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Kuvan® (sapropterin dihydrochloride) Tablets and Powder for Oral Solution for PKU

Kuvan 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

  • Kuvan Tablets approved in the United States, European Union, Canada, and Japan
  • Kuvan Powder approved in the United States
  • Designated as an orphan drug in the United States and European Union
  • Manufactured and commercialized worldwide with the exception of Kuvan in Japan
  • To learn more about Kuvan and our 15 year commitment to PKU, please check out “The Pursuit of PKU”.

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. Kuvan Powder was approved in the US in December 2013.

For additional information about Kuvan, please visit www.Kuvan.com.

Commercialization Plan

Kuvan tablets are commercialized by BioMarin. In 2015, BioMarin acquired the global rights to Kuvan and pegvaliase from Merck Serono (Merck). Under the terms, BioMarin has exclusive worldwide rights to Kuvan and pegvaliase with the exception of Kuvan in Japan. Biopten granules are commercialized by Daiichi-Sankyo Company. Limited in Japan.

Indication

Kuvan® (sapropterin dihydrochloride) Tablets and Powder for Oral Solution are approved to reduce blood Phe levels in people with a certain type of Phenylketonuria (PKU). Kuvan is to be used with a Phe-restricted diet.

Important Safety Information

What is KUVAN?

KUVAN® (sapropterin dihydrochloride) Tablets for Oral Use and Powder for Oral Solution are prescription medicines used to lower blood Phe levels in people with a certain type of Phenylketonuria (PKU). KUVAN is to be used along with a Phe-restricted diet.

What is the most important information I should know about KUVAN?
KUVAN can cause serious side effects, including:

  • Severe allergic reactions. Stop taking KUVAN and get medical help right away if you develop any of these signs or symptoms of a severe allergic reaction:
    • Wheezing or trouble breathing
    • Coughing
    • Feeling lightheaded or you faint
    • Feeling flushed
    • Nausea
    • Rash

  • Inflammation of the lining of the stomach (gastritis). Call your doctor right away if you have any of these signs or symptoms:
    • Severe upper stomach-area (abdominal) discomfort or pain, nausea, and vomiting
    • Blood in your vomit or stool
    • Black, tarry stools
  • Phe levels that are too low—some children under the age of 7 who take high doses of KUVAN each day may experience low Phe levels
  • Too much or constant activity (hyperactivity) can happen with KUVAN. Tell your doctor if you have any signs of hyperactivity, including:
    • Fidgeting or moving around too much
    • Talking too much

What should I tell my doctor before I take KUVAN?
Before you take KUVAN, tell your doctor about all your medical conditions, including if you:

  • Are allergic to sapropterin dihydrochloride or any of the ingredients in KUVAN
  • Have poor nutrition or have a loss of appetite
  • Are pregnant or plan to become pregnant
  • Are breastfeeding or plan to breastfeed. It is not known if KUVAN passes into your breast milk. Talk to your doctor about the best way to feed your baby if you take KUVAN

Tell your doctor about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal and dietary supplements. KUVAN and other medicines may interact with each other. Especially tell your doctor if you take:

  • A medicine that contains levodopa
  • An antifolate medicine
  • Sildenafil (Revatio, Viagra), tadalafil (Adcirca, Cialis), vardenafil
    (Staxyn, Levitra

Tell your doctor if you are not sure if your medicine is one that is listed above. Know the medicines you take. Keep a list of them to show your doctor and pharmacist when you get a new medicine.

How should I take KUVAN?
See the detailed “Instructions for Use” that comes with KUVAN for information about the correct way to dissolve and take a dose of KUVAN tablets or KUVAN powder for oral solution.

  • It is not possible to know if KUVAN will work for you until you start taking KUVAN. Your doctor will check your blood Phe levels when you start taking KUVAN to see if the medicine is working
  • Take KUVAN exactly as your doctor tells you
  • You can swallow KUVAN tablets whole or dissolve the tablets in water or apple juice
  • KUVAN powder for oral solution should be dissolved in water or apple juice
  • Any change you make to your diet may affect your blood Phe level. Follow your doctor’s instructions carefully and do not make any changes to your dietary Phe intake without first talking with your doctor. Even if you take KUVAN, if your blood Phe levels are not well controlled, you can develop severe neurologic problems
  • Your doctor should continue to monitor your blood Phe levels often during your treatment with KUVAN to make sure that your blood Phe levels are not too high or too low
  • If you have a fever, or if you are sick, your blood Phe level may go up. Tell your doctor as soon as possible so he or she can change your dose of KUVAN to help keep your blood Phe levels in the desired range
  • If you forget to take your dose of KUVAN, take it as soon as you remember that day. Do not take 2 doses in a day
  • If you take too much KUVAN, call your doctor for advice

The most common side effects of KUVAN are: headache, runny nose and nasal congestion, sore throat, diarrhea, vomiting, and cough.

These are not all the possible side effects of KUVAN. For more information, ask your doctor or pharmacist. Call your doctor for medical advice about side effects.

You may report side effects to the FDA at 1-800-FDA-1088 or www.fda.gov/medwatch. You may also report side effects to BioMarin Pharmaceutical Inc. at 1-866-906-6100.

Please read the full Prescribing Information by clicking here.

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BioMarin

Diseases

Lysosomal Storage Disorders (MPS I, MPS IVA, MPS VI, CLN2 disease) PKU, LEMS

Products

PALYNZIQ®
Brineura®
Vimizim®
Kuvan®
Naglazyme®
Aldurazyme®
Firdapse®

Patient/Physician Support Contact Information

BioMarin RareConnections
Tel: 866.906.6100
Fax: 888.863.3361
E-mail: support@biomarin-rareconnections.com

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