Eliglustat

How it works

It inhibits glucosylceramide synthase, reducing the production of glucosylceramide so that less substrate accumulates in cells where the deficient enzyme cannot break it down.

Prescribing in practice

• It is metabolised by CYP2D6 and CYP3A, so dosing depends on the patient's CYP2D6 metaboliser status and interacting drugs, and combinations that markedly raise its levels can prolong the QT interval and must be avoided or adjusted.
• Suitability and dosing are guided by CYP2D6 genotype, and it is not recommended in CYP2D6 ultra-rapid or indeterminate metabolisers in whom adequate levels cannot be assured.
• Treatment should be initiated and supervised by a clinician experienced in the management of Gaucher disease, with review of concomitant medicines for interactions.

Monitoring

Monitor disease response and review for drug interactions and cardiac (QT) risk, taking the patient's CYP2D6 metaboliser status into account.

Counselling the patient

• Take it as directed and tell your healthcare team about all other medicines, including over-the-counter products.
• Avoid grapefruit juice, which can raise drug levels.
• Do not stop or change the dose without specialist advice.

Evidence & guidelines

Its use is supported by randomised controlled trials in type 1 Gaucher disease, including in previously untreated and stabilised patients.