WARNING

Cipaglucosidase alfa (AT-GAA) is an investigational enzyme replacement therapy being developed for the treatment of Pompe disease, a rare genetic disorder. It’s important to note that developments in medical research may have occurred since then.

As with any medication or experimental treatment, there are potential risks and side effects associated with Cipaglucosidase alfa. Some of the general risks associated with enzyme replacement therapies for Pompe disease may include:

• Infusion-related reactions: Patients may experience infusion-related reactions, such as fever, chills, headache, and allergic reactions during or after the administration of the medication.
• Hypersensitivity: Some individuals may be hypersensitive to the drug, leading to more severe allergic reactions.
• Immune response: The body may develop an immune response to the medication, potentially reducing its effectiveness over time.
• Adverse effects on liver function: Enzyme replacement therapies may sometimes affect liver function, and regular monitoring is typically recommended.
• Interactions with other medications: Cipaglucosidase alfa may interact with other medications, and patients should inform their healthcare providers about all the medications they are taking.
• Unknown long-term effects: Since Cipaglucosidase alfa is still in the investigational stage, there may be unknown long-term effects or risks that have not yet been identified.

It’s crucial for individuals considering or undergoing treatment with Cipaglucosidase alfa to discuss potential risks and benefits thoroughly with their healthcare providers. Clinical trials and ongoing research play a significant role in understanding the safety and efficacy of such therapies, and the information provided here may not reflect the latest developments. Always consult with healthcare professionals for the most up-to-date and personalized information based on your specific health condition.

Why is this medication prescribed?

Cipaglucosidase alfa (AT-GAA) is an investigational enzyme replacement therapy designed for the treatment of Pompe disease, a rare genetic disorder. Pompe disease is characterized by a deficiency of the enzyme acid alpha-glucosidase (GAA), leading to the accumulation of glycogen in various tissues, particularly in muscles. Cipaglucosidase alfa aims to replace the deficient or dysfunctional GAA enzyme, reducing the glycogen buildup and addressing the underlying cause of the disease.

How should this medicine be used?

Cipaglucosidase alfa-atga is administered intravenously (into a vein) by a physician or nurse at a hospital or clinic. It is supplied as a powder that must be mixed with water. Every other week, it is often provided for more than four hours. After taking a dose of miglustat orally, you must begin a cipaglucosidase alfa-atga infusion within an hour and no later than three days. Your cipaglucosidase alfa-atga infusion will not be given to you if you neglect to take your miglustat. Before each dose of cipaglucosidase alfa-atga, your doctor may ask you to take or administer other drugs in order to ensure that you will tolerate your treatment.

As with any prescription medication, the use of Cipaglucosidase alfa-atga should be supervised by a qualified healthcare professional, and patients should inform their healthcare team about their medical history, ongoing medications, and any concerns they may have during the course of treatment.

Other uses for this medicine

This medication may be prescribed for other uses; ask your doctor or pharmacist for more information.

What special precautions should I follow?

Regarding special precautions, it’s important to note that specific guidance would be provided by healthcare professionals overseeing the treatment. However, general precautions for enzyme replacement therapies and investigational drugs may include:

• Monitoring: Regular monitoring of patients during and after infusion for any adverse reactions or infusion-related events.
• Hypersensitivity: Patients may be monitored for signs of hypersensitivity or allergic reactions during treatment.
• Liver function: Since enzyme replacement therapies can sometimes impact liver function, periodic monitoring of liver function may be recommended.
• Drug interactions: Informing healthcare providers about all medications, supplements, or other treatments being taken to avoid potential interactions.
• Pregnancy and breastfeeding: The safety of Cipaglucosidase alfa during pregnancy or breastfeeding may not be well-established, and healthcare providers would consider the potential risks and benefits in such situations.

As with any medication, individuals should follow their healthcare provider’s instructions and discuss any concerns or questions they may have. Since information is based on knowledge, individuals considering or undergoing treatment with Cipaglucosidase alfa should consult with their healthcare providers for the most up-to-date and personalized information based on the latest developments and clinical guidelines.

What special dietary instructions should I follow?

• In general, enzyme replacement therapies may not have specific dietary restrictions. However, it is crucial to maintain a balanced and healthy diet as advised by healthcare professionals.
• Individual dietary needs may vary, and any specific instructions regarding diet would be provided by the healthcare team.

What should I do if I forget a dose?

If you forget to take a dose of Cipaglucosidase alfa, the general guidance is as follows:

• Since Cipaglucosidase alfa is typically administered in a controlled healthcare setting, it is unlikely that individuals would be responsible for administering their own doses.
• However, if a dose is missed during a scheduled treatment session, it is important to inform the healthcare team promptly. The healthcare providers will decide on the appropriate course of action, which may include rescheduling the infusion.

What side effects can this medication cause?

However, based on the general class of medications known as enzyme replacement therapies, there are potential side effects and adverse reactions that could be associated with Cipaglucosidase alfa. It’s important to note that individual responses to medications can vary, and the following list is not exhaustive:

• Infusion-Related Reactions: Enzyme replacement therapies, when administered through infusion, may sometimes lead to infusion-related reactions. These reactions can include symptoms such as fever, chills, headache, and allergic reactions. Healthcare providers typically monitor patients closely during and after infusions to manage or prevent such reactions.
• Hypersensitivity or Allergic Reactions: Some individuals may be hypersensitive to the medication, resulting in more severe allergic reactions. These reactions can include rash, itching, swelling, or difficulty breathing. Prompt medical attention is crucial if any signs of severe allergic reactions occur.
• Liver Function Abnormalities: Enzyme replacement therapies can, in some cases, affect liver function. Regular monitoring of liver function may be recommended during treatment.
• Immune Response: The body may develop an immune response to the enzyme replacement therapy over time, potentially reducing its effectiveness. This is a consideration in the long-term management of the disease.
• Other: Other potential side effects may be identified through ongoing clinical trials and post-marketing surveillance.

It’s essential for individuals undergoing treatment with Cipaglucosidase alfa to be aware of potential side effects and to report any unusual symptoms or concerns to their healthcare providers promptly. Since information is based on knowledge, it’s crucial to consult with healthcare professionals for the most up-to-date and personalized information based on the latest developments in clinical trials and regulatory approvals.

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

Storage and Disposal of Cipaglucosidase alfa:

Storage:

• Enzyme replacement therapies are typically administered in a healthcare setting, and the storage of the medication is managed by healthcare professionals.
• If you are prescribed Cipaglucosidase alfa for home use, your healthcare provider will provide specific instructions on storage conditions. Follow these instructions carefully, and store the medication as directed.

Disposal:

• Proper disposal of unused or expired medication is essential. Follow any specific disposal instructions provided by your healthcare provider or pharmacist.
• In many cases, healthcare providers or pharmacies may offer guidance on proper disposal methods to ensure the medication is not accidentally ingested or accessed by unauthorized individuals.

In case of emergency/overdose

• In the case of an emergency or suspected overdose, seek immediate medical attention by calling emergency services (e.g., 911 in the United States) or your local emergency number.
• Provide healthcare professionals with as much information as possible, including the name of the medication (Cipaglucosidase alfa) and any details about the circumstances surrounding the emergency.

What other information should I know?

• Keep a record of your medical history, including allergies, current medications, and any known medical conditions.
• Attend regular follow-up appointments with your healthcare provider to monitor the progress of your treatment and address any concerns or questions.

Since Cipaglucosidase alfa is an investigational therapy, it’s essential to follow the guidance provided by your healthcare team and refer to any specific instructions or information they provide. If you have specific questions about storage, disposal, or emergency procedures for Cipaglucosidase alfa, consult directly with your healthcare provider or pharmacist for accurate and up-to-date information.