KUVAN is the first and only prescription medication that may help lower Phe levels. Along with careful dietary management, KUVAN may help you lower your blood Phe levels more than the use of diet alone.

Even when you are following a Phe-restricted diet, constantly keeping Phe under control can be a real challenge. KUVAN tips the challenges of Phe control in your favor, helping you to try to stay at the target blood Phe level that your doctor or dietitian determines is important for you to maintain.

KUVAN works in a different way than diet to keep Phe low. The low-Phe diet reduces the amount of Phe entering the body. KUVAN helps the body break down Phe so that Phe stays low. KUVAN works with a Phe-restricted diet to help keep your blood Phe low.

Research and results

In clinical studies, KUVAN has been studied in 579 PKU patients for periods from 1 to 30 weeks.

KUVAN helps patients with high Phe

In one study, PKU patients who had a high Phe level (over 13.3 mg/dL, or
800 μmol/L) were enrolled and did not alter their usual diet during the study.
In the group of patients taking KUVAN, blood Phe levels decreased to
10.1 mg/dL, or 607 μmol/L, after 6 weeks of treatment. However, Phe levels in the group not taking KUVAN increased to 14.9 mg/dL, or 891 μmol/L.

KUVAN helps patients even when Phe is already low

In another study, PKU patients with fairly low Phe levels at the start of the study (4.6 mg/dL, or 276 μmol/L) who were taking KUVAN were able to decrease their Phe level even further to 2.1 mg/dL (127 μmol/L). Patients taking a placebo (a pill without any medicine in it) did not see much of a change in their Phe levels.

Next: Safety Information for 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.