, chills, persistent sore throat), easy bruising/bleeding.

Your condition and this medication can cause liver problems. Tell the doctor immediately if any of these symptoms of liver problems occur: severe stomach/abdominal pain, persistent nausea or vomiting, yellowing eyes/skin, dark urine.

Your condition and this medication can cause high levels of tyrosine in the blood. Therefore, it is very important to follow the low protein/low tyrosine diet recommended by your doctor. See also Precautions section. Too much tyrosine may cause skin, eye, or brain problems. Tell the doctor immediately if any of these symptoms occur: eye redness/itching/discharge, vision changes, eye pain, eye sensitivity (especially to light), dry/itchy skin, sores on palms of hands/soles of feet, developmental delays (such as holding head up, rolling over, crawling).

A very serious allergic reaction to this drug is rare. However, seek immediate medical attention if you notice any symptoms of a serious allergic reaction, including: rash, itching/swelling (especially of the face/tongue/throat), severe dizziness, trouble breathing.

This is not a complete list of possible side effects. If you notice other effects not listed above, contact the doctor or pharmacist.

In the US -

Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088.

In Canada - Call your doctor for medical advice about side effects. You may report side effects to Health Canada at 1-866-234-2345.

PRECAUTIONS: Before giving nitisinone, tell the doctor or pharmacist if your child is allergic to it; or if your child has any other allergies. This product may contain inactive ingredients, which can cause allergic reactions or other problems. Talk to your pharmacist for more details.

Before using this medication, tell the doctor or pharmacist your child's medical history, especially of: eye problems (such as cataracts, corneal ulcers).

Patients taking this medication are at risk for developing high levels of tyrosine in the blood, possibly leading to eye, skin, and nervous system problems. (See Side effects section.) To decrease the risk of high tyrosine levels, patients should follow a diet low in tyrosine and phenylalanine. Avoid foods and beverages that contain aspartame or are high in tyrosine or phenylalanine. Foods high in tyrosine include soy products, turkey, fish, avocados, bananas, yogurt, and lima beans. Foods high in phenylalanine include milk, chicken, eggs, cheese, peanut butter. (See also Drug Interactions section.)

During pregnancy, this medication should be used only when clearly needed. Consult your doctor before breast-feeding.

It is not known whether this drug passes into breast milk. If you are taking this medication and wish to breast-feed, consult your doctor first.


Orfadin Patient Information Including Side Effects

Brand Names: Orfadin

Generic Name: nitisinone (Pronunciation: ni TIZ i none)

  • What is nitisinone (Orfadin)?
  • What are the possible side effects of nitisinone (Orfadin)?
  • What is the most important information I should know about nitisinone (Orfadin)?
  • What should I discuss with my health care provider before taking nitisinone (Orfadin)?
  • How should I take nitisinone (Orfadin)?
  • What happens if I miss a dose (Orfadin)?
  • What happens if I overdose (Orfadin)?
  • What should I avoid while taking nitisinone (Orfadin)?
  • What other drugs will affect nitisinone (Orfadin)?
  • Where can I get more information?

What is nitisinone (Orfadin)?

Nitisinone is used to treat a rare genetic condition called hereditary tyrosinemia type 1 (HT-1). HT-1 is a metabolic disorder that occurs when the body does not produce enough of an enzyme that breaks down proteins from certain foods. This condition occurs most often in young babies. The symptoms of HT-1 include unusual bleeding and signs of liver failure, such as dark urine, jaundice (yellowing of the skin or eyes), sudden stomach pain, nausea, vomiting, diarrhea, and fatigue.

Nitisinone works by preventing the body from breaking down an amino acid called tyrosine and by keeping other toxic substances from building up and causing harm to your liver or kidneys.

What are the possible side effects of nitisinone (Orfadin)?

Stop using nitisinone and get emergency medical help if you have any of these serious side side effects. Some of these side effects can be caused by the medication or by your diet:

  • an allergic reaction (difficulty breathing; closing of your throat; swelling of your lips, tongue, or face; or hives);
  • jaundice (yellowing of the skin or eyes);
  • eye redness, pain, burning, or sensitivity to light;
  • vision changes;
  • bloody or black stools; or
  • unusual bruising or bleeding.

Other, less serious side effects may be more likely to occur. Continue using nitisinone and talk with your doctor if you have any of these side effects:

  • fever, sore throat, cough, muscle aches;
  • itching or peeling of your skin;
  • diarrhea, nausea, or constipation;
  • nervousness, trouble sleeping;
  • thirst; or
  • mild stomach pain or diarrhea.

Side effects other than those listed here may also occur. Talk to your doctor about any side effect that seems unusual or that is especially bothersome. You may report side effects to FDA at 1-800-FDA-1088.

What is the most important information I should know about nitisinone (Orfadin)?

Nitisinone is only part of a complete program of treatment that also includes a special diet. It is very important to follow the diet plan created for you by your doctor or nutrition counselor. You should become very familiar with the list of foods you must avoid to help control your condition.

You may experience side effects that are caused by nitisinone. Similar side effects may also be caused by your diet. Call your doctor immediately if you have jaundice (yellowing of the skin or eyes), unusual bleeding, vision changes, or eye redness, pain, burning, or sensitivity to light.

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