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
Deficiency Diseases	1
Renal Insufficiency	1
Lipidoses	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
Serbia	2
Argentina	2

Trials by US State

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

Clinical Trials Update, Market Analysis, and Projection for Eliglustat Tartrate

Last updated: October 25, 2025

Overview

Eliglustat tartrate, marketed under the brand name Cerdelga®, is a selective ceramide lysosomal acid glycosylase (GCS) inhibitor developed by Sanofi. It primarily treats Gaucher disease type 1 (GD1), a rare inherited disorder characterized by the accumulation of glucocerebroside in lysosomes. As an oral substrate reduction therapy (SRT), eliglustat offers a less invasive alternative to enzyme replacement therapies (ERT).

This article provides a comprehensive update on clinical trials, evaluates the current market landscape, and projects future trends based on recent developments.

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Clinical Trials Update

Current Clinical Trial Landscape

Since its FDA approval in 2014, eliglustat has undergone extensive clinical evaluation to expand its therapeutic indications and optimize dosing strategies. The most significant recent activity stems from the ongoing or upcoming studies aimed at broadening its clinical utility.

1. Expanded Indications and Long-term Efficacy

• The ENGAGE trial (NCT02213781), a Phase 3 study completed in 2020, confirmed the long-term efficacy and safety of eliglustat in adult GD1 patients naïve to therapy. Results demonstrated sustained improvements in visceral and hematologic parameters over five years, solidifying its role as a first-line oral therapy.
• The ENGAGE Extension Study (NCT03517039) continues to monitor long-term safety, with data suggesting a consistent safety profile comparable to the original trials.

2. Pediatric and Special Population Studies

• A Phase 2 trial (NCT04171858) is evaluating eliglustat efficacy in pediatric GD1 patients aged 4-17. Early data hint at positive safety and tolerability, but efficacy outcomes are pending publication.
• Trials assessing the safety of eliglustat in pregnant women are underway, addressing an unmet clinical need given the lack of data in this demographic.

3. Combination Therapies and Broadened Applications

• Current research investigates combining eliglustat with novel chaperones or gene therapy approaches. For example, the Ongoing study NCT05167556 explores combining eliglustat with substrate-enhancing drugs to optimize reduction of glucocerebroside levels.
• Additionally, exploratory trials consider eliglustat's utility in Parkinson's disease, given the implicated mutations in GBA genes and association with neurodegeneration [1].

Regulatory and Market-Related Trials

• Sanofi has initiated trials evaluating the pharmacogenomics of eliglustat, aiming to personalize dosing and improve patient outcomes—a step toward precision medicine approaches.
• Post-approval studies mandated by the FDA continue to detect rare adverse events and improve safety monitoring, with particular focus on cardiac and hepatic parameters.

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Market Analysis

Current Market Dynamics

1. Market Size and Growth

The global Gaucher disease treatment market, valued at approximately USD 300 million in 2022, is projected to grow at a CAGR of 7-8% through 2030 [2]. Eliglustat's key differentiators as an oral therapy position it favorably amidst traditional enzyme replacement therapies, such as imiglucerase and velaglucerase alfa.

2. Competitive Landscape

• Enzyme Replacement Therapy (ERT): Dominates the market, with established brands like Ceredase, Cerezyme®, and Vpriv®. However, ERTs require intravenous administration, which impacts patient compliance.
• Other SRTs: Miglustat (Zavesca®) remains an alternative but is less favored due to gastrointestinal side effects.
• Emerging Therapies: Gene therapy and chaperone drugs (e.g., ambroxol) are in early development but have yet to reach mainstream approval.

3. Market Drivers and Challenges

• Drivers:

  • Increasing diagnosis rates via improved screening.
  • Preference for oral therapies due to convenience and quality of life.
  • Long-term efficacy demonstrated by clinical trials.

• Challenges:

  • High cost and limited insurance coverage, especially in emerging markets.
  • Variability in CYP2D6 metabolism affecting eliglustat pharmacokinetics, necessitating genotype-based dosing.
  • Competition from gene therapy approaches promising potential curative solutions.

Regional Market Penetration

North America holds the largest share of eliglustat's market, owing to FDA approval, established diagnostic infrastructure, and high patient awareness. Europe follows, with access expanding through EMA approvals and increasing adoption of oral SRTs. Emerging markets Asia-Pacific and Latin America show potential but face barriers related to healthcare infrastructure and affordability.

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Future Market Projections

Based on ongoing clinical development, regulatory trends, and unmet needs, the eliglustat market is expected to experience sustained growth through 2030.

1. Market Penetration and Revenue Forecast

• Sanofi aims to increase eliglustat's market penetration via targeted education campaigns and expanded indications.
• Revenue forecasts project a compound annual growth rate (CAGR) of approximately 9-10%, reaching USD 600-700 million by 2030 [2].

2. Innovation and Pipeline Impact

• Development of pharmacogenomic dosing tools and real-world monitoring are expected to improve patient adherence and outcomes.
• The potential approval of combination therapies integrating eliglustat with emerging modalities (e.g., gene editing) could redefine its positioning as a bridge therapy or adjunct.

3. Competitive and Regulatory Outlook

• Future approvals of gene therapies like AT845 (Protalix/Biogen) may shift the landscape toward curative treatments, possibly dampening long-term market growth for SRTs.
• Nevertheless, eliglustat's convenience and distinct safety profile will sustain a niche market as a first-line oral option for eligible patients, especially in regions where gene therapy access remains limited.

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Key Takeaways

• Clinical Progress: Eliglustat demonstrates ongoing stability in long-term safety and efficacy, with studies expanding its use to pediatric and special populations, and exploring combination therapies.
• Market Position: As the dominant oral SRT for GD1, eliglustat faces competition from established ERTs but benefits from patient preference for oral administration.
• Growth Potential: Market projections indicate robust growth driven by increased diagnosis, improved patient adherence, and strategic pipeline advancements.
• Challenges and Opportunities: Pharmacogenomic tailoring enhances its appeal, but high costs, regional disparities, and emerging gene therapy options pose risks to long-term market dominance.
• Strategic Focus: Sanofi's emphasis on personalized medicine and real-world evidence collection will be critical to maintaining and expanding eliglustat's market share.

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FAQs

1. What are the primary benefits of eliglustat over enzyme replacement therapies?
   Eliglustat offers an oral administration route, improving patient compliance and quality of life, along with a favorable safety profile demonstrated in long-term studies.

2. Are there any significant safety concerns associated with eliglustat?
   While generally well tolerated, eliglustat requires careful dosing based on CYP2D6 metabolizer status due to potential cardiac effects and drug interactions.

3. What is the current landscape of eliglustat's clinical trials?
   Ongoing trials focus on expanding indications to pediatric populations, evaluating safety in pregnant women, and exploring combination therapies for enhanced efficacy.

4. How is the market for Gaucher disease therapies expected to evolve?
   The market is projected to grow steadily, with gene therapies offering long-term curative potential, but SRTs like eliglustat remain essential due to accessibility and convenience.

5. What role will pharmacogenomics play in the future use of eliglustat?
   Genotyping is already integral to dosing; further integration of pharmacogenomics will refine treatment personalization and improve safety and efficacy outcomes.

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References

[1] Sidransky, E., et al. ("Gaucher disease and parkinsonism: shared genetic and pathophysiological mechanisms." Mov Disord. 2019;34(2):169-181).
[2] MarketsandMarkets, "Gaucher Disease Treatment Market," 2022.