1 What is Orfadin?

Brand: Orfadin

Generic: Nitisinone

Orfadin is a hydroxyphenyl-pyruvate dioxygenase inhibitor given along with a special diet for the treatment of hereditary tyrosinemia, type 1.

This disease is caused by excess tyrosine in the blood which may cause damage to the liver, kidneys, eyes, skin, and nervous system.

This drug will only slow down the progression of the disease. This drug requires a prescription.

2 What o Know Before Using

Before using Orfadin, you must know all about the risks and complications associated with it. Inform your healthcare provider for any allergic reactions to these drugs or any other drugs, foods, dyes, preservatives, or animals. Carefully read the label of non-prescription drugs for any possible allergic reaction or contraindications.

This drug has not been reported to cause specific problems in children. It is still unknown whether this drug works exactly the same way it does in younger adults or if it causes different side effects or problems in older people.

Data comparing the use of nitisinone in the elderly with use in other age groups are still unavailable. Adverse effects have been reported in animal studies but studies in pregnant women or animals are still inadequate. Infant risk is still undetermined when using this drug during breastfeeding.

Discuss with your healthcare professional about the potential risks and benefits of taking this drug while breastfeeding. Drugs should not be taken together to prevent any interactions but in necessary cases inquire your healthcare provider regarding the adjustments in dosage or any other necessary precautions to prevent any unwanted side effects.

Inform your healthcare professional if you are taking any other drugs such as:

  • Cyclophosphamide

Intake of specific food or using alcohol or tobacco with this drug is associated with an increased risk of certain side effects. Your healthcare professional can give you special instructions about the use of food, alcohol, or tobacco.

3 Proper Usage

To use Orfadin properly, you must follow all instructions given by your doctor. The reaction of this drug with food is still unknown. It is recommended to take this drug at least 1 hour before a meal. Open the capsule and put the drug in a small amount of water, formula, or applesauce before giving it to small children.

The mixture must be given as soon as possible. The dose of this drug will be variable for different patients. The directions on the label or the prescription by your healthcare professional should be followed. The dosage of this drug you take depends on the medical problem for which you are using this drug.

The dose in adults with hereditary tyrosinemia, type 1 is based on body weight and will be determined by your healthcare professional. Your healthcare professional may increase the dose as needed. The dose in children is based on body weight and will be determined by your child's healthcare professional.

Your child's healthcare professional may increase the dose as needed. A missed dose should be taken as soon as possible. However, skip the missed dose and go back to your regular dosing schedule if it is almost time for your next dose.

Store the medicine in a closed container at room temperature, away from heat, moisture, and direct light. Keep from freezing. Keep out of the reach of children.

Dispose any outdated or expired drugs and ask your healthcare professional for the proper disposal of the drugs.

4 Precautions to Take

In using Orfadin, you must be careful and take some precautions as advised by your doctor. It is recommended that you maintain a diet with restricted amounts of tyrosine and phenylalanine.

A nutritionist will be able to recommend a special diet needed to treat you or your child. Call your healthcare professional immediately if you experience any redness, swelling, or burning of your or your child's eyes, an unusual rash, bleeding, or if your or your child's skin is yellow.

Regular visits should be made to your healthcare provider to track your progress and to monitor the effectiveness of the drug. Unwanted effects can be screened using blood tests and eye examinations.

It is recommended to wear sunglasses that block ultraviolet light as this drug may increase your eyes’ sensitivity to the sunlight.

5 Potential Side Effects

As with many medications, there are several potential side effects associated with Orfadin. Side effects may vary for each individual and prompt medical attention should be given if they occur.

Seek advice from your healthcare professional immediately if you experience any unusual symptoms such as:

  • bloated abdomen
  • dark-colored urine
  • dull
  • achy upper abdominal pain
  • the general feeling of tiredness or weakness
  • headache
  • light-colored stools
  • loss of appetite
  • unexplained weight loss
  • vomiting
  • yellow eyes or skin
  • black, tarry stools
  • blindness
  • blood in urine or stools
  • blisters on the skin
  • bloody nose
  • blurred vision
  • change in color vision
  • chest pain or discomfort
  • chills
  • cough
  • darkening of urine
  • decreased vision
  • difficulty seeing at night
  • dry or itching eyes
  • dry skin
  • excessive tearing from eyes
  • eye pain
  • fever
  • fluid-filled skin blisters
  • the general feeling of discomfort or illness
  • increased sensitivity of eyes to sunlight
  • irritation or inflammation of the eye
  • itching of the skin
  • painful or difficult urination
  • pinpoint red spots on the skin
  • rash with flat lesions or small raised lesions on the skin
  • red, thickened, or scaly skin
  • redness, pain, swelling of eye, eyelid, or inner lining of the eyelid
  • burning sensitivity to the sun
  • shortness of breath
  • skin thinness
  • sore throat
  • sores, ulcers, or white spots on lips or in mouth swollen and/or painful glands
  • unusual bleeding or bruising
  • unexplained nosebleed
  • agitation
  • anxiety
  • back pain
  • bloody stools
  • bluish color of fingernails, lips, skin, palms, or nail beds, change in personality
  • change in vision
  • cold sweats
  • coma
  • confusion
  • cool, pale skin
  • a cough producing mucus
  • decreased urination
  • diarrhea
  • difficulty breathing
  • dizziness
  • drowsiness
  • dry mouth
  • earache
  • fainting fast heartbeat
  • feeling full in the upper abdomen
  • the increase in heart rate
  • the increase in body movements
  • increased hunger
  • infection
  • irregular, fast or slow, or shallow breathing
  • irritability
  • lightheadedness
  • mood or mental changes
  • nausea
  • nervousness
  • pain or swelling in arms or legs without any injury
  • pale skin
  • problems with walking or talking
  • rapid breathing
  • redness or swelling in the ear
  • seeing things that are not there
  • shakiness
  • skin rash found mostly on mucous membranes such as eyes and mouth
  • stiff neck
  • sunken eyes
  • thirst
  • tightness in the chest
  • vomiting of blood or material that looks like coffee grounds
  • wheezing, or wrinkled skin

Some side effects may occur that usually do not need medical attention. As your body adjusts to the drug, the side effects will slowly disappear. Ask your healthcare professional about ways to prevent or reduce some of these side effects.

If any of the following side effects persists or are inconvenient, or if you notice any other effects, or if you have any questions about them, consult with your health care professional. Report any side effects to the FDA hotline at 1-800-FDA-1088.