Package leaflet: Information for the patient

Sapropterin dihydrochloride

Read all of this leaflet carefully before you start taking this medicine because it contains important information for you.

• – Keep this leaflet. You may need to read it again.

• – If you have any further questions, ask your doctor or pharmacist.

• – This medicine has been prescribed for you only. Do not pass it on to others. It may harm them, even if

their signs of illness are the same as yours.

- if you get any side effects, talk to your doctor or pharmacist. this includes any possible side effects not listed in this leaflet. see section 4.

What is in this leaflet

• 1. What Kuvan is and what it is used for

• 2. What you need to know before you take Kuvan

• 3. How to take Kuvan

• 4. Possible side effects

• 5. How to store Kuvan

• 6. Contents of the pack and other information

1. what kuvan is and what it is used for

Kuvan contains the active substance sapropterin which is a synthetic copy of a body’s own substance called tetrahydrobiopterin (BH4). BH4 is required by the body to use an amino acid called phenylalanine in order to build another amino acid called tyrosine.

Kuvan is used to treat hyperphenylala­ninaemia (HPA) or phenylketonuria (PKU) in patients of all ages. HPA and PKU are due to abnormally high levels of phenylalanine in the blood which can be harmful. Kuvan reduces these levels in some patients who respond to BH4 and can help increase the amount of phenylalanine that can be included in the diet.

This medicine is also used to treat an inherited disease called BH4 deficiency in patients of all ages, in which the body cannot produce enough BH4. Because of very low BH4 levels phenylalanine is not used properly and its levels rise, resulting in harmful effects. By replacing the BH4 that the body cannot produce, Kuvan reduces the harmful excess of phenylalanine in the blood and increases the dietary tolerance to phenylalanine.

2. what you need to know before you take kuvan- if you are allergic to sapropterin or any of the other ingredients of this medicine (listed in section 6).

Warnings and precautions

Package leaflet: Information for the patient

Sapropterin dihydrochloride

Read all of this leaflet carefully before you start taking this medicine because it contains important information for you.

• – Keep this leaflet. You may need to read it again.

• – If you have any further questions, ask your doctor or pharmacist.

• – This medicine has been prescribed for you only. Do not pass it on to others. It may harm them, even if

their signs of illness are the same as yours.

- if you get any side effects, talk to your doctor or pharmacist. this includes any possible side effects not listed in this leaflet. see section 4.

What is in this leaflet

• 1. What Kuvan is and what it is used for

• 2. What you need to know before you take Kuvan

• 3. How to take Kuvan

• 4. Possible side effects

• 5. How to store Kuvan

• 6. Contents of the pack and other information

1. what kuvan is and what it is used for

Kuvan contains the active substance sapropterin which is a synthetic copy of a body’s own substance called tetrahydrobiopterin (BH4). BH4 is required by the body to use an amino acid called phenylalanine in order to build another amino acid called tyrosine.

Kuvan is used to treat hyperphenylala­ninaemia (HPA) or phenylketonuria (PKU) in patients of all ages. HPA and PKU are due to abnormally high levels of phenylalanine in the blood which can be harmful. Kuvan reduces these levels in some patients who respond to BH4 and can help increase the amount of phenylalanine that can be included in the diet.

This medicine is also used to treat an inherited disease called BH4 deficiency in patients of all ages, in which the body cannot produce enough BH4. Because of very low BH4 levels phenylalanine is not used properly and its levels rise, resulting in harmful effects. By replacing the BH4 that the body cannot produce, Kuvan reduces the harmful excess of phenylalanine in the blood and increases the dietary tolerance to phenylalanine.

2. what you need to know before you take kuvan- if you are allergic to sapropterin or any of the other ingredients of this medicine (listed in section 6).

Warnings and precautions

Talk to your doctor or pharmacist before taking Kuvan, particularly:

• – if you are 65 years of age or older

• – if you have problems with your kidney or liver

• – if you are ill. Consultation with a physician is recommended during illness as blood phenylalanine

levels may increase

• – if you have predisposition to convulsions

When you are treated with Kuvan, your doctor will test your blood to verify how much phenylalanine and tyrosine it contains and may decide to adjust the dose of Kuvan or your diet if needed.

You must continue your diet treatment as recommended by your doctor. Do not change your diet without contacting your doctor. Even if you take Kuvan, if your phenylalanine blood levels are not well controlled, you can develop severe neurologic problems. Your doctor should continue to monitor your blood phenylalanine levels often during your treatment with Kuvan, to make sure that your blood phenylalanine levels are not too high or too low.

Other medicines and Kuvan

Tell your doctor or pharmacist if you are taking, have recently taken or might take any other medicines. In particular you should tell your doctor if you are using:

• – levodopa (used to treat Parkinson’s di­sease)

• – medicines for treatment of cancer (e.g. methotrexate)

• – medicines for treatment of bacterial infections (e.g. trimethoprim)

• – medicines that cause dilation of blood vessels, (such as glyceryl trinitrate (GTN), isosorbide dinitrate (ISDN), sodium nitroprusside (SNP), molsidomin, minoxidil).

Pregnancy and breast-feeding

If you are pregnant or breast-feeding, think you may be pregnant or are planning to have a baby, ask your doctor or pharmacist for advice before taking this medicine.

If you are pregnant your doctor will tell you how to control phenylalanine levels adequately. If these are not strictly controlled before or when you become pregnant, this could be harmful to you and to your baby. Your doctor will monitor the restriction of dietary phenylalanine intake prior and during pregnancy.

If the strict diet does not adequately reduce phenylalanine amount in your blood your doctor will consider whether you must take this medicine.

You should not take this medicine if you are breast-feeding.

Driving and using machines

Kuvan is not expected to affect the ability to drive and use machines.

Kuvan contains potassium citrate (E332)

This medicine contains 1.6 mmol (62.7 mg) potassium per sachet. This should be taken into consideration by patients with reduced kidney function or patients on a controlled potassium diet.

3. how to take kuvan

Kuvan 500 mg is for use in patients above 25 kg body weight only.

Always take this medicine exactly as your doctor has told you. Check with your doctor if you are not sure.

Dosing for PKU

The recommended starting dose of Kuvan in patients with PKU is 10 mg for each kg of body weight. Take Kuvan as a single daily dose with a meal to increase the absorption, and at the same time each day, preferably in the morning. Your doctor may adjust your dose, usually between 5 and 20 mg for each kg of body weight per day, depending on your condition.

Dosing for BH4 deficiency

The recommended starting dose of Kuvan in patients with BH4 deficiency is 2 to 5 mg for each kg of body weight. Take Kuvan with a meal to increase the absorption. Divide the total daily dose into 2 or 3 doses, taken over the day. Your doctor may adjust your dose up to 20 mg for each kg of body weight per day, depending on your condition.

When you are treated with Kuvan, your doctor will test your blood to verify how much phenylalanine and tyrosine it contains and may decide to adjust the dose of Kuvan or your diet if needed.

You must continue your diet treatment as recommended by your doctor. Do not change your diet without contacting your doctor. Even if you take Kuvan, if your phenylalanine blood levels are not well controlled, you can develop severe neurologic problems. Your doctor should continue to monitor your blood phenylalanine levels often during your treatment with Kuvan, to make sure that your blood phenylalanine levels are not too high or too low.

Other medicines and Kuvan

Tell your doctor or pharmacist if you are taking, have recently taken or might take any other medicines. In particular you should tell your doctor if you are using:

• – levodopa (used to treat Parkinson’s di­sease)

• – medicines for treatment of cancer (e.g. methotrexate)

• – medicines for treatment of bacterial infections (e.g. trimethoprim)

• – medicines that cause dilation of blood vessels, (such as glyceryl trinitrate (GTN), isosorbide dinitrate (ISDN), sodium nitroprusside (SNP), molsidomin, minoxidil).

Pregnancy and breast-feeding

If you are pregnant or breast-feeding, think you may be pregnant or are planning to have a baby, ask your doctor or pharmacist for advice before taking this medicine.

If you are pregnant your doctor will tell you how to control phenylalanine levels adequately. If these are not strictly controlled before or when you become pregnant, this could be harmful to you and to your baby. Your doctor will monitor the restriction of dietary phenylalanine intake prior and during pregnancy.

If the strict diet does not adequately reduce phenylalanine amount in your blood your doctor will consider whether you must take this medicine.

You should not take this medicine if you are breast-feeding.

Driving and using machines

Kuvan is not expected to affect the ability to drive and use machines.

Kuvan contains potassium citrate (E332)

This medicine contains 1.6 mmol (62.7 mg) potassium per sachet. This should be taken into consideration by patients with reduced kidney function or patients on a controlled potassium diet.

3. how to take kuvan

Kuvan 500 mg is for use in patients above 25 kg body weight only.

Always take this medicine exactly as your doctor has told you. Check with your doctor if you are not sure.

Dosing for PKU

The recommended starting dose of Kuvan in patients with PKU is 10 mg for each kg of body weight. Take Kuvan as a single daily dose with a meal to increase the absorption, and at the same time each day, preferably in the morning. Your doctor may adjust your dose, usually between 5 and 20 mg for each kg of body weight per day, depending on your condition.

Dosing for BH4 deficiency

The recommended starting dose of Kuvan in patients with BH4 deficiency is 2 to 5 mg for each kg of body weight. Take Kuvan with a meal to increase the absorption. Divide the total daily dose into 2 or 3 doses, taken over the day. Your doctor may adjust your dose up to 20 mg for each kg of body weight per day, depending on your condition.

When you are treated with Kuvan, your doctor will test your blood to verify how much phenylalanine and tyrosine it contains and may decide to adjust the dose of Kuvan or your diet if needed.

You must continue your diet treatment as recommended by your doctor. Do not change your diet without contacting your doctor. Even if you take Kuvan, if your phenylalanine blood levels are not well controlled, you can develop severe neurologic problems. Your doctor should continue to monitor your blood phenylalanine levels often during your treatment with Kuvan, to make sure that your blood phenylalanine levels are not too high or too low.

Other medicines and Kuvan

Tell your doctor or pharmacist if you are taking, have recently taken or might take any other medicines. In particular you should tell your doctor if you are using:

• – levodopa (used to treat Parkinson’s di­sease)

• – medicines for treatment of cancer (e.g. methotrexate)

• – medicines for treatment of bacterial infections (e.g. trimethoprim)

• – medicines that cause dilation of blood vessels, (such as glyceryl trinitrate (GTN), isosorbide dinitrate (ISDN), sodium nitroprusside (SNP), molsidomin, minoxidil).