Medically reviewed by Drugs.com. Last updated on Apr 12, 2022.
Commonly used brand name(s)
In the U.S.
Available Dosage Forms:
Therapeutic Class: Endocrine-Metabolic Agent
Pharmacologic Class: Glucosylceramide Synthase Inhibitor
Uses for eliglustat
Eliglustat is used for the long-term treatment of type 1 Gaucher disease. However, it is only used in certain people with type 1 Gaucher disease. Your doctor will perform a test to make sure that eliglustat is right for you.
Type 1 Gaucher disease is a disease you get from both your parents. People with type 1 Gaucher disease lack an enzyme (naturally occurring substance in your body) that breaks down a chemical in your body called glucosylceramide. Too much glucosylceramide causes liver and spleen enlargement, changes in the blood, and bone disease. Eliglustat works by stopping the body from making glucosylceramide.
Eliglustat is available only with your doctor's prescription.
Before using eliglustat
In deciding to use a medicine, the risks of taking the medicine must be weighed against the good it will do. This is a decision you and your doctor will make. For eliglustat, the following should be considered:
Tell your doctor if you have ever had any unusual or allergic reaction to eliglustat or any other medicines. Also tell your health care professional if you have any other types of allergies, such as to foods, dyes, preservatives, or animals. For non-prescription products, read the label or package ingredients carefully.
Appropriate studies have not been performed on the relationship of age to the effects of eliglustat in the pediatric population. Safety and efficacy have not been established.
Although appropriate studies on the relationship of age to the effects of eliglustat have not been performed in the geriatric population, no geriatric-specific problems have been documented to date.
There are no adequate studies in women for determining infant risk when using this medication during breastfeeding. Weigh the potential benefits against the potential risks before taking this medication while breastfeeding.
Interactions with medicines
Although certain medicines should not be used together at all, in other cases two different medicines may be used together even if an interaction might occur. In these cases, your doctor may want to change the dose, or other precautions may be necessary. When you are taking eliglustat, it is especially important that your healthcare professional know if you are taking any of the medicines listed below. The following interactions have been selected on the basis of their potential significance and are not necessarily all-inclusive.
Using eliglustat with any of the following medicines is not recommended. Your doctor may decide not to treat you with this medication or change some of the other medicines you take.
- Abiraterone Acetate
Using eliglustat with any of the following medicines is usually not recommended, but may be required in some cases. If both medicines are prescribed together, your doctor may change the dose or how often you use one or both of the medicines.
- St John's Wort
Interactions with food/tobacco/alcohol
Certain medicines should not be used at or around the time of eating food or eating certain types of food since interactions may occur. Using alcohol or tobacco with certain medicines may also cause interactions to occur. The following interactions have been selected on the basis of their potential significance and are not necessarily all-inclusive.
Using eliglustat with any of the following is usually not recommended, but may be unavoidable in some cases. If used together, your doctor may change the dose or how often you use eliglustat, or give you special instructions about the use of food, alcohol, or tobacco.
- Grapefruit Juice
Other medical problems
The presence of other medical problems may affect the use of eliglustat. Make sure you tell your doctor if you have any other medical problems, especially:
- Arrhythmia (heart rhythm problem)—Use with caution. May make this condition worse.
- Congestive heart failure, or history of or
- Heart attack, recent or
- Heart block or
- Heart rhythm problems (eg, long QT syndrome, slow heartbeat, ventricular arrhythmia)—Use is not recommended in patients with these conditions. May increase risk for arrhythmia.
- Kidney disease, moderate to severe or
- Liver disease (including cirrhosis)—Use is not recommended in patients with these conditions.
Proper use of eliglustat
Take eliglustat exactly as directed by your doctor. Do not take more of it, do not take it more often, and do not take it for a longer time than your doctor ordered. To do so may increase the chance of side effects.
Eliglustat comes with a Medication Guide. Read and follow these instructions carefully. Ask your doctor if you have any questions.
Swallow the capsule whole with water. Do not crush, break, chew, or open it. It may be taken with or without food.
If you are also using imiglucerase, velaglucerase alfa, or taliglucerase alfa, take eliglustat 24 hours after taking the last dose of these medicines.
Avoid eating grapefruit or drinking grapefruit juice while you are using eliglustat.
The dose of eliglustat will be different for different patients. Follow your doctor's orders or the directions on the label. The following information includes only the average doses of eliglustat. If your dose is different, do not change it unless your doctor tells you to do so.
The amount of medicine that you take depends on the strength of the medicine. Also, the number of doses you take each day, the time allowed between doses, and the length of time you take the medicine depend on the medical problem for which you are using the medicine.
- For oral dosage form (capsules):
- For type 1 Gaucher disease:
- Adults—Your dose will be based on the results of the test given by your doctor.
- Children—Use and dose must be determined by your doctor.
- For type 1 Gaucher disease:
If you miss a dose of eliglustat, skip the missed dose and go back to your regular dosing schedule. Do not double doses.
Store the medicine in a closed container at room temperature, away from heat, moisture, and direct light. Keep from freezing.
Keep out of the reach of children.
Do not keep outdated medicine or medicine no longer needed.
Ask your healthcare professional how you should dispose of any medicine you do not use.
Precautions while using eliglustat
It is very important that your doctor check your progress at regular visits to make sure eliglustat is working properly. Blood tests may be needed to check for unwanted effects.
Contact your doctor right away if you have any changes to your heart rhythm. You might feel dizzy or faint, or you might have a fast, pounding, or uneven heartbeat. Make sure your doctor knows if you or anyone in your family has ever had a heart rhythm problem such as QT prolongation.
Do not take other medicines unless they have been discussed with your doctor. This includes prescription or nonprescription (over-the-counter [OTC]) medicines and herbal (eg, St. John's wort) or vitamin supplements.
Eliglustat side effects
Along with its needed effects, a medicine may cause some unwanted effects. Although not all of these side effects may occur, if they do occur they may need medical attention.
Check with your doctor immediately if any of the following side effects occur:
- Fast, irregular, pounding, or racing heartbeat or pulse
Incidence not known
Get emergency help immediately if any of the following symptoms of overdose occur:
Symptoms of overdose
- Blurred vision
- chest pain or discomfort
- dizziness, faintness, or lightheadedness when getting up suddenly from a lying or sitting position
- unusual tiredness or weakness
Some side effects may occur that usually do not need medical attention. These side effects may go away during treatment as your body adjusts to the medicine. Also, your health care professional may be able to tell you about ways to prevent or reduce some of these side effects. Check with your health care professional if any of the following side effects continue or are bothersome or if you have any questions about them:
- Back pain
- difficulty with moving
- excess air or gas in the stomach or bowels
- full feeling
- lack or loss of strength
- muscle pain or stiffness
- pain in the arms or legs
- pain in the joints
- passing gas
- sore throat
- stomach discomfort, upset, or pain
- upper abdominal or stomach pain
- Difficulty having a bowel movement
Other side effects not listed may also occur in some patients. If you notice any other effects, check with your healthcare professional.
Call your doctor for medical advice about side effects. You may report side effects to the FDA at 1-800-FDA-1088.
More about eliglustat
- Side effects
- Drug interactions
- Dosage information
- During pregnancy or Breastfeeding
- En español
- Drug class: miscellaneous metabolic agents
Related treatment guides
Always consult your healthcare provider to ensure the information displayed on this page applies to your personal circumstances.