Why is this medication prescribed?

Kuvan (generic name sapropterin dihydrochloride) is a medication prescribed for a specific genetic disorder called phenylketonuria (PKU). PKU is an inherited metabolic disorder in which the body is unable to properly process an amino acid called phenylalanine (Phe). This leads to a buildup of phenylalanine in the blood, which can be toxic to the brain and cause various cognitive and neurological problems if left untreated.

Kuvan is used in the treatment of PKU to help lower the levels of phenylalanine in the blood. It works by providing a synthetic form of the naturally occurring enzyme called tetrahydrobiopterin (BH4), which is essential for the breakdown of phenylalanine. Kuvan helps some individuals with PKU to produce more BH4, enabling them to better metabolize phenylalanine and maintain healthier blood phenylalanine levels.

How should this medicine be used?

Kuvan is available in the form of oral tablets and should be taken as directed by a healthcare professional. Here are some general guidelines for using Kuvan, but it’s important to follow the specific instructions provided by your doctor:

• Dosage: The dosage of Kuvan will be determined by your doctor based on your individual needs. It’s important to take the prescribed dose and not exceed or skip doses unless instructed by your doctor.
• Timing: Kuvan is usually taken once a day. It can be taken with or without food, but it’s important to be consistent in taking it at the same time every day to maintain stable blood phenylalanine levels.
• Compliance with diet: While taking Kuvan, it’s crucial to continue following a low-protein diet as prescribed by your doctor or dietitian. The combination of Kuvan and a restricted diet is usually necessary to effectively manage PKU.
• Monitoring: Regular monitoring of blood phenylalanine levels is necessary to assess the effectiveness of Kuvan therapy. Your doctor will advise you on the frequency of blood tests and the target range for phenylalanine levels.
• Adjustments: Based on your response to Kuvan and the results of blood tests, your doctor may need to adjust the dosage or make changes to your treatment plan. It’s important to communicate any concerns or changes in symptoms to your healthcare team.

Remember, Kuvan is a prescription medication, so it should only be used under the supervision and guidance of a healthcare professional experienced in the treatment of PKU. They will provide you with detailed instructions tailored to your specific condition and needs.

Other uses for this medicine

Kuvan (sapropterin dihydrochloride) is primarily prescribed for the treatment of phenylketonuria (PKU) and is not commonly used for other conditions. However, there may be instances where it is prescribed off-label for certain individuals with hyperphenylalaninemia (elevated phenylalanine levels) who do not have PKU.

What special precautions should I follow?

Regarding special precautions, here are some important considerations when using Kuvan:

• Allergies: Inform your doctor if you have any known allergies to sapropterin or any other ingredients present in Kuvan tablets. This medication should be used with caution or avoided altogether if you have a history of hypersensitivity reactions.
• Pregnancy and breastfeeding: The safety of Kuvan during pregnancy or while breastfeeding has not been established. If you are pregnant, planning to become pregnant, or breastfeeding, it’s crucial to discuss the potential risks and benefits with your doctor before using Kuvan.
• Medication interactions: Inform your doctor about all the medications, supplements, or herbal products you are taking, as they may interact with Kuvan. Certain medications, such as phenobarbital or phenytoin, may interfere with the effectiveness of Kuvan.
• Side effects: Like any medication, Kuvan may cause side effects. Common side effects include headache, diarrhea, vomiting, stomach pain, and runny or stuffy nose. If you experience any persistent or severe side effects, notify your doctor.
• Monitoring and adjustments: Regular monitoring of blood phenylalanine levels is necessary while taking Kuvan. Your doctor may need to adjust the dosage or treatment plan based on the results and your individual response to therapy.
• Compliance with diet: It’s important to understand that Kuvan is not a cure for PKU, and a strict low-protein diet is still essential. Adhering to the prescribed dietary restrictions is crucial for effective management of PKU, even while taking Kuvan.

Always consult with your healthcare professional for personalized advice and guidance regarding the use of Kuvan, as they will consider your specific medical history and needs.

What special dietary instructions should I follow?

• Kuvan is usually prescribed alongside a controlled diet that is low in phenylalanine (Phe), as PKU is characterized by the inability to metabolize this amino acid.
• Follow your healthcare provider’s instructions regarding your specific dietary restrictions and Phe intake.
• Work closely with a registered dietitian who specializes in PKU management to create a personalized meal plan.

What should I do if I forget a dose?

• If you forget to take a dose of Kuvan, take it as soon as you remember. However, if it is close to the time for your next scheduled dose, skip the missed dose and continue with your regular dosing schedule.
• Do not take a double dose to make up for a missed one.

What side effects can this medication cause?

Kuvan (sapropterin dihydrochloride) can cause side effects, although not everyone experiences them. Common side effects of Kuvan may include:

• Headache
• Diarrhea
• Vomiting
• Stomach pain or discomfort
• Runny or stuffy nose
• Dizziness
• Cough
• Fatigue
• Rhinitis (inflammation of the nasal passages)
• Upper respiratory tract infection

These side effects are generally mild and temporary. However, if any of these side effects persist or become severe, it’s important to consult your healthcare provider for further evaluation and guidance.

In rare cases, Kuvan can cause more serious side effects. If you experience any of the following symptoms, you should seek immediate medical attention:

• Allergic reactions: This may include rash, itching, swelling (especially of the face, tongue, or throat), severe dizziness, or difficulty breathing.
• Worsening neurological symptoms: Kuvan therapy aims to improve neurological symptoms associated with phenylketonuria (PKU). However, in some cases, there may be a paradoxical worsening of neurological symptoms. If you notice any unusual changes in behavior, mood, or cognitive function, inform your doctor.

It’s important to note that this is not an exhaustive list of side effects. Different individuals may react differently to medications, and some people may experience side effects not mentioned here. If you have any concerns about the side effects of Kuvan, it’s best to consult your healthcare provider. They can provide you with more detailed information and address any specific questions or concerns you may have.

What should I know about storage and disposal of this medication?

• Storage: Store Kuvan tablets at room temperature, away from excessive heat, moisture, and direct light. Follow the specific storage instructions provided by the manufacturer or on the medication packaging.
• Keep out of reach of children: Ensure that Kuvan is stored in a secure place out of the reach of children and pets.
• Do not use expired medication: Check the expiration date on the packaging and do not use Kuvan if it has expired. Dispose of expired or unused medication properly.
• Disposal: Dispose of unused or expired Kuvan tablets in accordance with local regulations. Do not flush them down the toilet or pour them into drains, as this can potentially contaminate the water supply. Instead, consult with your pharmacist or local waste management authorities for appropriate disposal methods, such as medication take-back programs.

In case of emergency/overdose

• Emergency assistance: If you or someone else may have taken an overdose of Kuvan or is experiencing severe symptoms, immediately contact emergency medical services or go to the nearest emergency room.
• Information for healthcare providers: Provide healthcare providers with details about the medication, dosage, and the amount taken (if known).
• National Poison Control Center: In the United States, you can contact the National Poison Control Center at 1-800-222-1222 for guidance on managing an overdose.

What other information should I know?

• Compliance with treatment: It is important to take Kuvan as prescribed by your healthcare professional and to follow their instructions regarding diet, blood tests, and other aspects of PKU management.
• Regular follow-up appointments: Attend scheduled follow-up appointments with your doctor to monitor your response to Kuvan therapy, adjust the treatment plan if needed, and discuss any concerns or questions you may have.
• Medication interactions: Inform your healthcare provider about all medications, supplements, or herbal products you are taking, as they may interact with Kuvan. Certain medications, such as phenobarbital or phenytoin, may interfere with the effectiveness of Kuvan.
• Genetics and family planning: If you are planning to have children, it’s important to discuss the genetic implications of PKU with a genetic counselor or healthcare professional, as PKU can be inherited. They can provide guidance on family planning options and prenatal testing.

Remember, this information serves as a general guide, and it’s crucial to consult with your healthcare provider or pharmacist for specific instructions, guidelines, and advice related to your individual circumstances and treatment plan.