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).
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:
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.