What is Sapropterin?
Sapropterin is a medication used to reduce the levels of phenylalanine that are present in blood samples of adults and young children under the age of one month with the specific type of phenylketonuria (PKU).Sapropterin is also employed for other purposes that are not covered in this guideline.
Side effects of Sapropterin
Seek medical attention immediately. If you are experiencing symptoms that indicate an allergic reaction, such as hives, rash, wheezing or coughing, difficulty breathing, nausea, feeling lightheaded or flushing (warmth or redness), or the sensation of tingling, or swelling of your lips, face, and tongue,
Sapropterin may cause serious side effects. Contact your physician immediately. If you suffer from:
- Behavior change, seizures, or issues with speech
- Fever loss, appetite difficulties swallowing, throat pain, severe stomach discomfort, nausea, pain, vomiting
- Bloody or tarry stool and coughing up blood. Vomit that resembles coffee grounds,
- Hyperactive behavior, for example, fidgeting, moving around a lot, or talking too much.
Common negative side effects of sapropterin can include:
- Headache;
- Diarrhea;
- Vomiting;
- Runny or stuffy nose, sore throat, cough
This isn't an exhaustive list of all the side effects. Others could happen. Contact your doctor to seek medical advice on the effects. You can report any side effects to the FDA at 1-800-FDA-1088.
Warnings
Only use it according to the directions. Inform your doctor if you are taking other medicines or have any other medical conditions or allergies.
Prior to use this drug
Sapropterin is not a good choice if you have an allergy to it.
Consult your physician If you've ever been afflicted by:
- Stomach ulcer or bleeding;
- Lack of appetite, loss of appetite, or if you're undernourished.
It is unknown whether sapropterin can harm an unborn baby. Inform your doctor if you are expecting or planning to be pregnant.
If you're pregnant, then your name might be added to the registry for pregnancy to monitor how sapropterin affects the infant.Check with your doctor to get advice on whether it is safe to breastfeed while taking sapropterin.
How to take Sapropterin?
Follow the directions on your prescription label, and go through all medication guides or instructions sheets. Your doctor may alter the dosage. Follow the medication precisely as prescribed.
Sapropterin could cause levels of phenylalanine to drop too much for children less than 7 years old who are taking this medication.Eat the medication every day.It is possible to take the tablet in its entirety or dissolve it in 4–8 cups of apple juice or water.
- It can take up to just a few minutes for the tablet to disintegrate, and you might notice small pieces floating in the liquid. The pieces are safe to eat.
- Mix it up and take a drink within 15 minutes after the tablet disintegrates.
- For the maximum dosage, add a small amount of water to the glass, stir it gently, and then drink it right away.
- It is also possible to grind the tablet into a mixture with the addition of a tiny amount of soft food like applesauce or pudding.
The powder to be used as an oral solution should be dissolvable in between 4 and 8 ounces of apple juice or water.
- If you're giving this medication to a baby that weighs less than 22, it is possible to dissolve the powder in just 1 teaspoon of apple juice or water. Then, you can give the liquid to the baby via an oral dosing syringe. If you do not own a dosing syringe, you can consult your pharmacist about one.
- Mix it up and consume it all within 30 minutes after it dissolves.
- You can also blend the powder into a tiny amount of soft food, like applesauce or pudding.
- Be sure that the powder has completely dissolved prior to eating or drinking the mix.
Doses are determined by the weight of children and teens. The dose of your child could alter if your child increases or decreases weight.
You might need to adhere to an individualized diet. Follow all the instructions given by your physician or dietitian. Find out about the foods you should avoid eating.When you take sapropterin for treatment, you might need to have regular blood teststhe bottle should be kept at room temperature and free from moisture and heat. Close the bottle when not being used.
Details on dosage
Usual Adult Dose for Phenylketonuria:
Initial dosage: 10 to 20 mg/kg, orally, once a day.
Blood phenylalanine (Phe) levels must be assessed after a week and again after that, and doses should be raised to 20 mg/kg/day when the blood Phe levels are not reduced.
Dosage for maintenance: Once the responsiveness has been established, the dose is to be adjusted in the interval of 5–20 mg/kg/day based on biochemical responses.
Non-Responders:
Patients whose phenylalanine levels don't decrease when treated at 20 mg/kg/day over a month should be considered not to be responding and should stop treatment.
Comments:
This drug should be used in conjunction with a phenylalanine-restricted diet.
Protein and phosphorus intakes should not be altered during the period of evaluation.
Response to treatment is measured by changes in the blood levels of phenylalanine.
Use: To reduce blood phenylalanine levels in patients with hyperphenylalaninemia due to tetrahydrobiopterin- (BH4)-responsive phenylketonuria (PKU).
Usual Pediatric Dose for Phenylketonuria:
One month to six years old:
Initial dose: 10 mg/kg, orally once a day.
7 years or older
Initial dosage: 10 to 20 mg/kg once orally
Check blood phenylalanine levels following one week, and after that, regularly; the dose will be increased to 20 mg/kg/day in the event that blood phenylalanine levels are not diminished.
Dosage for maintenance: Once the responsiveness has been established, the dose can be adjusted to 5–20 mg/kg/day based on biochemical responses.
Non-Responders:
Patients whose phenylalanine levels don't decrease when treated at 20 mg/kg/day for a month are considered non-responders and should stop treatment.
Comments:
This drug should be used in conjunction with a phenylalanine-restricted diet.
Protein and protein intake shouldn't be changed during the period of evaluation.
The response to therapy is measured by changes in the blood levels of phenylalanine.
Use: To reduce blood phenylalanine levels in patients with hyperphenylalaninemia due to tetrahydrobiopterin- (BH4)-responsive phenylketonuria (PKU).
What happens if I miss the dose?
Do not take the medication as fast as you can. However, do not miss your missed dosage if you are nearing the time to take the next dose. Don't take more than two doses at once in the course of a day.
What happens if I overdose?
Get medical attention in an emergency or contact the poison help line at 1-800-222-1222.
What should be avoided?
Follow the instructions of your physician regarding any limitations on foods, drinks, or activities.
Interaction with other drugs
Inform your doctor about all other medicines you take, particularly:
- Levodopa;
- Methotrexate, valproic acid, phenobarbital, trimethoprim,
- Medicine to treat impotence or pulmonary arterial hypertension: sildenafil (Viagra, Revatio), avanafil (Stendra), tadalafil (Adcirca, Cialis), and vardenafil (Levitra, Staxyn).
This list isn't complete. Other medications may interact with the sapropterin chemical, such as prescription and over-the-counter medicines, vitamins, and herbal products. There are many possible interactions between drugs that are included here.
