FAQs About Lumizyme for Pompe Disease

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by Brian Murphy, Ph.D. |

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Lumizyme FAQs

Lumizyme (alglucosidase alfa) is an enzyme replacement infusion therapy by Sanofi Genzyme for people with Pompe disease. In the European Union, it is available under the brand name Myozyme.

Here are answers to some frequently asked questions about this treatment.

How does Lumizyme work?

Lumizyme works to replace the missing or deficient acid alpha-glucosidase enzyme. The body normally uses this enzyme to break down a complex sugar molecule called glycogen. Mutations in the GAA gene cause cells not to be able to produce enough of this enzyme correctly. Its lack in Pompe disease patients leads to a buildup of glycogen.

Lumizyme is an artificial version of the alpha-glucosidase enzyme that is produced from genetically engineered Chinese hamster ovary (CHO) cells in the laboratory. These cells have been modified to produce the human enzyme, which is then collected and purified for patient use.

How do I take it?

Lumizyme is administered via an intravenous (IV) infusion directly into the bloodstream. Clinicians initially mix the Lumizyme powder with sterile water, and then transfer the mixture to an infusion bag of saline (water with various dissolved salts).

Once the solution is ready, an IV will be inserted into a vein in you or your child, typically in the arm or hand. A pump then slowly delivers the solution in a process estimated to take about four hours. Vital signs, such as oxygen levels and heart rate, are recorded periodically during the infusion to make sure that you or your child are not having a reaction to Lumizyme or the infusion process.

How often should I or my child receive infusions?

The recommended infusion schedule for Lumizyme is once every two weeks. Your doctor, however, may recommend a different schedule based on your individual enzyme levels and how you are responding to the treatment.

What are the side effects?

Lumizyme  is known to potentially carry serious side effects. The therapy can cause a severe allergic reaction called anaphylaxis that could be life-threatening. Symptoms of anaphylaxis include difficulty breathing, abnormal heart rate, low blood pressure, swelling of the face and tongue, kidney dysfunction, and skin lesions.

There is also a risk of damage to the lungs or heart, or an acute worsening of those organs, with children who already have heart or breathing problems due to Pompe disease.

The treatment may also lead to an immune reaction, in which the body makes antibodies against Lumizyme that can limit its future effectiveness.

Allergy reactions — hypersensitivity to a medication — were  most common side effects of Lumizyme’s use in clinical trials. These reactions include anaphylaxis, rash, fever, feeling hot, hives, headache, excessive sweating, nausea, cough, lower blood oxygen levels, a rapid heart rate, fast breathing, tightness or discomfort in the chest, dizziness, muscle twitches, skin discoloration, agitation, elevated blood pressure, paleness of the face, chills, tremors, vomiting, fatigue, and muscle pain.

It is important to tell your doctor of any reactions you experience or see in your child during or after an infusion.

Are there reasons I shouldn’t take Lumizyme?

If you are pregnant or breastfeeding, inform your doctor before taking Lumizyme.

The effect of Lumizyme on the mother during pregnancy or her fetus has not been formally studied, but case reports of women using Lumizyme while pregnant have not shown treatment-related increases in major birth defects, miscarriage risks, or other harm to the mother or unborn child.

Studies suggest that Lumizyme can pass into breastmilk, but no side effects have been reported to date in a nursing baby.

The Pompe Registry is monitoring Lumizyme’s use in all patients, including pregnant mothers and nursing infants, and participation is encouraged for a better understanding of potential risks.

Lumizyme has not been studied extensively in patients 65 or older, and the risks of its use by these people are not clear.  If you are in this age group, speak to your doctor before beginning treatment.

 

Last updated: Feb. 2, 2021

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