Kuvan® (sapropterin dihydrochloride) Tablets for PKU

KUVAN® (sapropterin dihydrochloride) Tablets is the first and only FDA-approved medication for PKU to reduce blood Phe levels in patients with hyperphenylalaninemia (HPA) due to tetrahydrobiopterin (BH4-) responsive PKU. Kuvan is a pharmaceutical formulation of BH4, the natural cofactor for the PAH enzyme, which stimulates activity of the residual PAH enzyme to metabolize Phe into tyrosine. Kuvan is to be used in conjunction with a Phe-restricted diet.

Kuvan at a Glance

  • Approved in the United States, European Union, Canada, and Japan
  • Designated an orphan drug in the United States and European Union
  • Manufactured and commercialized by BioMarin in the United States

Regulatory Status

In December 2007, Kuvan received marketing approval from the US Food and Drug Administration making it the first specific therapy for the treatment of PKU patients. A year later, Kuvan received approval in the European Union in December 2008. Kuvan has been designated an orphan drug in the US and EU, which grants market exclusivity for 7 and 10 years, respectively. Kuvan was approved in Canada in April 2010. (Biopten) was approved for PKU in Japan in July 2008.

For additional information about Kuvan, please visit

Commercialization Plan

Kuvan is commercialized by BioMarin in the United States and Canada, Merck Serono in the European Union, and Asubio Pharma Co., Ltd. in Japan.

Full Indication and Important Safety Information (United States)

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 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

Kuvan is available by prescription only. To learn more, please visit for full prescribing information. If you have any questions about this information, please talk with your doctor.