sapropterin

Generic Name: sapropterin (SAP roe TER in)
Brand Name: Kuvan

What is sapropterin?

Sapropterin is a synthetic form of a natural salt that contributes to the function of a certain enzyme in the body. In people with a condition called phenylketonuria (PKU), this enzyme is lacking or missing in the body. Without the enzyme, phenylalanine levels can become elevated and cause a variety of neurologic symptoms.

Sapropterin is used to help the body activate the enzyme needed to control blood levels of phenylalanine. This medication is used in people with PKU.

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

What is the most important information I should know about sapropterin?

Before using sapropterin, tell your doctor if you are allergic to any drugs, or if you have liver disease, a history of stomach ulcer or bleeding, or a seizure disorder.

Also tell your doctor about any medications you use, especially levodopa (Larodopa, Altamet, Parcopa, Sinemet), methotrexate (Rheumatrex, Trexall), or any medication to treat erectile dysfunction, such as sildenafil (Viagra), tadalafil (Cialis), or vardenafil (Levitra).

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To be sure sapropterin is helping your condition, your blood will need to be tested on a regular basis. Do not miss any scheduled appointments.

Sapropterin 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.

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 if you have any of these symptoms.

Follow your doctor's instructions about any restrictions on food, beverages, or activity while you are using sapropterin.

What should I discuss with my health care provider before taking sapropterin?

Before using sapropterin, tell your doctor if you are allergic to any drugs, or if you have:

  • liver disease;

  • a history of stomach ulcer or bleeding; or

  • epilepsy or other seizure disorder.

If you have any of these conditions, you may not be able to take sapropterin, or you may need a dose adjustment or special tests during treatment.

FDA pregnancy category C. This medication may be harmful to an unborn baby. Tell your doctor if you are pregnant or plan to become pregnant during treatment.

It is not known whether sapropterin passes into breast milk or if it could harm a nursing baby. Do not use this medication without telling your doctor if you are breast-feeding a baby.

How should I take sapropterin?

Take this medication exactly as prescribed by your doctor. Do not take it in larger amounts or for longer than recommended. Follow the directions on your prescription label.

Sapropterin is usually given once daily. Follow your doctor's instructions.

Sapropterin works best if you take it with food.

Dissolve the tablet in 4 to 8 ounces of water or apple juice. It will take about 15 minutes for the tablet to dissolve. Stir this mixture and then drink all of it right away. To make sure you get the entire dose, add a little more water or juice to the same glass, swirl gently and drink right away.

The tablet may not completely dissolve and you may see small pieces in the liquid. These pieces are safe for you to swallow.

To be sure this medication is helping your condition, your blood will need to be tested on a regular basis. Do not miss any scheduled appointments.

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.

Sapropterin 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.

Store sapropterin 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. If it is almost time for your next dose, wait until then to take the medicine and skip the missed 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 if you think you have used too much of this medicine.

Overdose symptoms may include headache and dizziness.

What should I avoid while taking sapropterin?

Follow your doctor's instructions about any restrictions on food, beverages, or activity while you are using sapropterin.

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 any of these signs of an allergic reaction: hives; difficulty breathing; swelling of your face, lips, tongue, or throat.

Call your doctor at once if you have any of the serious side effects below:

  • seizure (convulsions);

  • chest pain or heavy feeling, pain spreading to the arm or shoulder, general ill feeling;

  • easy bruising or bleeding;

  • fever, chills, body aches, flu symptoms;

  • swelling in your hands or feet;

  • pain or burning when you urinate;

  • weakness or fainting;

  • black, bloody, or tarry stools; or

  • coughing up blood or vomit that looks like coffee grounds.

Less serious side effects may include:

  • nausea, vomiting, stomach pain, diarrhea;

  • runny or stuffy nose, cough;

  • headache, feeling agitated;

  • dizziness; or

  • joint pain.

This is not a complete list of side effects and others may occur. Tell your doctor about any unusual or bothersome side effect. You may report side effects to FDA at 1-800-FDA-1088.

See also: Side effects (in more detail)

Sapropterin Dosing Information

Usual Adult Dose for Phenylketonuria:

Initial dose: 10 mg/kg orally once a day with food
Maintenance dose: Once responsiveness established, dose may be adjusted within the range of 5 to 20 mg/kg/day according to response to therapy.

Comments:
-Response to therapy is determined by change in blood phenylalanine (Phe) after treatment with sapropterin (10 mg/kg/day) for up to 1 month.
-Blood Phe levels should be checked after 1 week of therapy and periodically for up to a month.
-If blood Phe does not decrease from baseline at 10 mg/kg/day, dose may be increased to 20 mg/kg/day.
-If blood Phe does not decrease after 1 month at 20 mg/kg/day, patient is a non-responder and sapropterin should be discontinued.

Approved indication: In conjunction with a Phe-restricted diet, to reduce blood Phe levels in patients with hyperphenylalaninemia due to tetrahydrobiopterin- (BH4-) responsive phenylketonuria

Usual Pediatric Dose for Phenylketonuria:

4 years or older:
Initial dose: 10 mg/kg orally once a day with food
Maintenance dose: Once responsiveness established, dose may be adjusted within the range of 5 to 20 mg/kg/day according to response to therapy.

Comments:
-Response to therapy is determined by change in blood Phe after treatment with sapropterin (10 mg/kg/day) for up to 1 month.
-Blood Phe levels should be checked after 1 week of therapy and periodically for up to a month; frequent blood monitoring recommended to ensure adequate blood Phe level control.
-If blood Phe does not decrease from baseline at 10 mg/kg/day, dose may be increased to 20 mg/kg/day.
-If blood Phe does not decrease after 1 month at 20 mg/kg/day, patient is a non-responder and sapropterin should be discontinued.

Approved indication: In conjunction with a Phe-restricted diet, to reduce blood Phe levels in patients with hyperphenylalaninemia due to BH4-responsive phenylketonuria

What other drugs will affect sapropterin?

The following drugs can interact with sapropterin. Tell your doctor if you are using any of these:

  • levodopa (Larodopa, Altamet, Parcopa, Sinemet);

  • methotrexate (Rheumatrex, Trexall);

  • sildenafil (Viagra);

  • tadalafil (Cialis); or

  • vardenafil (Levitra).

This list is not complete and there may be other drugs that can interact with sapropterin. Tell your doctor about all your prescription and over-the-counter medications, vitamins, minerals, herbal products, and drugs prescribed by other doctors. Do not start a new medication without telling your doctor.

Where can I get more information?

  • Your pharmacist can provide more information about sapropterin.
  • 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.
  • Disclaimer: Every effort has been made to ensure that the information provided by Cerner Multum, Inc. ('Multum') is accurate, up-to-date, and complete, but no guarantee is made to that effect. Drug information contained herein may be time sensitive. Multum information has been compiled for use by healthcare practitioners and consumers in the United States and therefore Multum does not warrant that uses outside of the United States are appropriate, unless specifically indicated otherwise. Multum's drug information does not endorse drugs, diagnose patients or recommend therapy. Multum's drug information is an informational resource designed to assist licensed healthcare practitioners in caring for their patients and/or to serve consumers viewing this service as a supplement to, and not a substitute for, the expertise, skill, knowledge and judgment of healthcare practitioners. The absence of a warning for a given drug or drug combination in no way should be construed to indicate that the drug or drug combination is safe, effective or appropriate for any given patient. Multum does not assume any responsibility for any aspect of healthcare administered with the aid of information Multum provides. The information contained herein is not intended to cover all possible uses, directions, precautions, warnings, drug interactions, allergic reactions, or adverse effects. If you have questions about the drugs you are taking, check with your doctor, nurse or pharmacist.

Copyright 1996-2012 Cerner Multum, Inc. Version: 1.03. Revision Date: 2010-12-15, 5:01:39 PM.

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