eliglustat tartrate at a glance
Medicine class
Glucosylceramide synthase inhibitor; substrate-reduction therapy.
Main use
Gaucher disease type 1 in eligible adults and selected children.
Before treatment
CYP2D6 genotyping is required to establish suitability.
Administration
Swallow the capsule whole with water, with or without food; avoid grapefruit.
What is eliglustat tartrate used for?
Licensed uses represented by the selected product are:
Adults with Gaucher disease type 1 who are CYP2D6 poor, intermediate or extensive metabolisers.
Children aged 6 years or older who weigh at least 15 kg, have Gaucher disease type 1 controlled on enzyme-replacement therapy, and are CYP2D6 poor, intermediate or extensive metabolisers.
How does eliglustat tartrate work?
In Gaucher disease type 1, glucosylceramide is not broken down effectively and accumulates in organs and bone. Eliglustat inhibits glucosylceramide synthase, reducing formation of this material.
eliglustat tartrate preparations and strengths
Capsule
Route: Oral
Strengths: 84 mg
How to take eliglustat tartrate
Follow the specialist's instructions and check that the correct capsule has been supplied.
Swallow capsules whole, preferably with water.
Do not open, crush, chew or dissolve them.
They may be taken with or without food.
Do not mix the contents into food or drinks; this has not been studied.
Avoid grapefruit and grapefruit juice.
eliglustat tartrate side effects
Common or expected effects
- Indigestion or heartburn, abdominal discomfort, nausea, vomiting, diarrhoea, constipation, bloating or wind
- Headache, dizziness or altered taste
- Palpitations
- Cough or throat irritation
- Dry skin or hives
- Joint, limb or back pain
- Tiredness
Get urgent medical advice
- Palpitations with severe dizziness or fainting require urgent assessment.
- Marked dizziness, loss of balance, light-headedness or a slow heartbeat after taking too much requires urgent medical advice.
eliglustat tartrate in pregnancy
Human pregnancy information is limited, so eliglustat should generally be avoided as a precaution. Anyone who is pregnant, planning pregnancy or thinks they may be pregnant should discuss treatment promptly with their specialist.
eliglustat tartrate while breastfeeding
It is unknown whether eliglustat metabolites enter human milk, and risk to a breastfed child cannot be excluded. The specialist and parent should decide whether to stop breastfeeding or avoid eliglustat after considering the benefits of each.
eliglustat tartrate interactions
Interaction risk partly depends on CYP2D6 metaboliser status. Check all prescription, non-prescription and herbal products before starting or changing treatment.
Strong or moderate CYP2D6 inhibitors, such as paroxetine, fluoxetine, quinidine or terbinafine
These can substantially increase eliglustat exposure; some combinations require avoidance or specialist modification.
Strong or moderate CYP3A inhibitors, such as clarithromycin, itraconazole, ritonavir, fluconazole or verapamil
These can increase eliglustat exposure and may be contraindicated or unsuitable depending on CYP2D6 status and liver function.
Combined CYP2D6 and CYP3A inhibitors
Certain combinations are contraindicated because eliglustat concentrations can rise substantially.
Common questions about eliglustat tartrate
Answers are fully visible for fast scanning and source review.
Why is a CYP2D6 test needed?
CYP2D6 affects how quickly eliglustat is cleared. Testing identifies whether treatment is suitable and helps the specialist assess interaction and toxicity risks.
Can every person with Gaucher disease type 1 take it?
No. Suitability depends on CYP2D6 status, age and weight where relevant, previous treatment, liver and kidney function, heart conditions and interacting medicines.
Can children take it?
Sources
These sources were used to prepare and review this medicine page.
Prepared and reviewed by the iatroX editorial team.