What Is Alglucosidase Alfa?

Alglucosidase alfa is a medication prescribed to treat Pompe disease, which is a rare disorder that causes severe heart problems. Patients with this disease cannot naturally produce a key enzyme called acid alpha-glucosidase. Alglucosidase alfa is a glycoprotein that acts as a replacement for this enzyme. Patients should discuss the potential risks and side effects of this drug with a physician before taking it.

This medication is administered intravenously at a hospital, the dosage based on the patient's weight. Each infusion typically takes about four hours to administer, and the patient will be closely monitored for signs of an adverse reaction. Throughout the treatment and once per year afterward, patients will need to undergo blood tests to monitor their reactions to the drug.

Alglucosidase alfa carries the risk of a serious allergic reaction in some patients. This may occur up to three hours following a dose, and requires immediate medical care. Patients may exhibit coughing, wheezing, and shortness of breath. Their face, lips, and throat may swell, and they may develop a skin rash with itching. Rarely, allergic reactions to alglucosidase alfa may also result in cardiac arrest, and can be life-threatening.

Some side effects may occur with the use of this drug, which should be reported to the prescribing physician if they become severe. Patients may experience vomiting, constipation, or diarrhea. A runny nose and heartburn may develop, along with an upset stomach and flushing.

More serious side effects require immediate medical care, such as increased tear production, excessive sweating, and muscle twitching. Patients should see their doctors if they experience a slow or rapid heartbeat, problems breathing, and dizziness. White patches in the mouth, blue skin or nails, and purplish skin discoloration have been reported as well as muscle pain or stiffness, changes in hearing, and ear pain. Alglucosidase alfa may also cause vision changes, a severe headache, and irritability.

Before using alglucosidase alfa injections to treat Pompe disease, patients must disclose their other medical conditions, medications, and supplements to avoid a possible interaction. Women who are pregnant, may become pregnant, or breastfeeding should discuss the potential risks with their doctors. Patients should inform their doctors if they have any allergies, or if they have ever had an adverse reaction to any intravenous medicines. Alglucosidase alfa injections may be contraindicated for use by those with lung problems or heart problems, including an enlarged heart.