What Is Sapropterin?

Sapropterin is a prescription medication that is used to treat a medical condition known as phenylketonuria, or PKU. This drug is a synthetic form of a type of salt that is needed by the body for normal enzyme functioning. Some of the most common side effects associated with the use of sapropterin include nausea, headaches, and dizziness. More serious side effects may include swelling of the feet or hands, chest pain, or seizures. Any specific questions or concerns about sapropterin usage should be discussed with a doctor or other medical professional.

Phenylketonuria, more commonly known as PKU, is an inherited disorder that can lead to mental retardation if left untreated. Those born with this condition are unable to properly process a protein found in most foods known as phenylalanine. This disorder causes the body to have low levels of an enzyme that converts phenylalanine into substances that can be used by the body. Sapropterin is used to treat this condition and prevent mental retardation from occurring.

The primary use of sapropterin is to assist the body in properly breaking down phenylalanine so that it doesn't build up in the blood. This medication is typically made in the form of a tablet that is meant to be dissolved in water or apple juice and taken once per day with food. The entire mixture should be consumed within 15 minutes of being combined. Periodic blood testing may be required so the doctor can adjust the dosage of sapropterin as necessary.

A low phenylalanine diet must be followed while using sapropterin. Foods such as dairy products, meat, and nuts may have to be eliminated from the diet. The supervising physician will help to coordinate a healthy eating plan for patients who are taking this medication.

Most patients are able to tolerate this medication without experiencing any negative side effects, although bothersome symptoms may develop in some patients. Side effects of sapropterin are usually mild, although any persistent or bothersome symptoms that develop after beginning treatment with this medication should be reported to a doctor for further evaluation. Some of the most common side effects include gastrointestinal disturbances such as nausea, vomiting, or diarrhea. Headaches, dizziness, or flu-like symptoms may be experienced by some patients. Severe side effects such as bloody stool, seizures, or chest pain should be reported to a doctor immediately, as these symptoms may indicate the presence of severe complications.