PKU in the News

Stay informed

There’s a lot to know about PKU, especially as we gain a better understanding of this condition. Check here often for information on recent breakthroughs and other news as it becomes available.

2007—KUVAN™ (sapropterin dihydrochloride) Tablets approved for the treatment of PKU

The US Food and Drug Administration (FDA) approved KUVAN, the first prescription drug therapy for the treatment of PKU. Produced by BioMarin Pharmaceutical Inc, KUVAN is proven to reduce blood Phe levels in patients with hyperphenylalaninemia (HPA) due to BH4-responsive PKU and is to be used in conjunction with a Phe-restricted diet.

“The approval of KUVAN represents an important milestone for PKU patients and their families,” said Jean-Jacques Bienaime, Chief Executive Officer of BioMarin.

“In clinical trials, KUVAN has been shown to help control blood Phe levels in PKU patients, and I am thrilled that this new therapy is now available to the PKU community,” stated Dr Barbara Burton, Professor of Pediatrics, Northwestern University Feinberg School of Medicine; Director, PKU Clinic at Children’s Memorial Hospital; and Clinical Investigator in the KUVAN Phase 2 and Phase 3 trials. “With KUVAN now approved, physicians and patients have, for the first time, a drug therapy option to manage the disease.”

Learn more about KUVAN.

2005—BioMarin begins clinical trials for testing BH4 in the treatment of PKU

BioMarin Pharmaceutical Inc began testing the effect of a pharmaceutical formulation of BH4 on lowering Phe levels in people with PKU. BH4 is the enzyme cofactor that works together with phenylalanine hydroxylase (PAH) to break down Phe. Some researchers think that the BH4, taken orally, works with the residual amounts of active PAH to speed up the Phe breakdown, actively lowering Phe levels in patients.

2000—NIH recommends "diet for life"¹²

As recently as 2000, there was no national agreement in the United States on how long someone with PKU should be on the diet. In the past, many people had believed that it was only important to be on the diet during certain years when the brain was growing the most.

In light of a 2000 National Institutes of Health statement, it is now agreed upon that a person with PKU should never go off the diet.

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 for KUVAN

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 change 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 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 the FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.