NDAORALCAPSULEPriority Review
Approved
Aug 2014
Lifecycle
Peak
Competitive Pressure
0/100
Clinical Trials
16
Data completeness
8/8 — Comprehensive
Priority Review
CERDELGA (eliglustat) by Sanofi is glucosylceramide synthase inhibitors [moa]. Approved for glucosylceramide synthase inhibitor [epc]. First approved in 2014.
Drug data last refreshed 13h ago · AI intelligence enriched 1w ago
AI summarySynthesized from FDA + DailyMed + ClinicalTrials.gov data
CERDELGA (eliglustat) is an oral small-molecule glucosylceramide synthase inhibitor approved in 2014 for Gaucher disease, a rare lysosomal storage disorder. It works by reducing the synthesis of glucosylceramide, the substrate that accumulates in Gaucher disease patients. The drug represents a substrate reduction therapy approach in the ultra-rare disease space.
$0.038BPart D
Peak12.2 years to LOEProduct is in peak commercial phase with modest Part D spending (~$38M) and small patient population (1,242 claims in 2023), indicating a niche, specialized commercial team.
Mechanism of Action
Glucosylceramide Synthase Inhibitors
Pharmacologic Class:
Glucosylceramide Synthase Inhibitor
Indications
Indication data is being enriched from DailyMed and FDA labeling. Check back soon for approved therapeutic uses.
Clinical Trials (16)
Efficacy and Safety of Eliglustat in Chinese Pediatric Patients With Gaucher Disease Type 1 and Type 3
Started Aug 2024
5 enrolled
Gaucher Disease
Eliglustat on Gaucher Disease Type IIIB
Started Apr 2018
4 enrolled
Gaucher Disease, Type III
Safety and Efficacy of Eliglustat With or Without Imiglucerase in Pediatric Patients With Gaucher Disease (GD) Type 1 and Type 3
Started Apr 2018
57 enrolled
Gaucher's Disease Type IGaucher's Disease Type III
A Study to Evaluate Pharmacokinetic Parameters of Eliglustat in Healthy Volunteers Who Are CYP2D6 Extensive or Poor Metabolizers
Started Jan 2018
18 enrolled
Gaucher's Disease
Phase 3b Study to Evaluate Skeletal Response to Eliglustat in Adult Patients Who Completed Phase 2 or Phase 3 Studies
Started Oct 2015
31 enrolled
Gaucher Disease
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Data Sources
- Drug label: DailyMed / FDA
- Clinical trials: ClinicalTrials.gov
- Patent data: FDA Orange Book
- Spending data: CMS Medicare
The information on this page is for informational purposes only and should not be used as a substitute for professional medical advice. Drug information is sourced from FDA, DailyMed, and other government databases. Adverse event data from FAERS does not establish causation. Always consult a healthcare professional for medical decisions.