Myozyme

What is this drug and what is it used for ?

Myozyme is used to treat adults, children and adolescents of all ages who have a confirmed diagnosis of Pompe disease.
People with Pompe disease have low levels of an enzyme called alpha-glucosidase. This enzyme helps the body control levels of glycogen (a type of carbohydrate). Glycogen provides the body with energy, but in Pompe disease the levels of glycogen can get too high.
Myozyme contains an artificial enzyme called alglucosidase alfa – this can replace the natural enzyme which is lacking in Pompe disease.

How is this drug given?

The dose you receive is based on your body weight. The recommended dosage of Myozyme is 20 mg per kg of body weight. It will be given to you once every 2 weeks.

Instructions for proper use

Myozyme is given through a drip into a vein (by intravenous infusion). It is supplied as a powder which will be mixed with sterile water before it is given.

Possible side effects

Very severe generalised allergic reactions and anaphylactic shock, have been reported in some patients.

Very common: may affect more than 1 in 10 people

• Hives
• Rash
• Increased heart rate
• (Facial) flushing
• Fever or increased body temperature
• Cough
• Increased breathing rate
• Vomiting
• Low level of oxygen in the blood

Common: may affect up to 1 in 10 people

• Paleness
• Increased or high blood pressure
• Bluish discolouration of the skin
• Chills
• Agitation
• Tremor
• Headache
• Tingling
• Pain or local reaction at the site of the drip
• Dizziness
• Irritability
• Itchy skin
• Retching
• Swelling of the face, swelling of the throat or severe combined swelling of the face, throat and tongue due to a severe allergic reaction
• Swelling of the arms and legs
• Nausea
• Chest discomfort
• Throat tightness
• Diarrhoea
• Tiredness
• Muscle pain
• Muscle spasms
• Severe ulcerative lesions of the skin
• Redness of the skin