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Last Updated: January 9, 2025

CLINICAL TRIALS PROFILE FOR ELIGLUSTAT TARTRATE


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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.
>Trial ID >Title >Status >Phase >Start Date >Summary

Clinical Trial Conditions for eliglustat tartrate

Condition Name

Condition Name for eliglustat tartrate
Intervention Trials
Gaucher Disease, Type 1 3
Gaucher Disease 3
Gaucher Disease, Non-Neuronopathic Form 1
Glucocerebrosidase Deficiency Disease 1
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Condition MeSH

Condition MeSH for eliglustat tartrate
Intervention Trials
Gaucher Disease 6
Renal Insufficiency 1
Lipidoses 1
Deficiency Diseases 1
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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
Netherlands 2
Italy 2
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Trials by US State

Trials by US State for eliglustat tartrate
Location Trials
New York 4
Florida 3
Virginia 3
Georgia 3
Connecticut 3
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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
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Clinical Trial Status

Clinical Trial Status for eliglustat tartrate
Clinical Trial Phase Trials
Completed 6
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Clinical Trial Sponsors for eliglustat tartrate

Sponsor Name

Sponsor Name for eliglustat tartrate
Sponsor Trials
Genzyme, a Sanofi Company 5
Sanofi 1
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Sponsor Type

Sponsor Type for eliglustat tartrate
Sponsor Trials
Industry 6
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Eliglustat tartrate Market Analysis and Financial Projection

Eliglustat Tartrate: Clinical Trials, Market Analysis, and Projections

Introduction to Eliglustat Tartrate

Eliglustat tartrate, marketed under the brand name Cerdelga, is a novel oral substrate reduction therapy used in the treatment of Gaucher disease type 1 (GD1), a genetic disorder characterized by the accumulation of glucocerebroside in certain organs.

Clinical Trials Overview

ENCORE Trial

The ENCORE trial, a phase 3 study, was pivotal in establishing the efficacy and safety of eliglustat tartrate. This trial involved patients with GD1 who had already achieved therapeutic goals with enzyme replacement therapy (ERT). The study demonstrated that eliglustat was noninferior to imiglucerase in maintaining stable platelet counts, hemoglobin concentrations, and spleen and liver volumes over a 1-year period. In the long-term extension phase, all patients received eliglustat, and data showed that mean hemoglobin concentration, platelet count, and spleen and liver volumes remained stable for up to 4 years. Bone mineral density also remained stable and within the healthy reference range throughout the treatment period[1].

ENGAGE Trial

The ENGAGE trial was another phase 3 study, conducted as a randomized, double-blind, placebo-controlled trial. This study involved treatment-naive patients with GD1 and showed significant improvements in spleen volume, hemoglobin levels, liver volume, and platelet count after 9 months of eliglustat treatment compared to placebo. After the primary analysis period, patients entered an open-label, long-term treatment phase with eliglustat[4].

EDGE Trial

The EDGE trial, a phase 3 open-label study, provided additional safety data from the eliglustat lead-in period. This study involved treatment-naive patients and supported the pharmacokinetic and pharmacodynamic profiles of eliglustat, further reinforcing its safety and efficacy[3].

Key Clinical Findings

  • Stability of Clinical Parameters: Eliglustat maintained stable mean hemoglobin concentration, platelet count, and spleen and liver volumes for up to 4 years in the ENCORE trial[1].
  • Bone Health: Bone mineral density z scores remained stable and within the healthy reference range throughout the treatment period[1].
  • Safety Profile: Eliglustat was well tolerated over 4 years, with only 2.5% of patients withdrawing due to adverse events related to the study drug[1].
  • Efficacy in Treatment-Naive Patients: The ENGAGE trial demonstrated significant improvements in clinical manifestations such as spleen volume, hemoglobin levels, liver volume, and platelet count in treatment-naive patients[4].

Market Analysis

Market Size and Growth

The global eliglustat tartrate market is expected to grow at a compound annual growth rate (CAGR) of over 9% during the forecast period from 2022 to 2031. This growth is driven by an increase in the number of diagnoses of Gaucher disease, improved access to healthcare services, and advancements in pharmacological research[5].

Market Segmentation

The market is segmented based on product type, application, and geography. The product types include eliglustat tartrate with different purity levels (less than 98%, 98%-99%, and more than 99%). Applications primarily cover research and pharmaceutical uses. Geographically, the market is dominated by North America and Europe, with emerging markets in the Asia-Pacific region showing promising potential[2][5].

Major Market Players

Key players in the eliglustat tartrate market include Biophore, Hangzhou Utanpharma Biology, Tecoland, Raks Pharma, Symbio Generics, Cangzhou Enke Pharma Tech, Clearsynth, and Dr. Reddy’s Laboratories. These companies are investing heavily in research and development to enhance the performance and application range of eliglustat tartrate[2][5].

Market Projections

Regional Growth

North America and Europe are expected to continue dominating the market due to advanced healthcare infrastructure and high awareness of rare diseases. The Asia-Pacific region, however, is anticipated to show significant growth due to rising healthcare investments and increasing awareness of Gaucher disease[5].

Technological Advancements

Technological innovations and advancements in biotechnology are expected to further optimize the performance of eliglustat tartrate, enabling it to acquire a wider range of applications in the downstream market. The integration of digital health solutions will also play a critical role in monitoring treatment efficacy and enhancing patient compliance[2].

Personalized Medicine

There is a rising emphasis on personalized medicine, with a focus on developing dual-targeting therapies that offer better treatment outcomes for patients. This trend is expected to drive market growth and innovation in the coming years[5].

Future Outlook

Increasing Awareness and Diagnoses

The growing awareness of Gaucher disease and the increasing number of diagnoses are expected to drive the demand for eliglustat tartrate. Improved access to healthcare services and advancements in pharmacological research will further contribute to market expansion[5].

Strategic Partnerships

Strategic partnerships among pharmaceutical companies are likely to play a pivotal role in market expansion. Collaborative innovations and investments in rare disease therapies will continue to fuel the growth of the eliglustat tartrate market[5].

Key Takeaways

  • Eliglustat tartrate has demonstrated long-term clinical stability and efficacy in patients with Gaucher disease type 1.
  • The global market for eliglustat tartrate is projected to grow significantly, driven by increasing diagnoses and advancements in healthcare.
  • Technological innovations and personalized medicine approaches are expected to enhance the market's growth potential.
  • Major market players are investing heavily in research and development to expand the applications of eliglustat tartrate.

FAQs

What is the primary use of eliglustat tartrate?

Eliglustat tartrate is primarily used in the treatment of Gaucher disease type 1, a genetic disorder characterized by the accumulation of glucocerebroside in certain organs.

What were the key findings of the ENCORE trial?

The ENCORE trial showed that eliglustat tartrate maintained stable mean hemoglobin concentration, platelet count, and spleen and liver volumes for up to 4 years, and it was noninferior to imiglucerase in maintaining these parameters over a 1-year period[1].

What is the projected growth rate of the eliglustat tartrate market?

The global eliglustat tartrate market is expected to grow at a CAGR of over 9% during the forecast period from 2022 to 2031[5].

Which regions dominate the eliglustat tartrate market?

North America and Europe currently dominate the market, with emerging markets in the Asia-Pacific region showing promising potential[5].

What are the major drivers of the eliglustat tartrate market growth?

The growth is driven by an increase in the number of diagnoses of Gaucher disease, improved access to healthcare services, and advancements in pharmacological research[5].

Sources

  1. Eliglustat maintains long-term clinical stability in patients with Gaucher disease type 1. Blood, 129(17), 2375-2386.
  2. Global Eliglustat Tartrate Market Research Report 2022. MarketResearch.com.
  3. Extract from the Clinical Evaluation Report for Eliglustat (as tartrate). Therapeutic Goods Administration.
  4. Effect of Oral Eliglustat on Splenomegaly in Patients With Gaucher Disease Type 1. JAMA, 313(7), 695-703.
  5. Eliglustat Tartrate Reagent Market Size, Share & Trends Analysis Report. Reliable Business Insights.

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