How does this medication work? What will it do for me?

Taliglucerase alfa belongs to the class of medications called enzyme replacement therapies. Specifically, it is a replacement for the enzyme glucocerebrosidase, a protein that is normally found in the body and is responsible for breaking down certain types of fatty substances.

It is used to treat Type 1 Gaucher disease in adults and Type 1 and Type 3 Gaucher disease in children. Gaucher disease is a rare genetic condition where the protein responsible for breaking down certain waste products in the cells is not present or as active as it needs to be.

Taliglucerase alfa works by replacing this enzyme, so the body can recycle the wastes normally.

This medication may be available under multiple brand names and/or in several different forms. Any specific brand name of this medication may not be available in all of the forms or approved for all of the conditions discussed here. As well, some forms of this medication may not be used for all of the conditions discussed here.

Your doctor may have suggested this medication for conditions other than those listed in these drug information articles. If you have not discussed this with your doctor or are not sure why you are taking this medication, speak to your doctor. Do not stop taking this medication without consulting your doctor.

Do not give this medication to anyone else, even if they have the same symptoms as you do. It can be harmful for people to take this medication if their doctor has not prescribed it.

What form(s) does this medication come in?

Each vial of sterile, non-pyrogenic, white-to-off-white lyophilyzed powder for reconstitution and intravenous infusion contains 212 units of taliglucerase alfa to allow 200 units to be withdrawn from the vial. Nonmedicinal ingredients: mannitol, sodium citrate, polysorbate 80, and citric acid anhydrous to adjust pH.

How should I use this medication?

The appropriate dose of taliglucerase alfa is based on body weight and varies from person to person, depending on how well it is tolerated and how much is needed to be effective.

Taliglucerase alfa is given by intravenous (into a vein) infusion over 1 to 2 hours, every 2 weeks.

Careful handling of this medication is required, and it is commonly given in a hospital or similar setting with access to sterile equipment for preparation. Under some circumstances, you may be taught to prepare and administer this medication at home.

Many things can affect the dose of medication that a person needs, such as body weight, other medical conditions, and other medications. If your doctor has recommended a dose different from the ones listed here, do not change the way that you are taking the medication without consulting your doctor.

It is important this medication be given exactly as recommended by your doctor. If you miss an appointment to receive taliglucerase alfa, contact your doctor as soon as possible to reschedule your appointment.

If you are storing this medication at home, keep it in a refrigerator (2°-8°C) and transport refrigerated (2°-8°C). Protect from light. Keep the vial in the outer carton.

Do not dispose of medications in wastewater (e.g. down the sink or in the toilet) or in household garbage. Ask your pharmacist how to dispose of medications that are no longer needed or have expired.

Who should NOT take this medication?

Do not take this medication if you are allergic to taliglucerase alfa or any ingredients of the medication.

What side effects are possible with this medication?

Many medications can cause side effects. A side effect is an unwanted response to a medication when it is taken in normal doses. Side effects can be mild or severe, temporary or permanent.

The side effects listed below are not experienced by everyone who takes this medication. If you are concerned about side effects, discuss the risks and benefits of this medication with your doctor.

The following side effects have been reported by at least 1% of people taking this medication. Many of these side effects can be managed, and some may go away on their own over time.

Contact your doctor if you experience these side effects and they are severe or bothersome. Your pharmacist may be able to advise you on managing side effects.

  • abdominal pain
  • dizziness
  • fatigue
  • flushing
  • headache
  • nausea
  • vomiting
  • weight gain

Although most of the side effects listed below don't happen very often, they could lead to serious problems if you do not seek medical attention.

Check with your doctor as soon as possible if any of the following side effects occur:

  • back pain
  • bone pain
  • infusion site pain
  • joint pain
  • pain in extremities (e.g., wrists, hands, ankles, feet)
  • swelling
  • throat irritation
  • rash, itching

Stop taking the medication and seek immediate medical attention if any of the following occur:

  • difficulty breathing
  • signs of a serious allergic reaction (e.g., abdominal cramps, difficulty breathing, nausea and vomiting, or swelling of the face and throat)
  • symptoms of infusion reaction (e.g., low blood pressure, dizziness, slow heart beat, shortness of breath, fever, chills, hives)

Some people may experience side effects other than those listed. Check with your doctor if you notice any symptom that worries you while you are taking this medication.

Are there any other precautions or warnings for this medication?

Before you begin using a medication, be sure to inform your doctor of any medical conditions or allergies you may have, any medications you are taking, whether you are pregnant or breast-feeding, and any other significant facts about your health. These factors may affect how you should use this medication.

Allergic reactions: Some people may develop a serious allergic reaction to this medication. This reaction can occur up to 24 hours after receiving a dose of taliglucerase alfa. Signs of an allergic reaction include a severe rash, hives, swollen face or throat, or difficulty breathing. If these occur, get immediate medical attention.

Dizziness: Taliglucerase may cause dizziness. Do not drive, operate machinery, or perform other hazardous tasks until you have determined how this medication affects you.

Infusion-related reactions: As with other enzyme therapies, taliglucerase alfa can cause severe infusion-related reactions, including severe allergic reactions. Your health care provider will closely monitor you during and after the infusion, and may give you other medications before you receive the taliglucerase alfa, to prevent these reactions.

If you experience symptoms of an infusion reaction, such as fever, chills, difficulty breathing, tightness of chest or throat, stomach upset, and rash, tell your health care provider immediately. Symptoms can usually be reduced by slowing down or temporarily stopping the medication, as well as using other medications to treat the reaction.

It is important to take the medication(s) recommended by your doctor before your infusion of taliglucerase alfa. If you experience a severe infusion reaction, your doctor will give you additional medications to treat the reaction. Ask your doctor for more information.

Pregnancy: This medication should not be used during pregnancy unless the benefits outweigh the risks. If you become pregnant while taking this medication, contact your doctor immediately.

Breast-feeding: It is not known if taliglucerase alfa passes into breast milk. If you are a breast-feeding mother and are taking this medication, it may affect your baby. Talk to your doctor about whether you should continue breast-feeding.

Children: The safety and effectiveness of using this medication have not been established for children less than 2 years old.

What other drugs could interact with this medication?

Tell your doctor or prescriber about all prescription, over-the-counter (non-prescription), and herbal medications that you are taking. Also tell them about any supplements you take. Since caffeine, alcohol, the nicotine from cigarettes, or street drugs can affect the action of many medications, you should let your prescriber know if you use them. Depending on your specific circumstances, your doctor may want you to:

  • stop taking one of the medications,
  • change one of the medications to another,
  • change how you are taking one or both of the medications, or
  • leave everything as is.

An interaction between two medications does not always mean that you must stop taking one of them. Speak to your doctor about how any drug interactions are being managed or should be managed.