Sapropterin Dihydrochloride Tablets 100 mg

What is sapropterin?

Sapropterin is a synthetic form of a natural substance that activates an enzyme needed to break down phenylalanine in the body.

Phenylketonuria (PKU) is a disorder in which the body cannot break down phenylalanine. Elevated phenylalanine levels can cause problems with thinking and behavior.

Sapropterin is used to lower blood levels of phenylalanine in people with a certain type of PKU.

Sapropterin may also be used for purposes not listed in this medication guide.

Important Information

Follow all directions on your medicine label and package. Tell each of your healthcare providers about all your medical conditions, allergies, and all medicines you use.

Before taking this medicine

You should not use sapropterin if you are allergic to it.

To make sure sapropterin is safe for you, tell your doctor if you have:

liver or kidney disease;

a fever;

a history of stomach ulcer or bleeding;

cancer (and you are being treated with chemotherapy); or

little or no appetite, or if you are malnourished.

It is not known whether sapropterin will harm an unborn baby. Tell your doctor if you are pregnant or plan to become pregnant.

If you are pregnant, your name may be listed on a pregnancy registry. This is to track the outcome of the pregnancy and to evaluate any effects of sapropterin on the baby.

It is not known whether sapropterin passes into breast milk or if it could harm a nursing baby. Tell your doctor if you are breast-feeding a baby.

How should I take sapropterin?

Sapropterin is usually given once daily. Follow all directions on your prescription label. Your doctor may occasionally change your dose to make sure you get the best results. Do not use this medicine in larger or smaller amounts or for longer than recommended.

Read all patient information, medication guides, and instruction sheets provided to you. Ask your doctor or pharmacist if you have any questions.

Sapropterin is not approved for use by anyone younger than 1 month old. Do not give this medicine to a child of any age without medical advice. Sapropterin can cause phenylalanine levels to get too low in children younger than 7 years old who use this medicine.

Older children may be able to use sapropterin for a longer period of time than younger children. Follow your doctor's dosing instructions very carefully.

Sapropterin works best if you take it with food. Take the medicine at the same time each day.

You may swallow the tablet whole or dissolve it in 4 to 8 ounces of water or apple juice.

It will take a few minutes for the tablet to dissolve, and you may see small pieces in the liquid. These pieces are safe for you to swallow.

Stir the mixture and then drink all of it within 15 minutes after the tablet dissolves.

To get the entire dose, add a little more water to the same glass, swirl gently and drink right away.

You may also crush the tablet and mix it with a small amount of soft food such as pudding or applesauce.

The powder for oral solution must be dissolved in 4 to 8 ounces of water or apple juice.

If you are giving this medicine to a baby who weighs less than 22 pounds, you may dissolve the powder in as little as 1 teaspoon of water or apple juice. Then give the liquid to the baby using an oral dosing syringe. If you do not have a dosing syringe, ask your pharmacist for one.

Stir the mixture and then drink all of it within 30 minutes after the powder dissolves.

You may also mix the powder with a small amount of soft food such as pudding or applesauce.

Make sure the powder is completely dissolved before drinking or eating the mixture.

Sapropterin can reduce your phenylalanine blood levels within 24 hours of when you take it. However, it may take up to 30 days of using this medicine before it reaches its fullest effect in your body. For best results, keep using the medication as directed.

Tell your doctor if you have any changes in weight. Sapropterin doses are based on weight (especially in children), and any changes may affect the dose.

Sapropterin is only part of a complete program of treatment that also includes a special diet. Follow the diet plan created for you by your doctor or nutrition counselor. Get familiar with the list of foods you must avoid to help control your condition.

While using sapropterin, you may need frequent blood tests.

Store at room temperature away from moisture and heat. Keep the bottle tightly closed when not in use.

What happens if I miss a dose?

Take the missed dose as soon as you remember. Skip the missed dose if it is almost time for your next scheduled dose. Do not take extra medicine to make up the missed dose.

Do not take 2 doses on the same day.

What happens if I overdose?

Seek emergency medical attention or call the Poison Help line at 1-800-222-1222.

What should I avoid while taking sapropterin?

Follow your doctor's instructions about any restrictions on food, beverages, or activity.

Sapropterin side effects

If sapropterin is not controlling your condition properly, you may have symptoms such as behavior changes, numbness or tingling, or problems with speech, vision, or balance. Call your doctor right away if you have any of these symptoms.

Get emergency medical help if you have signs of an allergic reaction: hives, rash; wheezing, cough, difficult breathing; nausea, feeling light-headed; flushing (warmth, redness, or tingly feeling); swelling of your face, lips, tongue, or throat.

Call your doctor at once if you have:

heartburn or indigestion, severe stomach discomfort, nausea, vomiting;

bloody or tarry stools, coughing up blood or vomit that looks like coffee grounds;

hyperactive behavior;

seizure (convulsions); or

chest pain or pressure, pain spreading to your jaw or shoulder.

Common side effects may include:

headache;

diarrhea;

vomiting; or

runny or stuffy nose, sore throat, cough.

This is not a complete list of side effects and others may occur. Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088.

Sapropterin dosing information

Usual Adult Dose for Phenylketonuria:

Initial dose: 10 to 20 mg/kg orally once a day
-Blood phenylalanine levels should be measured after 1 week, and then periodically; dose should be increased to 20 mg/kg/day if blood phenylalanine levels have not decreased
Maintenance dose: Once responsiveness established, dose may be adjusted within the range of 5 to 20 mg/kg/day according to response to therapy

Non-Responders:
-Patients whose phenylalanine levels do not decrease with treatment at 20 mg/kg/day for 1 month should be considered non-responders and treatment should be discontinued.

Comments:
-This drug should be used in conjunction with a phenylalanine-restricted diet.
-Response to therapy is determined by change in blood phenylalanine levels.

Use: To reduce blood phenylalanine levels in patients with hyperphenylalaninemia due to tetrahydrobiopterin- (BH4-) responsive phenylketonuria (PKU).

Usual Pediatric Dose for Phenylketonuria:

Initial dose:
1 month to 6 years: 10 mg/kg orally once a day
7 years or older: Initial dose: 10 to 20 mg/kg orally once a day
-Blood phenylalanine levels should be measured after 1 week, and then periodically; dose should be increased to 20 mg/kg/day if blood phenylalanine levels have not decreased
Maintenance dose: Once responsiveness established, dose may be adjusted within the range of 5 to 20 mg/kg/day according to response to therapy

Non-Responders:
-Patients whose phenylalanine levels do not decrease with treatment at 20 mg/kg/day for 1 month should be considered non-responders and treatment should be discontinued.

Comments:
-This drug should be used in conjunction with a phenylalanine-restricted diet.
-Response to therapy is determined by change in blood phenylalanine levels.

Use: To reduce blood phenylalanine levels in patients with hyperphenylalaninemia due to tetrahydrobiopterin- (BH4-) responsive phenylketonuria (PKU).

What other drugs will affect sapropterin?

Tell your doctor about all your current medicines and any you start or stop using, especially:

levodopa;

methotrexate;

cancer medicine; or

medicine to treat impotence or pulmonary arterial hypertension--sildenafil (Viagra, Revatio), avanafil (Stendra), tadalafil (Adcirca,Cialis) vardenafil (Levitra, Staxyn).

This list is not complete. Other drugs may interact with sapropterin, including prescription and over-the-counter medicines, vitamins, and herbal products. Not all possible interactions are listed in this medication guide.

Further information

Remember, keep this and all other medicines out of the reach of children, never share your medicines with others, and use this medication only for the indication prescribed.

Always consult your healthcare provider to ensure the information displayed on this page applies to your personal circumstances.

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