Substance / Medication

Eliglustat

Overview

Active Ingredient
eliglustat
RxNorm CUI
1547220

Indications

Dosage and Administration (2.1) [see] CERDELGA is indicated for the long-term treatment of adult patients with Gaucher disease type 1 (GD1) who are CYP2D6 extensive metabolizers (EMs), intermediate metabolizers (IMs), or poor metabolizers (PMs) as detected by an FDA-cleared test.

Labeler: Genzyme CorporationUpdated: 2024-03-03T00:00:00.000ZFull label on DailyMed

Contraindications

When this intervention should not be used

CERDELGA is contraindicated in the following patients based on CYP2D6 metabolizer status due to the risk of cardiac arrhythmias from prolongation of the PR, QTc, and/or QRS cardiac intervals.

Indications & Related Conditions

Conditions associated via SNOMED clinical relationships

Administration & Protocol

Dosing, route, and treatment protocol

Detailed dosage and administration information is available in the full FDA drug label.

View full prescribing information on DailyMed

Monitoring & Follow-Up

Biomarkers relevant to this intervention via related conditions

No monitoring biomarkers have been mapped yet. Biomarker-intervention linkages are derived through related conditions and will expand as the knowledge graph grows.

Research Evidence

Published studies and systematic reviews

Sort:
Clinical outcomes after 4.5 years of eliglustat therapy for Gaucher disease type 1: Phase 3 ENGAGE trial final results.
Mistry Pramod K, Lukina Elena, Ben Turkia Hadhami et al. · Am J Hematol · 2021
PMID: 34161616TrialFull text (PMC)
Impact of hepatic and renal impairment on the pharmacokinetics and tolerability of eliglustat therapy for Gaucher disease type 1.
Li Jing, Chen Jun, Kanamaluru Vanaja et al. · Mol Genet Metab · 2020
PMID: 31924461Trial
Outcomes after 8 years of eliglustat therapy for Gaucher disease type 1: Final results from the Phase 2 trial.
Lukina Elena, Watman Nora, Dragosky Marta et al. · Am J Hematol · 2019
PMID: 30264864TrialFull text (PMC)
Long-term adverse event profile from four completed trials of oral eliglustat in adults with Gaucher disease type 1.
Peterschmitt M Judith, Freisens Selena, Underhill Lisa H et al. · Orphanet J Rare Dis · 2019
PMID: 31174576TrialFull text (PMC)
Once- versus twice-daily dosing of eliglustat in adults with Gaucher disease type 1: The Phase 3, randomized, double-blind EDGE trial.
Charrow Joel, Fraga Cristina, Gu Xuefan et al. · Mol Genet Metab · 2018
PMID: 29358012Trial
Eliglustat maintains long-term clinical stability in patients with Gaucher disease type 1 stabilized on enzyme therapy.
Cox Timothy M, Drelichman Guillermo, Cravo Renata et al. · Blood · 2017
PMID: 28167660TrialFull text (PMC)

Research data from MEDLINE/PubMed

Benefits & Expected Outcomes

Benefits, expected outcomes, efficacy data, and NNT (Number Needed to Treat) are pending physician authorship and evidence review.

Risks & Side Effects

Adverse reaction and safety data for this drug is sourced from the FDA-approved label.

View adverse reactions & drug interactions on DailyMed

Related Symptoms

Symptoms associated with conditions this intervention addresses

No related symptoms have been mapped yet. Symptom linkages are derived through associated conditions.

Alternatives & Comparisons

Alternative treatments, comparison data, and clinical decision support are pending physician authorship.

Medical Disclaimer

This information is for educational purposes only and does not constitute medical advice, diagnosis, or treatment. Always consult with a qualified healthcare provider regarding any medical condition or treatment plan.

Do not start, stop, or change any treatment without consulting your healthcare provider.

Quick Facts

Type
Substance / Medication
Fully Specified Name
Eliglustat (substance)
SNOMED CT
714006001
UMLS CUI
C3272698
RxNorm CUI
1547220
Labeler
Genzyme Corporation

Clinical Data

This intervention maps to 3 entities in the Ltrl knowledge graph.

3
Conditions
0
Biomarkers
0
Specialists
0
Symptoms

Data is sourced from SNOMED CT, UMLS, and the Ltrl clinical knowledge graph. Content sections marked as pending require physician authorship. Consult a healthcare provider before starting any treatment.