What is KUVAN?
What is the function of BH4 in the body?
How does KUVAN work?
Why should I consider KUVAN?
Does KUVAN work for everyone?
Will KUVAN continue to work over time?
What if I don’t initially respond to KUVAN?
What are the possible side effects of KUVAN?
What should I tell my doctor before taking KUVAN?
How should I store KUVAN?
What is KUVAN?
KUVAN is a medication for PKU. KUVAN functions like BH4, a substance that naturally occurs in the body.
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What is the function of BH4 in the body?
Normally, BH4 helps the enzyme known as PAH (short for phenylalanine hydroxylase) work. The PAH enzyme breaks down the amino acid Phe, found in many foods, into other needed chemicals in the body. In
PKU, the PAH enzyme isn’t working, which allows too much Phe to build up in the blood. This can eventually affect the brain if left untreated.
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How does KUVAN work?
In many patients, KUVAN increases the activity of the PAH enzyme that isn’t working properly. In other words, the PAH enzyme that isn’t working “wakes up” and starts to process the Phe in some patients
with PKU. This helps to lower the amount of Phe in the blood in these patients.
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Why should I consider KUVAN?
The Phe-restricted diet is challenging, and so is consistently keeping your Phe level low. A low-Phe diet still allows a certain amount of Phe to pass from the food you eat into your bloodstream. KUVAN,
together with a Phe-restricted diet, helps you lower your blood Phe level and keep it low day after day. KUVAN addresses the problem from a different angle than diet does. KUVAN works by stimulating the
PAH enzyme to break down the Phe in your body, thereby lowering your blood Phe levels.
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Does KUVAN work for everyone?
KUVAN does not work for everyone. However, studies have found that some people with mild, moderate, and severe PKU have responded to treatment with KUVAN. It is not possible to know whether KUVAN will
work until you start taking it. Your doctor will determine whether or not KUVAN is working by checking to see if your blood Phe drops while the Phe-restricted diet is held constant.
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Will KUVAN continue to work over time?
Yes. In clinical trials, the benefit of KUVAN continued throughout the length of the study. KUVAN has been studied for periods from 1 to 30 weeks.
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What if I don’t initially respond to KUVAN?
If you do not initially respond to KUVAN, your doctor may consider another trial of KUVAN if there is reason to believe that diet, change in use of medical food, or other factors affecting blood Phe level
(such as fever or illness) may have affected your results. Unfortunately, KUVAN may not work for everyone.
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What are the possible side effects of KUVAN?
In studies, side effects in patients taking KUVAN generally occurred at a similar rate as they
did in patients who received placebo (a pill without any medicine in it).
The most common side effects reported when taking KUVAN included:
- Headache
- Diarrhea
- Abdominal pain
- Upper respiratory tract infection (like a cold)
- Throat pain
- Vomiting
- Nausea
The side effects listed are not all the side effects seen with KUVAN. You should talk to your doctor or pharmacist if you have concerns about these or other side effects.
You should also inform your doctor if you have any side effects when taking KUVAN.
Please see the KUVAN Patient Package Insert for more information.
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What should I tell my doctor before taking KUVAN?
Before taking KUVAN, tell your doctor if you:
- Have a fever
- Are pregnant or planning to become pregnant
- Are breast feeding
- Have or had liver or kidney problems
- Are allergic to KUVAN or any other medications
- Have poor nutrition or are anorexic
- Are taking levodopa
- Are taking medications that inhibit folate metabolism like methotrexate, because
these medications could affect how KUVAN works in your body
- Are taking medicines for erectile dysfunction like Viagra (sildenafil), Levitra (vardenafil), or Cialis (tadalafil)
You should also tell your doctor about any other medicines you are taking, including prescription and nonprescription medicines, vitamins, and herbal supplements. KUVAN and many other medicines may interact
with each other. Your doctor needs to know what medicines you take so he or she can decide if KUVAN is right for you.
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How should I store KUVAN?
- Store in a cool, dry place between 68°F–77°F (20°–25°C), with the cap closed tightly.
- Do not leave KUVAN in hot or humid places (such as your car or bathroom cabinet).
- Protect from moisture. Do not remove the desiccant (the small packet included with your tablets). The desiccant absorbs moisture.
- The color of the tablets may turn light yellow over time. This is normal, and you should take these tablets.
- Do not keep KUVAN that is out of date or that you no longer need. Be sure that if you throw any medicine away, it is out of the reach of children.
- Remember to keep KUVAN and all other medications out of the reach of children.
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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.