Myozyme – Alglucosidase alfa uses, dose and side effects


50 mg powder for concentrate for solution for infusion alglucosidase

What Myozyme is and what it is used for

Myozyme is used to treat adults, children, and adolescents of all ages where the diagnosis of Pompe disease is established.

People with Pompe disease have low levels of an enzyme called alpha-glucosidase. This enzyme helps control glycogen levels (a type of carbohydrate) in the body. Glycogen provides the body with energy, but if you suffer from Pompe disease, glycogen levels can become too high.

Myozyme contains an artificial enzyme called alglucosidase alfa which can replace the human enzyme that is missing in Pompe disease.

What you need to know before you get Myozyme

Do not use Myozyme

If you have experienced life-threatening allergic (hypersensitivity) reactions to alglucosidase alfa or any of the other ingredients of this medicine (listed in section 6) and if re-administration of the medicine was not successful. Symptoms of life-threatening allergic reactions include (but are not limited to) low blood pressure, very fast heart rate, difficulty breathing, vomiting, swelling of the face, hives, or rash.

Warnings and cautions

If you are being treated with Myozyme, you may experience an infusion-related side effect when you receive the medicine or in the next few hours after the infusion. Such a side effect can consist of various symptoms such as low blood pressure, chest discomfort, swelling of the throat, swelling of the face, lips, or tongue ( angioedema ), hives ( urticaria ), dizziness, rash, itchy skin, nausea, vomiting, cough, and bronchospasm (see section 4 for an overview of all infusion-related side effects ). Such an infusion-related side effect can in some cases be very serious. If you experience a similar side effect, you should contact a doctor immediately. You may need medication before treatment to prevent an allergic reaction (eg antihistamines and/or corticosteroids ) or antipyretics.

To reduce the production of antibodies, doctors in studies have used drugs that inhibit the immune system. Because you have Pompe disease, there is a risk of getting a serious infection in the airways or lungs. By using drugs that inhibit the immune system, the risk can increase further.

If you experience severe wound damage to the skin, consult a doctor. If your legs swell or if you feel generally swollen, consult a doctor. Your doctor should consider stopping Myozyme treatment and starting appropriate medical treatment. Your doctor should consider the risks and benefits of reintroducing Myozyme.

Other medicines and Myozyme

Tell your doctor or pharmacist if you are taking, have recently taken, or might take any other medicines.

Pregnancy, breastfeeding and fertility

There is no experience with the use of Myozyme in pregnant women. You should not receive Myozyme during pregnancy unless necessary. It is recommended that you stop breastfeeding during treatment with Myozyme. Ask your doctor or pharmacist for advice before taking any medicine. If you are pregnant or breastfeeding, think you may be pregnant, or are planning to have a baby, ask your doctor or pharmacist for advice before taking this medicine.

Driving and using machines

Use caution when driving or using machines shortly after infusion of Myozyme. You may experience dizziness.

Myozyme contains sodium

This medicine contains less than 1 mmol sodium (23 mg) per vial and is therefore essentially ‘sodium-free’.

How Myozyme is given

You will be given Myozyme by a doctor who has experience in treating patients with Pompe disease.

You will receive a dose based on your body weight. The recommended dose for Myozyme is 20 mg/kg body weight every two weeks.

Use for children and adolescents

The recommended dose for children and adolescents is the same as for adults.

Instructions for proper use

Myozyme is given by a drip into a vein (as an intravenous infusion ). It is supplied in powder form and must be mixed with sterile water before use.

If you take more Myozyme than you should

There is no experience with Myozyme overdose.

If you forget to use Myozyme

If you have missed an infusion, consult your doctor.

If you have any further questions on the use of this product, ask your doctor, pharmacist, or nurse.

Possible side effects

Like all medicines, this medicine can cause side effects, although not everybody gets them.

Adverse reactions were mainly reported when patients were being treated or shortly thereafter (infusion-related adverse reactions ). Some of these side effects are serious or life-threatening. Life-threatening side effects, including very severe generalized allergic reactions and anaphylactic shock, have been reported in some patients. Symptoms of such reactions include low blood pressure, very fast heart rate, difficulty breathing, vomiting, swelling of the face, lips, or tongue, hives, or rash. Some patients have experienced side effects with flu-like symptoms associated with the infusion one. The symptoms persisted for a few days after the end of the infusion. If you get a similar side effect, contact your doctor immediately. You may need medication before treatment to prevent an allergic reaction (eg antihistamines and/or corticosteroids ) or antipyretics.

Very common: may affect more than 1 in 10 people

  • hives
  • rash
  • increased heart rate
  • flushing
  • fever or elevated body temperature
  • cough
  • faster breathing
  • vomiting
  • low oxygen level in the blood

Common: may affect up to 1 in 10 people

  • pallor
  • high or high blood pressure
  • bluish discoloration of the skin
  • overindulge
  • indignation
  • tremors
  • headache
  • knitting
  • pain or local reactions at the infusion site
  • dizziness
  • irritability
  • itching
  • nausea
  • swollen face, swollen throat or severe, combined swelling of the face, throat or tongue due to a severe allergic reaction
  • swelling in arms and legs
  • nausea
  • chest discomfort
  • tightness in the throat
  • diarrhea
  • fatigue
  • muscle pain
  • muscle spasms
  • severe wound damage to the skin
  • reddening of the skin

No known frequency: can not be calculated from the available data.

  • swelling around the eyes
  • abnormal breathing sounds, including a whistling sound
  • difficulty breathing (including shortness of breath)
  • cold in the extremities (eg hands, feet)
  • low blood pressure
  • narrowed blood vessels that reduce blood flow
  • sudden contraction of the bronchi which limits the amount of air that passes into and out of the lungs (bronchospasm)
  • feeling of warmth
  • sweating
  • increased tear flow
  • mottled skin
  • restlessness
  • wheezing breathing sounds
  • lowered heart rate
  • cardiac arrest
  • chest pain (not in the heart)
  • inflammation of the membranes covering the eyeball and eyelids
  • abdominal pain
  • joint pain
  • temporary respiratory arrest or sudden respiratory arrest
  • protein loss in the urine
  • nephrotic syndrome: swelling of the legs, general swelling and loss of protein in the urine
  • swelling and thickening of the skin at the infusion site in case of leakage of the product outside the blood vessels

How to store Myozyme

Keep this medicine out of the sight and reach of children.

Do not use this medicine after the expiry date which is stated on the label after “EXP”. The expiration date is the last day of the specified month.

Store in a refrigerator (2 ° C – 8 ° C).

After dilution, the product should be used immediately. However, chemical and physical in-use stability has been demonstrated for up to 24 hours at 2 to 8 ° C and when stored in light protection.

Medicines should not be disposed of via wastewater or household waste. Ask your pharmacist how to dispose of medicines no longer required. These measures will help to protect the environment.

Contents of the pack and other information

Content declaration

  • The active substance is algal glucosidase alfa. One vial contains 50 mg of alglucosidase alfa. After reconstitution, the solution contains 5 mg of alglucosidase alfa / ml and, after dilution, the concentration varies between 0.5 mg and 4 mg per ml.
  • The other ingredients are
    • Mannitol (E421)
    • Sodium hydrogen phosphate monohydrate (E339)
    • Sodium dihydrogen phosphate heptahydrate (E339)
    • Polysorbate 80 (E433).

What the medicine looks like and contents of the pack

Myozyme is a powder for concentrate for solution for infusion in a vial (50 mg/vial). Each pack contains 1, 10, or 25 vials. Not all pack sizes may be marketed.

The powder is white to off-white. After reconstitution, it is clear, colorless to a pale yellow solution, which may contain particles. The reconstituted solution must be further diluted.

Marketing Authorisation Holder

Genzyme Europe BV, Paasheuvelweg 25, 1105 BP Amsterdam, The Netherlands

Genzyme Ireland Limited, IDA Industrial Park, Old Kilmeaden Road, Waterford, Ireland

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