Sapropterin is used along with a restricted diet to control blood phenylalanine levels in adults and children 1 month of age and older who have phenylketonuria (PKU; an inborn condition in which phenylalanine may build up in the blood and causes decreased intelligence and a decreased ability to focus, remember, and organize information). Sapropterin will only work for some people who have PKU, and the only way to tell if sapropterin will help a particular patient is to give the medication for a period of time and see whether his or her phenylalanine level decreases. Sapropterin is in a class of medications called cofactors. It works by helping the body to break down phenylalanine so it will not build up in the blood.
Sapropterin comes as a tablet and as a powder to be mixed with liquid or soft foods and take by mouth. It is usually taken once a day with food. Take sapropterin at around the same time every day. Follow the directions on your prescription label carefully, and ask your doctor or pharmacist to explain any part you do not understand. Take sapropterin exactly as directed. Do not take more or less of it or take it more often than prescribed by your doctor.
If you cannot swallow the tablets, place the number of sapropterin tablets you were told to take in a cup that contains 4 to 8 ounces (1/2 to 1 cup or 120 to 240 milliliters) of water or apple juice. Stir the mixture or crush the tablets with a spoon to dissolve the tablets. The tablets may not dissolve completely; there may still be small pieces of tablet floating at the top of the liquid. When the tablets are mostly dissolved, drink the entire mixture. If pieces of tablets remain in the cup after you drink the mixture, pour more water or apple juice into the cup and drink it to be sure you swallow all the medication. Be sure to drink the entire mixture within 15 minutes after you prepare it. Sapropterin tablets can also be crushed and mixed with soft foods such as applesauce and pudding.
To prepare sapropterin powder, add the contents of the powder packet(s) with 4 to 8 ounces (1/2 to 1 cup or 120 to 240 milliliters) of water or apple juice, or a small amount of soft food such as applesauce or pudding. Mix the powder in the liquid or soft food very well until the powder is completely dissolved. Be sure to drink or eat the entire mixture so you will get the complete dose. Eat or drink the mixture within 30 minutes of preparing.
If you are a parent or caregiver giving the powder to a child who weighs 22 pounds (10 kg) or less, you will need to get specific instructions from the doctor about how much water or apple juice to use, and how much of the prepared mixture to give to your child. Measure the amount of water or apple juice you are using with a medicine cup and use an oral dosing syringe to measure and give the dose to the child. Throw away any mixture remaining after the dose is given.
Your doctor will start you on adose of sapropterin and will check your blood phenylalanine level regularly. If your phenylalanine level does not decrease, your doctor may increase your dose of sapropterin. If your phenylalanine level does not decrease after 1 month of treatment with a high dose of sapropterin, you and your doctor will know that your condition does not respond to sapropterin. Your doctor will tell you to stop taking the medication.
Sapropterin may help to control blood phenylalanine levels, but it will not cure PKU. Continue to take sapropterin even if you feel well. Do not stop taking sapropterin without talking to your doctor.
Ask your pharmacist or doctor for a copy of the manufacturer's information for the patient.
This medication may be prescribed for other uses; ask your doctor or pharmacist for more information.
Before taking sapropterin,
You must continue to follow a low phenylalanine diet while you are taking sapropterin. Follow your doctor and nutritionist's directions carefully. Do not change your diet in any way without talking to your doctor or nutritionist.
If you remember the missed dose later the same day, take the missed dose as soon as you remember it. However, if you do not remember until the next day, skip the missed dose and continue your regular dosing schedule. Do not take more than one dose in one day or take a double dose to make up for a missed one.
Sapropterin may cause side effects. Tell your doctor if any of these symptoms are severe or do not go away:
Some side effects can be serious. If you experience any of these symptoms call your doctor immediately:
Sapropterin may cause other side effects. Call your doctor if you have any unusual problems while taking this medication.
If you experience a serious side effect, you or your doctor may send a report to the Food and Drug Administration's (FDA) MedWatch Adverse Event Reporting program online ( Web Site ) or by phone (1-800-332-1088).
Keep this medication in the container it came in, tightly closed, and out of reach of children. Store it in a cool, dry place, away from excess heat and moisture (not in the bathroom or car). Do not remove the desiccant (small packet included with medication to absorb moisture).
Unneeded medications should be disposed of in special ways to ensure that pets, children, and other people cannot consume them. However, you should not flush this medication down the toilet. Instead, the best way to dispose of your medication is through a medicine take-back program. Talk to your pharmacist or contact your local garbage/recycling department to learn about take-back programs in your community. See the FDA's Safe Disposal of Medicines website ( Web Site) for more information if you do not have access to a take-back program.
It is important to keep all medication out of sight and reach of children as many containers (such as weekly pill minders and those for eye drops, creams, patches, and inhalers) are not child-resistant and young children can open them easily. To protect young children from poisoning, always lock safety caps and immediately place the medication in a safe location – one that is up and away and out of their sight and reach. Web Site
In case of overdose, call the poison control helpline at 1-800-222-1222. Information is also available online at Web Site. If the victim has collapsed, had a seizure, has trouble breathing, or can't be awakened, immediately call emergency services at 911.
Symptoms of overdose may include the following:
Keep all appointments with your doctor and the laboratory. Your doctor will order certain lab tests to check your body's response to sapropterin.
Do not let anyone else take your medication. Ask your pharmacist any questions you have about refilling your prescription.
It is important for you to keep a written list of all of the prescription and nonprescription (over-the-counter) medicines you are taking, as well as any products such as vitamins, minerals, or other dietary supplements. You should bring this list with you each time you visit a doctor or if you are admitted to a hospital. It is also important information to carry with you in case of emergencies.
AHFS® Consumer Medication Information. © Copyright, The American Society of Health-System Pharmacists, Inc., 7272 Wisconsin Avenue, Bethesda, Maryland. All Rights Reserved. Duplication for commercial use must be authorized by ASHP.
Selected Revisions: May 15, 2019.