Why is this medication prescribed?

Fabrazyme (generic name: agalsidase beta) is a medication used to treat Fabry disease, a rare genetic disorder characterized by the buildup of a fatty substance called globotriaosylceramide (Gb3) in various tissues and organs of the body. This buildup can lead to serious complications affecting the kidneys, heart, nervous system, and other organs.

Fabrazyme works by replacing the deficient enzyme alpha-galactosidase A in patients with Fabry disease. This enzyme is responsible for breaking down Gb3, and its deficiency leads to the accumulation of Gb3 in the body.

How should this medicine be used?

Fabrazyme (agalsidase beta) is typically administered intravenously by a healthcare professional in a clinical setting. Here’s a general outline of how Fabrazyme is used:

• Administration: Fabrazyme is administered intravenously (IV), meaning it is delivered directly into the bloodstream through a vein. The healthcare provider will prepare the medication and administer it using appropriate medical equipment.
• Dosage: The dosage of Fabrazyme varies depending on the individual patient’s condition, weight, and response to treatment. The healthcare provider will determine the appropriate dosage for each patient.
• Frequency: Fabrazyme is usually administered every two weeks. Again, the frequency of administration may vary based on the patient’s specific needs and response to treatment, as determined by the healthcare provider.
• Monitoring: During treatment with Fabrazyme, patients will be closely monitored by healthcare professionals to assess the effectiveness of the medication and watch for any potential side effects or adverse reactions.
• Follow-Up: Patients receiving Fabrazyme will typically have regular follow-up appointments with their healthcare provider to evaluate their response to treatment, adjust the dosage if necessary, and address any concerns or questions.

It’s crucial for patients to adhere to the treatment schedule and follow the instructions provided by their healthcare provider regarding the use of Fabrazyme. Additionally, patients should report any unusual symptoms or side effects to their healthcare provider promptly.

Other uses for this medicine

Fabrazyme (agalsidase beta) is primarily used for the treatment of Fabry disease, a rare genetic disorder caused by the deficiency of the enzyme alpha-galactosidase A (AGAL). However, there are no other approved uses for Fabrazyme beyond treating Fabry disease.

What special precautions should I follow?

As for special precautions to follow when using Fabrazyme:

• Medical Supervision: Fabrazyme should only be administered under the supervision of a healthcare professional experienced in the treatment of Fabry disease.
• Allergic Reactions: Some patients may experience allergic reactions to Fabrazyme. It’s important to inform your healthcare provider of any known allergies before starting treatment. Signs of an allergic reaction may include rash, itching, difficulty breathing, or swelling of the face, lips, tongue, or throat. Seek immediate medical attention if you experience any of these symptoms.
• Infusion Reactions: Infusion-related reactions, such as fever, chills, headache, flushing, or nausea, may occur during or after Fabrazyme administration. Healthcare providers typically monitor patients closely during infusion to manage any potential reactions.
• Risk of Hypersensitivity: Individuals with known hypersensitivity to agalsidase beta or any of its components should not receive Fabrazyme.
• Pregnancy and Breastfeeding: The safety of Fabrazyme in pregnant or breastfeeding women has not been established. It’s important to discuss the risks and benefits of using Fabrazyme during pregnancy or while breastfeeding with your healthcare provider.
• Drug Interactions: Inform your healthcare provider about all medications, vitamins, and supplements you are currently taking, as they may interact with Fabrazyme.
• Liver or Kidney Problems: Patients with pre-existing liver or kidney problems may require special monitoring during Fabrazyme treatment.
• Pediatric Use: The safety and effectiveness of Fabrazyme in pediatric patients have not been established.

Always follow your healthcare provider’s instructions and recommendations regarding the use of Fabrazyme, and inform them of any medical conditions or medications you are currently taking to ensure safe and effective treatment.

What special dietary instructions should I follow?

There are no specific dietary instructions associated with Fabrazyme treatment. However, maintaining a healthy and balanced diet is generally recommended to support overall health and well-being.

What should I do if I forget a dose?

If you miss a scheduled dose of Fabrazyme, contact your healthcare provider as soon as possible for guidance. They will advise you on the appropriate course of action, which may involve rescheduling the missed dose or adjusting your treatment plan as necessary. It’s essential to follow your healthcare provider’s instructions closely and not to double the dose to make up for the missed one unless directed to do so by your healthcare provider.

What side effects can this medication cause?

Fabrazyme (agalsidase beta) may cause side effects in some individuals. These side effects can vary in severity and may include:

• Infusion Reactions: Common infusion-related reactions may include fever, chills, headache, flushing, sweating, nausea, vomiting, chest pain, shortness of breath, dizziness, or low blood pressure. These reactions typically occur during or shortly after Fabrazyme infusion.
• Allergic Reactions: Some individuals may experience allergic reactions to Fabrazyme, which can manifest as rash, itching, hives, swelling of the face, lips, tongue, or throat, difficulty breathing, or wheezing. Severe allergic reactions, such as anaphylaxis, are rare but can be life-threatening.
• Flu-like Symptoms: Patients may experience flu-like symptoms such as fatigue, muscle or joint pain, and headache.
• Hypersensitivity Reactions: Hypersensitivity reactions, including anaphylaxis, have been reported in patients receiving Fabrazyme.
• Kidney Problems: Fabrazyme treatment may lead to worsening of kidney function in some patients, especially those with pre-existing kidney problems.
• Liver Problems: Liver enzyme abnormalities and liver function test abnormalities have been reported in patients receiving Fabrazyme.
• Heart Problems: Patients with Fabry disease may experience worsening of heart problems, including heart rhythm abnormalities or heart failure, during Fabrazyme treatment.
• Injection Site Reactions: Injection site reactions such as redness, swelling, pain, or irritation may occur.
• Other: Other potential side effects may include abdominal pain, diarrhea, constipation, peripheral edema (swelling of the limbs), or urinary tract infections.

It’s important to report any unusual or bothersome side effects to your healthcare provider promptly. They can help monitor your symptoms and determine whether any adjustments to your treatment plan are necessary. Additionally, seek immediate medical attention if you experience signs of a severe allergic reaction or other serious side effects.

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

Regarding the storage and disposal of Fabrazyme:

• Storage: Fabrazyme should be stored in the refrigerator at a temperature between 2°C to 8°C (36°F to 46°F). Do not freeze Fabrazyme, and protect it from light. Keep the medication in its original carton until ready to use to protect it from light. Do not use Fabrazyme after the expiration date printed on the carton or vial.
• Disposal: Unused or expired Fabrazyme should be disposed of properly according to local regulations. Consult with your healthcare provider or pharmacist for guidance on the appropriate disposal method.

In case of emergency/overdose

If you suspect an overdose or experience severe symptoms after receiving Fabrazyme, seek immediate medical attention or contact your local poison control center. Be prepared to provide information about the medication and the amount ingested or administered.

What other information should I know?

• Regular Monitoring: Patients receiving Fabrazyme should undergo regular monitoring by healthcare professionals to assess the effectiveness of treatment, monitor for any adverse reactions, and evaluate kidney and heart function.
• Continued Treatment: Fabrazyme is typically administered on a regular schedule as prescribed by your healthcare provider. It’s important to continue treatment as directed, even if you feel well, to maintain the benefits of therapy.
• Follow-up Care: Attend all scheduled follow-up appointments with your healthcare provider to discuss your progress, any concerns or side effects you may be experiencing, and to adjust your treatment plan as needed.
• Patient Support: Some patients may benefit from support groups or resources available for individuals with Fabry disease. These groups can provide emotional support, educational resources, and helpful tips for managing the condition.
• Travel Considerations: If you need to travel with Fabrazyme, consult with your healthcare provider for guidance on proper storage and transportation of the medication. Carry necessary documentation and contact information for emergency medical assistance while traveling.

Always follow your healthcare provider’s instructions and recommendations regarding the use of Fabrazyme and any other medical treatments. If you have any questions or concerns, do not hesitate to discuss them with your healthcare provider or pharmacist.