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Generic name: taliglucerase alfa
Drug class: Lysosomal enzymes

Medically reviewed by  A Ras MD.

What is Elelyso?

Elelyso is a prescription medicine that is used to treat Gaucher’s disease.


Taliglucerase alfa is a hydrolytic lysosomal glucocerebroside-specific enzyme produced by recombinant DNA technology using plant cell culture (carrot). Taliglucerase alfa is a monomeric glycoprotein enzyme containing 4 N-linked glycosylation sites (kDa=60.8). Taliglucerase alfa differs from native human glucocerebrosidase by two amino acids at the N terminal and up to 7 amino acids at the C terminal. Taliglucerase alfa is a glycosylated protein with oligosaccharide chains at the glycosylation sites having terminal mannose sugars. These mannose-terminated oligosaccharide chains of taliglucerase alfa are specifically recognized by endocytic carbohydrate receptors on macrophages, the cells that accumulate lipid in Gaucher disease.

A unit is the amount of enzyme that catalyzes the hydrolysis of 1 micromole of the synthetic substrate para-nitrophenyl-β-D-glucopyranoside (pNP-Glc) per minute at 37°C.

ELELYSO (taliglucerase alfa) for injection is supplied as a sterile, preservative-free, lyophilized powder for reconstitution and dilution prior to intravenous infusion. Each single-dose vial contains 200 units of taliglucerase alfa and D-mannitol (206.7 mg), polysorbate 80 (0.56 mg), and sodium citrate (30.4 mg). Citric acid may be added to adjust the pH at the time of manufacture. After reconstitution with 5.1 mL Sterile Water for Injection, USP, taliglucerase alfa concentration is 40 units/mL . Reconstituted solutions have a pH of approximately 6.0.

Mechanism of Action

Gaucher disease is an autosomal recessive disorder caused by mutations in the human glucocerebrosidase gene, which results in a reduced activity of the lysosomal enzyme glucocerebrosidase. Glucocerebrosidase catalyzes the conversion of the sphingolipid glucocerebroside into glucose and ceramide. The enzymatic deficiency results in accumulation of substrate glucocerebroside primarily in the lysosomal compartment of macrophages, giving rise to foam cells or “Gaucher cells,” which accumulate in the liver, spleen and bone marrow.

ELELYSO, a long term enzyme replacement therapy, is a recombinant analog of human lysosomal glucocerebrosidase that catalyzes the hydrolysis of glucocerebroside to glucose and ceramide, reducing the amount of accumulated glucocerebroside. ELELYSO uptake into cellular lysosomes is mediated by binding of ELELYSO mannose oligosaccharide chains to specific mannose receptors on the cell surface leading to internalization and subsequent transport to the lysosomes.

Before taking Elelyso, tell your doctor:

  • If you are allergic to Elelyso; any part of this medicine; or any other drugs, foods, or substances. Tell your doctor about the allergy and what signs you had.

This medicine may interact with other drugs or health problems.

Tell your doctor and pharmacist about all of your drugs (prescription or OTC, natural products, vitamins) and health problems. You must check to make sure that it is safe for you to take Elelyso with all of your drugs and health problems. Do not start, stop, or change the dose of any drug without checking with your doctor.

What are some things I need to know or do while I take Elelyso?

  • Tell all of your health care providers that you take Elelyso. This includes your doctors, nurses, pharmacists, and dentists.
  • Have blood work checked as you have been told by the doctor. Talk with the doctor.
  • Some people have unsafe allergic effects or bad side effects during the infusion or within 3 hours of the infusion. Talk with the doctor.
  • Other drugs may be given before Elelyso to help avoid side effects.
  • Tell your doctor if you are pregnant, plan on getting pregnant, or are breast-feeding. You will need to talk about the benefits and risks to you and the baby.

How is Elelyso best taken?

Use Elelyso as ordered by your doctor. Read all information given to you. Follow all instructions closely.

  • It is given as an infusion into a vein over a period of time.

What do I do if I miss a dose?

  • Call your doctor to find out what to do.

What are the side effects of Elelyso that I need to call my doctor about immediately?

WARNING/CAUTION: Even though it may be rare, some people may have very bad and sometimes deadly side effects when taking a drug. Tell your doctor or get medical help right away if you have any of the following signs or symptoms that may be related to a very bad side effect:

  • Signs of an allergic reaction, like rash; hives; itching; red, swollen, blistered, or peeling skin with or without fever; wheezing; tightness in the chest or throat; trouble breathing, swallowing, or talking; unusual hoarseness; or swelling of the mouth, face, lips, tongue, or throat.
  • Dizziness or passing out.
  • Cough.
  • Flushing.
  • Upset stomach or throwing up.
  • Throat irritation.

What are some other side effects of Elelyso?

All drugs may cause side effects. However, many people have no side effects or only have minor side effects. Call your doctor or get medical help if any of these side effects or any other side effects bother you or do not go away:

  • Headache.
  • Joint pain.
  • Feeling tired or weak.
  • Stomach pain.
  • Pain in arms or legs.

These are not all of the side effects that may occur. If you have questions about side effects, call your doctor. Call your doctor for medical advice about side effects.

You may report side effects to the FDA at 1-800-332-1088. You may also report side effects at https://www.fda.gov/medwatch.

If overdose is suspected:

If you think there has been an overdose, call your poison control center or get medical care right away. Be ready to tell or show what was taken, how much, and when it happened.

How do I store and/or throw out Elelyso?

  • If you need to store Elelyso at home, talk with your doctor, nurse, or pharmacist about how to store it.



  • NDC 0069-0106-01
  • Pfizer
  • Elelyso®
    (taliglucerase alfa)
    for injection
  • 200 units/vial
  • For intravenous infusion only
  • Single-Dose Vial. Discard any
    unused portion.
    Rx only
  • PRINCIPAL DISPLAY PANEL - 200 unit Vial Carton




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