Alglucosidase Alfa (Intravenous)

Alglucosidase Alfa is a drug that contains a human enzyme known as acid alfa-glucosidase. It is used to aid digestion and absorption of glycogen.


Alglucosidase Alfa is used in people with Pompe disease (GAA deficiency), which is an incapacitating and often fatal disorder of the muscles. Patients with this problem are not able to make enough of the enzyme involved in digestion and absorption of glycogen. It is available in the U.S. under the brand names, Lumizyme and Myozyme. It belongs to the drug therapeutic class enzyme replacement and the pharmacologic class of enzymes.

Life-threatening anaphylactic, immune-mediated and severe allergic reactions have been seen in patients after and during infusion of Alglucosidase alfa. Patients must be observed, and appropriate supportive measures and treatment should be on standby.

Alglucosidase alfa is FDA approved. Myozyme, which is manufactured by Genzyme Corp, got its first FDA approval on April, 28th 2006. This medication is obtained by prescription and is administered by a trained healthcare professional only.

Conditions Treated

  • Infantile-onset Pompe disease

Type of Medicine

  • Enzyme

Side Effects

As with any drug, Alglucosidase Alfa, Intravenous Route does have some side effects, some of which are seen quite often. The doctor prescribing the drug will weigh the benefits of giving this drug up against the risks involved in using it. A doctor will need to be informed of all the diseases the patient may have and every medication they may be on. This information will help the doctor decide on the relative safety of giving the drug.

A doctor will look out for the following conditions which are quite often seen after administering the drug. The percentage refers to the frequency in which the side effects are observed.

  • Pyrexia (92%)
  • Diarrhea (62%)
  • Rash (54%)
  • Infusion reactions (51%)
  • Vomiting (49%)
  • A cough (46%)
  • Diaper dermatitis (46%)
  • Pneumonia (46%)
  • Otitis media (44%)
  • URI (44%)
  • Gastroenteritis (41%)
  • Pharyngitis (36%)
  • Respiratory distress (33%)
  • Oral candidiasis (31%)
  • Respiratory Failure (31%)
  • Anemia (31%)
  • Catheter-related infection (28%)
  • Rhinorrhea (28%)
  • GERD (26%)
  • Post-procedural pain (26%)
  • Constipation (23%)
  • Tachycardia (23%)
  • Nasopharyngitis (23%)
  • Bronchiolitis (23%)
  • Tachypnea (23%)
  • Flushing (21%)
  • Bradycardia (21%)
  • Urticaria (21%)

The frequency of the following side effects are is not well defined:

  • Agitation
  • Abdominal pain
  • Bronchospasm
  • Blurred vision
  • Conjunctivitis
  • Coronary artery disease
  • Dyspnea
  • Facial Erythema
  • Hypokalemia
  • Insomnia
  • Irritability
  • Pallor
  • Restlessness
  • Tremor
  • Vertigo

The following are common side effects that a patient should look out for. Inform a doctor immediately if they are observed:

  • Blue fingertips, lips, and skin
  • Chest pain and discomfort
  • Body pains and aches
  • Cough
  • Chills
  • Labored or difficult breathing. Breathing may also become irregular, shallow, fast, or slow
  • Swallowing difficulties
  • Dizziness
  • Skin that is hot, red, dry, and irritated
  • Congestion in the ears
  • Heartbeat or pulse becoming fast, irregular or pounding
  • Feeling warm
  • Fever
  • Headache
  • More sweating
  • Itching, hives or skin rash
  • Dizziness, lightheadedness or fainting.
  • Nasal congestion
  • Losing voice
  • Skin turning pale
  • Swelling or puffiness around the eyes, on the eyelids, lips face or tongue
  • Reddening of arms, neck, face and at times upper chest - the catheter site can also become red and painful
  • Runny nose
  • Shakiness of arms, hands, legs, or feet
  • Sneezing
  • Sore throat
  • Lymph glands in the armpits, neck or groin may become swollen, tender, and painful
  • Unusual bruising or bleeding
  • Unusual tiredness and weakness

The following side effects are not so common, but can also be severe:

  • Passing bloody urine
  • Having decreased urine output
  • Convulsions
  • Dry mouth
  • Increased thirst
  • Appetite loss
  • Mood changes
  • Muscle cramps or pain
  • Vomiting or nausea
  • Tingling or numbness in the lips, feet or hands
  • Genital or groin pain
  • Sharp pain the back just below the ribs

The incidence of the following effects is not known, but a doctor must be notified when they occur.

  • Skin discoloration ranging from blue-green to black
  • Confusion
  • Blurred vision
  • Dilated neck veins
  • Extreme fatigue
  • The heart may stop
  • Difficulties speaking or slurred speech
  • Breathing may stop, and there may be no pulse or blood pressure
  • Seizures
  • Severe sudden headache or pain in the chest
  • Severe and sudden weakness on the arms or legs on any one side of the body
  • Severe breathing difficulties that happen suddenly
  • Temporary blindness
  • Weight gain

The preceding is not in any way a comprehensive list of all side effects that the patient may observe. A doctor should be notified of all signs seen after use of this drug, as only a trained healthcare professional can determine the seriousness of any symptoms.


Alglucosidase Alfa is available as a powder for a solution and is administered intravenously. For reconstitution and preparation techniques, the manufacturer's leaflet should be consulted. The final concentration should be about 0.5 - 4mg/mL.

The following dosages apply to drug strengths of 50mg.

1. Adult dose for Pompe disease

The usual dose is 20mg/kg of body weight administered via IV infusion every two weeks.

The total volume of drug administered depends on the body weight of the patient and should be infused over four hours.

2. Pediatric dose of Pompe disease

The usual dose is 20mg per Kg of body weight administered by IV infusion every two weeks.

The volume of the drug administered is determined by the patient's body weight and should be infused over about over four hours.

3. Liver and Rena Dose Adjustments

There is no data available on dose adjustments for people with kidney and liver problems.

4. Dialysis

No data is available on drug adjustments for people undergoing dialysis.

NOTE: Healthcare providers should be prepared to handle any life-threatening and severe hypersensitivity reactions that may occur during or after administration of this drug.


For a patient using this medication, frequent visits should be made to the doctor to ensure the medicine works well. Alglucosidase Alfa administered intravenously does not interact much with most medications. However, a doctor must be informed of all the drugs a patient receiving this therapy is taking.

There may be some insignificant interactions that the doctor may note and monitor. At times, a doctor may advise that certain medications be stopped as they may interact in clinically significant ways, and might endanger the patient. There are instances where the doctor will allow certain drugs to be used together despite interactions, but he may adjust dosages.

Disease interactions

Alglucosidase Alfa reacts with the following diseases and should be used with caution in patients with the following conditions:

  • Heart disease
  • Heart rhythm problems (arrhythmia)
  • Cardiac hypertrophy (where the heart is larger than usual)
  • Breathing problems


Patients with infantile-onset Pompe disease who also have compromised respiratory or cardiac function must be monitored carefully after administration of Alglucosidase Alfa as they are at risk of a severe acute worsening due to fluid overload. A doctor must carefully watch the patient during and after treatment to be sure the medicine is working well. Blood tests need to be done regularly to check for any unwanted effects.

The drug is given by a nurse or other healthcare professional in a hospital and is administered by a needle placed in the veins. The needle remains in place for a few hours, and additional medicine must be administered to prevent allergic reactions to Alglucosidase Alfa.

A doctor must be informed of all the substances that a patient receiving this drug is allergic to. These include all preservatives, additives, pesticides, animal products, foods, and medicines that the patient reacts to.

Alglucosidase Alfa can cause some severe symptoms a few hours after it is given. A doctor must be informed of all observed symptoms. Alglucosidase may cause some severe types of allergic reactions such as anaphylaxis. Anaphylaxis is life-threatening and requires immediate medical attention. If a patient shows the following symptoms, a doctor should be informed immediately:

  • Hoarseness
  • Rash
  • Itching
  • Lightheadedness
  • Dizziness
  • Fainting
  • Trouble breathing and swallowing
  • Swelling of face, mouth or hands.

Pregnancy warnings

Animal studies may have shown an adverse effect on fetuses, but adequate studies are unavailable among pregnant women.

Breastfeeding warnings

The studies to determine the risk posed to a nursing infant when a breastfeeding mother uses this drug are inadequate. This medicine should be used with caution by a breastfeeding mother.


Vials of freeze-dried powder or cake should be stored in the refrigerator at 2-8 degrees centigrade (36 to 46 degrees F).

The diluted and reconstituted solution is stable up to 24 hours at 2 to 8 degrees C (36 to 46 degrees F) when the immediate use of the solution is not possible. The reconstituted solution should not be stored at room temperature. It should also not be frozen or shaken.

The same IV line should not be used to infuse Alglucosidase Alfa and other products.


Despite the apparently severe side effects of Alglucosidase Alfa, a doctor will prescribe this drug if the benefits of using it outweigh the risks. A patient should never use information like the preceding to determine whether or not a drug is right for them. A healthcare professional is the only one who has the necessary training and expertise to prescribe medication.

All concerns about a particular medication should be discussed with a healthcare provider. A patient should never decide how to remedy any of their symptoms or side effects or choose to add a drug, supplement or other natural product to what they have prescribed by a medical professional. Only a person with the required expertise can determine possible interactions of combined products or drugs, and be able to make any dose adjustments that may be necessary.

The preceding information on Alglucosidase Alfa is not complete, as it is not possible to include everything here. The information is not meant to replace the advice of a medical practitioner and should be used for general purposes only. A doctor must always be consulted in case of any symptoms, and the input of a pharmacist or other professional sought when over the counter drugs are to be purchased. It does not in any way endorse Alglucosidase Alfa as safe, approved or useful for the treatment of any condition or patient.

Last Reviewed:
December 10, 2017
Last Updated:
April 04, 2018