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Information for Patients & Caregivers
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What is Phenylketonuria?

Importance of Phe

Managing PKU


KUVAN

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Importance of Low & Consistent Phe

The importance of keeping Phe levels low

To protect your brain and mood, it's important to keep Phe low for life.2 Even a small increase in blood Phe levels can cause problems. IQ levels may drop as much as 4 points for every 100 μmol/L in lifetime Phe. For some people with PKU, that can be a big loss of IQ.3

The importance of keeping Phe levels consistent

New research has shown that children with large fluctuations in blood Phe levels tend to have lower intelligence (IQ) scores than children whose Phe levels are more or less the same all the time. Big changes in Phe seem to be hard on the brain.8

It can be hard to keep Phe low all the time, especially for people with classic PKU. A spike in Phe once in a while is probably not a problem. But people with PKU should try hard to keep Phe levels both low and constant.8

  • Big changes in blood Phe levels are associated with lower IQ*
  • Existing guidelines recommend maintaining blood Phe levels between 120 and 360 μmol/L†
* Anastasoaie V, et al. Stability of blood phenylalanine levels and IQ in children with phenylketonuria. Mol Genet Metab. 2008;95:17-20.

† NIH Consensus Conference, 2000.

Work with your doctor to set your own target level

In general, lower is better* to protect your brain and help you be your best.1,2

People who don't have PKU have Phe in the 55-60 μmol/L (1 mg/dL) range.7 So, even the lowest recommended levels for people with PKU are much higher than normal. Lower is better for your brain.*1,2

* Lower is better until you get Phe into the normal range of 1-2 mg/dL. Going lower than 1-2 mg/dL can be unhealthy because your body needs a minimum amount of Phe. Talk with your doctor about the right target range for you.1,2

Long-term studies of neurocognitive outcomes with KUVAN treatment have not been conducted. BioMarin is conducting a trial to evaluate long-term neurocognitive outcomes in PKU patients treated with KUVAN.

Managing PKU

Indication

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


This site is intended for use by U.S. residents only.

KUVAN® is a registered trademark of BioMarin Pharmaceutical Inc. Developed in collaboration with Asubio Pharma Co., Ltd.
©2010 BioMarin Pharmaceutical Inc. All rights reserved.