This is a true story about a real patient with PKU and her experience with KUVAN. Read on; you just might find that it sounds pretty familiar! Please note that KUVAN may not work for everyone. Because medicines work differently for different people, individual response to KUVAN may vary.

Lauren’s story

Lauren is 13 and lives in Shreveport, LA. Her parents did not know she had PKU until 19 days after birth. When she was diagnosed, her family was “freaked out” and “scared”; her pediatrician had no other PKU babies under his care and immediately referred the family to a PKU clinic at Tulane. There, the clinic staff had a PKU workshop, explained the disease, and provided formula and guidelines for diet. According to Lauren’s family, the dietitian at the clinic “put the fear of God” into the family about the importance of sticking to diet. As a small child, Lauren had blood tests every week to ensure blood Phe levels were within normal/healthy range.

Lauren’s mother subscribes to PKU News for low-Phe recipes and information on how to manage the Phe-restricted diet. She also purchases several products from Cambrooke. Lauren has a non-PKU sibling, so the family is used to making two separate meals for Lauren and the rest of the family. Lauren knows the importance of managing her diet and today has no desire to eat foods that are “off-limits” to her.

Last fall, her doctor informed her family about KUVAN, and indicated that Lauren would be a good candidate for the drug. During the first week of treatment with KUVAN, Lauren’s blood Phe levels dropped substantially. Now Lauren has something in addition to her diet to help keep her Phe levels low.

Her family is excited that KUVAN is now on the market, and that it will help her and many others keep blood Phe levels under control as they grow older. And they were able to find insurance support with the help of BioMarin, the company that makes KUVAN.

Lauren is a very active teenager: she sings in the church choir, she’s on the basketball and tennis teams at school, is secretary of her student council, and is in the National Junior Honor Society. She also loves to swim and spend time with her friends.

Lauren recently did her science project on her PKU and how KUVAN helps her keep her Phe levels low. Lauren’s project displayed a chart of her blood Phe levels while following her Phe-restricted diet, and depicted how KUVAN lowered her blood Phe levels even more than before. Best of all, she won first place at her school’s science fair and came in third in regionals!

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Indication

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 and may adjust 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 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.