Normally, BH4 in your body helps the enzyme PAH (short for phenylalanine hydroxylase) to do its job. The PAH enzyme breaks down the amino acid Phe, found in many foods, into other needed chemicals in the body.

In PKU, the PAH enzyme isn’t working, which allows too much Phe to build up in the blood. This can eventually affect the brain if left untreated.

KUVAN, which functions just like the BH4 that is naturally produced by your body, increases the activity of the PAH enzyme that isn’t working. In other words, the PAH enzyme that isn’t working “wakes up” and starts to process the Phe. This helps keep overall Phe levels in the blood low.

 

Next: The Benefits of KUVAN

 

Indication

KUVAN^® (sapropterin dihydrochloride) Tablets is approved to reduce blood Phe levels in patients with hyperphenylalaninemia (HPA) due to tetrahydrobiopterin- (BH4-) responsive Phenylketonuria (PKU). KUVAN is to be used with a Phe-restricted diet.

Important Safety Information

High blood Phe levels are toxic to the brain and can lead to lower intelligence and decrease in the ability to focus, remember and organize information. Any change you make to your diet may impact your blood Phe level. Follow your doctor’s instructions carefully. Your doctor and dietitian will continue to monitor and may adjust your diet throughout your treatment with KUVAN.

If you have a fever, or if you are sick, your Phe level may go up. Tell your doctor and dietitian as soon as possible so they can see if they have to adjust your treatment to help keep your blood Phe levels in the desired range.

KUVAN is a prescription medicine and should not be taken by people who are allergic to any of its ingredients. Tell your doctor if you have ever had liver or kidney problems, are nursing or pregnant or may become pregnant, have poor nutrition or are anorexic. Your doctor will decide if KUVAN is right for you. Tell your doctor about all the medicines you take.

The most common side effects reported when using KUVAN are headache, diarrhea, abdominal pain, upper respiratory tract infection (like a cold), throat pain, vomiting, and nausea.

To report SUSPECTED ADVERSE REACTIONS, contact BioMarin Pharmaceutical Inc. at 1-866-906-6100, or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

Please read the full Patient Information by clicking here.