Eliglustat

CERDELGA

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Pronunciation: el-i-GLOO-stat (sir-DEL-ga)

Eliglustat ballicule: receptor binding, kinetics and half-life diagram — Mechanism of action: Eliglustat ballicule

What it isGlucosylceramide synthase inhibitor; Gaucher disease type 1.

Why this oneOral substrate reduction rather than biweekly enzyme infusion.

What limits it⚑ CYP2D6 genotype determines dose, and poor metabolizers cannot use it at all. QT prolongation; many contraindicated interactions.

Eliglustat molecular structure (skeletal formula) — Molecular structure: Eliglustat

FDA-approved for

Gaucher disease type 1 (2014)

Principal riskCYP2D6 genotype determines dose; poor metabolizers cannot use it

Classes and tags

Clinical profile

Therapeutic safety burden2 / 4

Overdose danger1 / 4

Weight-gain liability0 / 4

Sedation liability0 / 4

QT / Torsades0 / 4

Direct muscarinic antagonism0 / 4

Embryofetal risk1 / 4

Legacy pregnancy categoryUnknown / historical label unavailable

Defining liabilityGenerally tolerated at therapeutic doses; clinically important risks are agent-specific and increase with interactions or organ impairment.

FDA label

Read the full label on DailyMed ↗

NO BOXED WARNINGS

FDA dosing	Population	Start	Target	Max
Gaucher disease type 1	CYP2D6 extensive or intermediate metabolizer	84 mg BID	84 mg BID	84 mg BID
Gaucher disease type 1	CYP2D6 poor metabolizer	84 mg QD	84 mg QD	84 mg QD

Renal EMs: avoid in end-stage renal disease; otherwise use per CYP2D6 metabolizer status

Hepatic Contraindicated in IMs/PMs with any hepatic impairment and in EMs with moderate-severe impairment (or mild impairment plus a CYP2D6 inhibitor); genotype-dependent

Dose frequency set by CYP2D6 metabolizer status and interacting CYP2D6/CYP3A inhibitors

Instructions

Select patients with an FDA-cleared CYP2D6 genotype test.
Swallow capsules whole; do not crush, dissolve, or open.
Avoid grapefruit or grapefruit juice.
Reduce to 84 mg once daily in EMs/IMs taking CYP2D6 or CYP3A inhibitors.

Available strengths

cap 84 mg

Contraindications

EMs taking a strong/moderate CYP2D6 inhibitor with a strong/moderate CYP3A inhibitor
EMs with moderate or severe hepatic impairment, or mild hepatic impairment plus a strong/moderate CYP2D6 inhibitor
IMs taking a strong/moderate CYP2D6 inhibitor with a strong/moderate CYP3A inhibitor, or a strong CYP3A inhibitor
IMs or PMs with any degree of hepatic impairment
PMs taking a strong CYP3A inhibitor

Cautions

ECG interval prolongation (PR, QTc, QRS) and potential for cardiac arrhythmias at elevated plasma concentrations
Avoid in pre-existing cardiac disease or long QT syndrome combined with Class IA/III antiarrhythmics

Adverse reactions

Fatigue
Headache
Nausea
Diarrhea
Back pain
Pain in extremities
Upper abdominal pain

Pregnancy Insufficient human data; developmental abnormalities in rats at ~6x human dose.

Lactation No human data; eliglustat present in rat milk.

Cost

Eliglustat (CERDELGA)

Strength		#/mo	generic $/mo	CostPlus
84 mg	cap	60 BID		not offered

NADAC is a voluntary CMS survey of what retail community pharmacies pay for the drugs they stock. Eliglustat is dispensed outside retail pharmacies, so it rarely appears on surveyed invoices.

Generic entry2021

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Self-check

Which genotype determines eliglustat dose?
CYP2D6.
How does eliglustat therapy differ from enzyme replacement?
Oral substrate reduction rather than biweekly enzyme infusion.