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Sapropterin - Oral

Patient Medicine Information
Why do I need this medicine?
Sapropterin helps to lower the high levels of phenylalanine (an amino acid) in the blood caused by phenylketonuria (an inherited disorder where your body is not able to break down phenylalanine) or tetrahydrobiopterin deficiency (an inherited condition in which the body does not produce enough tetrahydrobiopterin, a substance needed to help break down phenylalanine).

This medicine is meant to be taken as part of a complete care programme that should include a phenylalanine-restricted diet and regular monitoring of blood phenylalanine levels.
How do I take this medicine?
Take Sapropterin exactly as directed by your doctor or according to the instructions on the label. Do not take more or less than instructed by your doctor.

Take it together with food or immediately after a meal. Try to take it at the same time each day.

Sapropterin is available as a tablet or as a powder for oral solution.

If you are taking the tablet, you may swallow it whole. You may also dissolve the tablet in water or apple juice, or crush and mix it in a small amount of soft food (e.g. apple sauce, pudding) before taking it. Administration of tablets may vary between formulations. Ask your doctor or pharmacist how you should properly take your medicine.

If you are taking the powder for oral solution, dissolve the powder in water or apple juice or in a small amount of soft food (e.g. apple sauce, pudding) and mix thoroughly.

The dose of this medicine will be decided by your doctor. Your doctor will advise you on the treatment timeframe depending on the type of your condition and your response to the medication.

Sapropterin must be taken regularly for it to be effective. Continue taking this medicine even when you feel better. Do not stop taking it unless instructed by the doctor.
What should I do if I have forgotten to take this medicine?
Take the missed dose as soon as you remember. If it is almost time for your next dose, skip the missed dose and return to your normal dosing schedule.

DO NOT double a dose under any circumstances.

If you often forget to take your medicine, let your doctor and pharmacist know.
What should I take note of while taking this medicine?
Inform your doctor if you have the following conditions:
  • history of fits or seizures
  • history of severe allergic reaction to Sapropterin
  • kidney disease
  • liver disease
Let your doctor know if you are pregnant or breastfeeding.

Be sure to check with your doctor or pharmacist before giving this medicine to a child. Children may be more sensitive to the side effects.

For as long as you are taking Sapropterin, you may need to have regular blood tests to check your body's response to the medicine. Your doctor will advise you about how often you need to have blood tests.
What side effects could I experience?
Sapropterin may cause any of the following side effects: headache, cough, runny nose, stuffy nose, and sore throat.

Some side effects may need immediate medical help. Alert your doctor quickly if you experience any of the following:
  • rashes, breathlessness, swelling of the face, eyes or mouth
  • severe stomach pain, throat pain, trouble swallowing, loss of appetite, pass out bloody or black as tar stools, blood in your vomit
  • fidgeting, moving around too much, excessive talking
Inform your doctor if any of these side effects do not go away or are severe, or if you experience other side effects.
Can I take this with other medicines?
Inform your doctor and pharmacist if you are taking any of these medicines:
  • levodopa (medicine for Parkinson's disease [disorder that affects the movement of a person, causing symptoms such as tremors, muscle stiffness, slowed movement and impaired balance or walking])
  • methotrexate (medicine for cancer)
  • trimethoprim (antibiotic)
  • medicines for high blood pressure e.g. sodium nitroprusside
  • medicines used to treat impotence e.g. sildenafil, tadalafil
  • medicines for chest pain e.g. glyceryl trinitrate, isosorbide dinitrate
This list does not include all medicines that may interact with Sapropterin.

Always notify your doctor and pharmacist if you are taking any other medicines, including herbal tonics such as traditional Chinese medicines, supplements, and medicines that you buy without a prescription.
What special dietary instructions should I follow?
Avoid alcohol.

It is important to adhere to the diet prescribed by your doctor. Do not make any changes to your dietary intake without first asking your doctor.
How should I store this medicine?
Store in a cool, dry place away from the reach of children.

Protect from moisture.

Medicines must not be used past the expiry date.
This information is independently developed by MIMS based on Sapropterin - Oral and is provided for your reference only. It is not a replacement for and should only be used in conjunction with full consultation with a licensed healthcare professional, the information provided by your pharmacist and/or the manufacturer of the medication. It may not contain all the available information you require and cannot substitute professional medical care, nor does it take into account all individual circumstances. Although great effort has been made to ensure content accuracy, we shall not be held responsible or liable for any claims or damages arising from the use or misuse of the information contained herein, its contents or omissions, or otherwise. Copyright © 2025 MIMS. All rights reserved. Powered by MIMS.com
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