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Generic name: eliglustat
Brand name: Cerdelga
Dosage form: oral capsule (84 mg)
Drug class: Miscellaneous metabolic agents

Medically reviewed by  A Ras MD.

What is eliglustat?

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


CERDELGA (eliglustat) capsules contain eliglustat tartrate, which is a small molecule inhibitor of glucosylceramide synthase that resembles the ceramide substrate for the enzyme, with the chemical name N-((1R,2R)-1-(2,3-dihydrobenzo[b][1,4]dioxin-6-yl)-1-hydroxy-3-(pyrrolidin-1-yl)propan-2-yl)octanamide (2R,3R)-2,3-dihydroxysuccinate. Its molecular weight is 479.59, and the empirical formula is C23H36N2O4+½(C4H6O6) with the following chemical structure:

Chemical Structure

Each capsule of CERDELGA for oral use contains 84 mg of eliglustat (equivalent to 100 mg of eliglustat tartrate). The inactive ingredients are candurin silver fine, FD&C blue 2, gelatin, glyceryl behenate, hypromellose, lactose monohydrate, microcrystalline cellulose, and yellow iron oxide.

Mechanism of Action

Gaucher disease is caused by a deficiency of the lysosomal enzyme acid β-glucosidase. Acid β-glucosidase catalyzes the conversion of the sphingolipid glucocerebroside into glucose and ceramide. The enzymatic deficiency causes an accumulation of glucosylceramide (GL-1) primarily in the lysosomal compartment of macrophages, giving rise to foam cells or “Gaucher cells.”

The clinical features of this lysosomal storage disorder (LSD) are reflective of the accumulation of Gaucher cells in the reticuloendothelial system (liver, spleen, bone marrow, and other organs). The accumulation of Gaucher cells in the liver, spleen, and bone marrow leads to organomegaly and skeletal disease. Presence of Gaucher cells in the bone marrow and spleen leads to clinically significant anemia and thrombocytopenia.

CERDELGA is a specific inhibitor of glucosylceramide synthase (IC50=10 ng/mL) and acts as a substrate reduction therapy for GD1 by reducing the production of GL-1. By reducing GL-1 production, CERDELGA alleviates the accumulation of GL-1 in the target organs.

Before taking eliglustat, tell your doctor:

  • If you are allergic to eliglustat; any part of this medicine; or any other drugs, foods, or substances. Tell your doctor about the allergy and what signs you had.
  • If you have heart problems, like heart block, heart failure, long QT syndrome, or slow heartbeat.
  • If you have had a recent heart attack.
  • If you have any of these health problems: Kidney disease or liver disease.
  • If you are taking any drugs used for a heartbeat that is not normal.
  • If you take any drugs (prescription or OTC, natural products, vitamins) that must not be taken with eliglustat, like certain drugs that are used for seizures, fungal infections, or rifampin. There are many drugs that must not be taken with eliglustat.

This is not a list of all drugs or health problems that interact with eliglustat.

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 eliglustat 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 eliglustat?

  • Tell all of your health care providers that you take eliglustat. This includes your doctors, nurses, pharmacists, and dentists.
  • You may need to have your genes tested before starting eliglustat. Talk with your doctor.
  • Avoid grapefruit and grapefruit juice.
  • Tell your doctor if you are pregnant or plan on getting pregnant. You will need to talk about the benefits and risks of using eliglustat while you are pregnant.
  • Tell your doctor if you are breast-feeding. You will need to talk about any risks to your baby.

How is eliglustat best taken?

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

  • Swallow whole. Do not chew, break, open, or dissolve.
  • Take with or without food.
  • Take with a full glass of water.
  • Keep taking eliglustat as you have been told by your doctor or other health care provider, even if you feel well.

What do I do if I miss a dose?

  • Skip the missed dose and go back to your normal time.
  • Do not take 2 doses at the same time or extra doses.

What are the side effects of eliglustat 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.
  • A fast heartbeat or passing out.
  • A heartbeat that does not feel normal.
  • Dizziness.
  • Very bad headache.

What are some other side effects of eliglustat?

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:

  • Belly pain.
  • Diarrhea.
  • Upset stomach.
  • Gas.
  • Back pain.
  • Headache.
  • Feeling tired or weak.
  • Joint pain.
  • Mouth pain.
  • Throat pain.
  • Pain in arms or legs.
  • Cough.
  • Constipation.

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 eliglustat?

  • Store at room temperature.
  • Store in a dry place. Do not store in a bathroom.
  • Keep all drugs in a safe place. Keep all drugs out of the reach of children and pets.
  • Throw away unused or expired drugs. Do not flush down a toilet or pour down a drain unless you are told to do so. Check with your pharmacist if you have questions about the best way to throw out drugs. There may be drug take-back programs in your area.




  • NDC 58468-0220-1
  • Cerdelga®
    (eliglustat) capsules
  • 84 mg*
  • *Each capsule contains 84 mg of eliglustat which is
    equivalent to 100 mg of eliglustat tartrate
  • Dispense the enclosed Medication Guide to each patient
  • Four cartons of 14 capsules each
  • Rx Only


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