Innovative therapeutics



Kuvan® (sapropterin dihydrochloride) Patient
Chicago, Illinois

Julie - BioMarin Patient StoriesJulie, an industrial interior designer, has a rare genetic disorder known as PKU (phenylketonuria). She has been on treatment with Kuvan since 2005 when she enrolled in BioMarin’s Phase I clinical trials. “Initially, I enrolled in the Kuvan clinical study to help younger people with PKU have more options,” she says. “I sort of felt it was too late for me. I was 23 then and believed I had pretty good control of my diet and couldn’t do much more than that. But almost immediately I became one of the highest responders in my clinic! Now, a decade later, I feel a major improvement, especially when I train for runs and triathlons, and my Phe levels remain well within the normal range.”

Currently, BioMarin’s second product candidate for PKU, Pegvaliase (also known as PEG PAL) is in human clinical trials. Julie is also looking forward to future advancements in PKU treatment.

“Advancements in the treatment of PKU have made a huge impact in my life in an incredibly short period of time. I’ve had PKU all my life—and the advances made just within the last five years blow the advancements from all the previous years out of the water!”

* Individual patient response to Kuvan® therapy will vary.

Full Indication and Important Safety Information
Kuvan® (sapropterin dihydrochloride) Tablets for Oral Use and Powder for Oral Solution 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.

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Patient/Physician Support Contact Information

BioMarin RareConnections
Tel: 866.906.6100
Fax: 888.863.3361

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