Innovative therapeutics



Kuvan® (sapropterin dihydrochloride) Patient
Nanuet, New York

BioMarin Patient Stories - GinaGina’s all-time favorite violin concertos are Antonio Vivaldi’s Four Seasons. Despite her musical preferences, she’s rather pleased that her Phe levels are no longer as changeable as the seasons.

“Now that I have my Phe levels under control,” she says, “I have more energy to do what I love most: reading, playing the violin, getting outside and walking my dog, and participating in other activities that were once difficult for me.”

Gina’s physician prescribes Kuvan, along with a Phe-restricted diet to bring her blood Phe levels down. While on Kuvan, she achieved her best day ever. Her Phe levels were as low as 3 mg/dL; normally they range somewhere between 5–8 mg/dL—a moderate but welcomed decrease from what she was once able to maintain with diet alone.*

Following a strict diet all of her life has also proved beneficial to Gina. She credits her accomplishments, including two graduate degrees—one Masters in childhood education and an MBA in finance—to the discipline she developed in treating her disease.

“I think because I stayed on a strict diet all my life I was able to succeed. This takes a lot of willpower,” she says. “Learning these skills early in life allowed me to follow my dream of becoming a school teacher.”

Since initiating therapy in 2007, Gina takes 15 Kuvan Tablets with water every morning after breakfast. Working with her dietitian, Gina maintains a Phe-restricted diet, but is now able to make adjustments to ensure adequate Phe control and nutritional balance.

“My diet has changed moderately since starting Kuvan. I still drink formula, but I am now able to eat two low-protein meals a day and one meal higher in natural protein. I keep a diet diary and I exercise. It’s pretty easy—all I need is a water bottle and my Kuvan tablets.”

Once uncomfortable eating in public, Gina now enjoys luncheons in the staff room with her colleagues.

“It was difficult dealing with PKU growing up, eating different foods, and not wanting to participate in normal childhood activities like Girl Scouts,” she recalls. “Staying on diet is the most challenging part of my life.”

“I know that Kuvan is working for me because my Phe levels have come down; I also feel better about myself. Despite what some people think that PKU patients cannot achieve success, I have been able to achieve my goals and succeed,” she maintains.

* 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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