CLINICAL TRIALS PROFILE FOR ELIGLUSTAT TARTRATE

All Clinical Trials for ELIGLUSTAT TARTRATE

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00358150 ↗	A Study of the Efficacy and Safety of Eliglustat Tartrate (Genz-112638) in Type 1 Gaucher Patients	Completed	Genzyme, a Sanofi Company	Phase 2	2006-06-01	Gaucher disease is a genetic disease that results in a deficiency of an enzyme acid beta-glucosidase, also known as glucocerebrosidase. This enzyme is needed to digest a substrate (lipid) called glucosylceramide and, to a lesser degree, glucosylsphingosine. In participants with Gaucher disease, the liver, spleen, bone marrow and brain show increases in lipid concentration, specifically in cells derived from the monocyte/macrophage system. Eliglustat tartrate (Genz-112638) is an oral drug that may regulate the Gaucher disease process by decreasing the synthesis of glucosylceramide. The primary objective of this study is to evaluate the efficacy, safety and pharmacokinetics (PK) of eliglustat tartrate, administered as an oral dose of either 50 milligram (mg) twice daily (BID) or 100 mg BID, to men and women with Gaucher disease Type 1 for 52 weeks.
NCT00891202 ↗	A Study of Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease (ENGAGE)	Completed	Genzyme, a Sanofi Company	Phase 3	2009-11-01	This Phase 3 study was designed to confirm the efficacy and safety of eliglustat tartrate (Genz-112638) in participants with Gaucher disease Type 1.
NCT00943111 ↗	A Study of Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease Who Have Reached Therapeutic Goals With Enzyme Replacement Therapy (ENCORE)	Completed	Genzyme, a Sanofi Company	Phase 3	2009-09-01	This Phase 3 study was designed to confirm the efficacy and safety of eliglustat tartrate (Genz-112638) in participants with Gaucher disease type 1 who had reached therapeutic goals with enzyme replacement therapy (ERT).
NCT01074944 ↗	A Study of Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease to Evaluate Once Daily Versus Twice Daily Dosing (EDGE)	Completed	Genzyme, a Sanofi Company	Phase 3	2010-06-01	The primary objective of this study was to evaluate the efficacy and safety of once daily (QD) versus twice daily (BID) dosing of eliglustat tartrate (Genz-112638) in participants with Gaucher disease type 1 who had demonstrated clinical stability on BID dosing of eliglustat tartrate (Genz-112638). The secondary objective was to evaluate the pharmacokinetics (PK) of Genz-99067 when eliglustat tartrate (Genz-112638) was administered QD and BID in participants with Gaucher disease type 1 who had demonstrated clinical stability on BID dosing of eliglustat tartrate (Genz-112638).
NCT02536911 ↗	A Study of the Effects of Hepatic Impairment on the Pharmacokinetics and Tolerability of Eliglustat Tartrate	Completed	Genzyme, a Sanofi Company	Phase 1	2015-09-01	Primary Objective: To study the effect of mild and moderate hepatic impairment on the pharmacokinetics (PK) of eliglustat. Secondary Objective: To assess the tolerability of eliglustat tartrate given as a single dose in subjects with mild and moderate hepatic impairment in comparison with matched subjects with normal hepatic function.
NCT02536937 ↗	A Study of the Effects of Renal Impairment on the Pharmacokinetics and Tolerability of Eliglustat Tartrate	Completed	Sanofi	Phase 1	2015-09-01	Primary Objective: To study the effect of mild, moderate, and severe renal impairment on the pharmacokinetics (PK) of eliglustat. Secondary Objective: To assess the tolerability of eliglustat tartrate given as a single dose in subjects with mild, moderate, and severe renal impairment in comparison with matched subjects with normal renal function.

Clinical Trial Conditions for ELIGLUSTAT TARTRATE

Condition Name

Condition Name for ELIGLUSTAT TARTRATE
Intervention	Trials
Gaucher Disease	3
Gaucher Disease, Type 1	3
Cerebroside Lipidosis Syndrome	1
Gaucher Disease, Non-Neuronopathic Form	1

Condition MeSH

Condition MeSH for ELIGLUSTAT TARTRATE
Intervention	Trials
Gaucher Disease	6
Lipidoses	1
Deficiency Diseases	1
Renal Insufficiency	1

Clinical Trial Locations for ELIGLUSTAT TARTRATE

Trials by Country

Trials by Country for ELIGLUSTAT TARTRATE
Location	Trials
United States	35
Russian Federation	4
Canada	3
United Kingdom	2
Israel	2

Trials by US State

Trials by US State for ELIGLUSTAT TARTRATE
Location	Trials
New York	4
Florida	3
Virginia	3
Georgia	3
Connecticut	3

Clinical Trial Progress for ELIGLUSTAT TARTRATE

Clinical Trial Phase

Clinical Trial Phase for ELIGLUSTAT TARTRATE
Clinical Trial Phase	Trials
Phase 3	3
Phase 2	1
Phase 1	2

Clinical Trial Status

Clinical Trial Status for ELIGLUSTAT TARTRATE
Clinical Trial Phase	Trials
Completed	6

Clinical Trial Sponsors for ELIGLUSTAT TARTRATE

Sponsor Name

Sponsor Name for ELIGLUSTAT TARTRATE
Sponsor	Trials
Genzyme, a Sanofi Company	5
Sanofi	1

Sponsor Type

Sponsor Type for ELIGLUSTAT TARTRATE
Sponsor	Trials
Industry	6

Eliglustat Tartrate: Clinical Trial Update, Market Analysis, and Projection

Last updated: April 27, 2026

What is eliglustat tartrate and what is its clinical status?

Eliglustat tartrate (Brand: Cerdelga) is an oral substrate reduction therapy for Gaucher disease type 1 (GD1). The drug’s current clinical footprint is dominated by labeling and real-world positioning rather than new Phase 3 pivots; subsequent activity centers on confirmation studies, long-term safety follow-up, and population-specific use tied to CYP2D6 metabolizer status.

Core clinical use

• Indication: Moderate-to-severe Gaucher disease type 1 in adults who are CYP2D6 metabolizer–matched (per label).
• Dosing logic: stratified by CYP2D6 phenotype and drug interaction profile.

Regulatory anchor points (label scope drivers)

• Dosing and eligibility depend on CYP2D6 metabolizer status, and the drug’s market access tracks prescriber comfort with genotype-based selection and payer policies around genetic testing.
• This structure tends to stabilize demand in established markets, but it narrows accessible patient segments versus therapies without genotype constraints.

(No additional trial-phase detail can be stated from the information provided in the prompt.)

What do recent clinical-trial updates translate to commercially?

Because the modern commercial life of eliglustat is label-led, “trial updates” typically affect market shape via:

• Long-term safety and persistence: supports reimbursement durability where payers evaluate chronic tolerability.
• Treatment adherence: oral, once- or twice-daily regimens generally improve persistence versus infusion regimens, which affects real-world throughput more than incremental efficacy curves.
• CYP2D6 testing workflows: any evidence that reduces testing friction (or clarifies edge cases) lowers administrative cost per start.

Commercially, that means the market is primarily influenced by:

1. Switching and retention from enzyme replacement therapy (ERT)
2. Coverage decisions conditioned on genotype testing and guideline fit
3. Competition for GD1 therapy slots across ERT, SRT, and newer pipeline entrants

How big is the GD1 eliglustat market and what does the competitive set look like?

Eliglustat competes in Gaucher disease type 1 against:

• Enzyme replacement therapies (ERT): imiglucerase, velaglucerase, taliglucerase
• Other substrate reduction: the class includes competing agents, where applicable by region and approvals
• New entrants in GD1 pipeline: typically small-molecule or next-generation SRT assets, where timeline and approval status drive sentiment

Market segmentation that drives eliglustat uptake

• Diagnosed and genotype-assessed GD1 adults
• Treatment-naïve vs switchers from ERT
• Geographic payer systems:
  • Countries with robust genetic testing infrastructure reduce friction for initiation.
  • Countries with restrictive prior authorization slow uptake and increase churn risk.

Commercial adoption mechanics

Eliglustat’s addressable uptake is constrained by:

• CYP2D6 metabolizer assignment requirements
• Drug-drug interactions that force exclusion or monitoring
• Payer preference for ERT in patients who fail genotype eligibility or where testing is not reimbursed

In markets where these constraints are operationalized smoothly, eliglustat usually benefits from:

• Lower administration burden (no infusion)
• Patient preference for oral dosing
• Higher persistence relative to ERT, which creates a compounding installed base

What are the key commercial indicators for projection?

Market projection for eliglustat should be built on four measurable drivers:

1) Diagnosed GD1 incidence and diagnosis rates

• Higher diagnosis rates expand the number of eligible adults entering treatment.
• More active newborn screening or earlier specialist referral drives the top-of-funnel.

2) Share of ERT switching to oral SRT

• Eliglustat captures a share of patients who:
  • find infusion burdensome
  • have stable disease control or tolerate switching
  • have coverage that makes switch cost-effective

3) Retention and discontinuation

• Oral therapies often show strong persistence, but discontinuations can rise if:
  • CYP2D6 testing creates eligibility resets
  • interaction restrictions complicate comedication
  • adverse events or adherence issues occur

4) Competitive dynamics and payer controls

• Payer controls can cap share growth even when clinical outcomes are favorable.
• Tendering or preferred formulary placement can swing the installed base.

What is the projection for eliglustat tartrate over the next 5 years?

A numeric revenue forecast requires region- and source-specific inputs (GD1 prevalence, diagnosed-treated population, testing uptake rates, and payer coverage). The prompt does not provide those inputs. Under the constraints, no complete and accurate numeric projection can be produced.

What business conclusions follow for R&D and investment decisions?

R&D

• The competitive zone for eliglustat’s “next phase” is less about proving efficacy and more about:
  • minimizing testing and interaction friction
  • expanding eligibility without compromising safety
  • sustaining long-term safety evidence in real-world populations

Investment

• The risk profile is driven by:
  • payer access tied to CYP2D6 workflows
  • competitive share shifts between SRT and ERT
  • geographic variability in reimbursement for genotype testing and for oral chronic therapy

Key Takeaways

• Eliglustat tartrate is label-led in GD1: its market behavior hinges on CYP2D6 metabolizer eligibility, drug interaction constraints, and payer authorization practices tied to genetic testing workflows.
• Clinical updates in this category typically reinforce long-term safety, persistence, and operational feasibility, which influence patient starts and retention more than incremental short-term efficacy.
• A complete 5-year numeric market projection cannot be stated from the information provided in the prompt; projection depends on diagnosed-treated population size, testing reimbursement, and payer coverage mechanics by region.
• The commercial moat is operational: oral administration plus durable disease control outcomes, counterbalanced by genotype-dependent restrictions and coverage constraints.

FAQs

1. What disease is eliglustat tartrate approved for?
   Gaucher disease type 1 (GD1) in adults with CYP2D6 metabolizer–matched eligibility per label.

2. Why does CYP2D6 matter for eliglustat?
   Label dosing and eligibility depend on CYP2D6 phenotype, and drug-drug interactions can affect safety and suitability.

3. Does eliglustat face competition from ERT?
   Yes. ERTs remain a major comparator for GD1 and can win patients based on coverage, testing availability, and prescriber workflow.

4. What drives eliglustat uptake in the real world?
   Patient identification, genotype testing processes, payer access, switch feasibility from ERT, and persistence on chronic oral therapy.

5. Can a precise 5-year revenue projection be produced from this prompt alone?
   No. A credible numeric forecast requires diagnosed-treated population and payer/testing adoption inputs by market.

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References

[1] FDA. Cerdelga (eliglustat) prescribing information. U.S. Food and Drug Administration.