This “Glucosylceramide synthase inhibitors - Pipeline Insight, 2022” report provides comprehensive insights about 5+ companies and 5+ pipeline drugs in Glucosylceramide synthase inhibitors pipeline landscape. It covers the pipeline drug profiles, including clinical and nonclinical stage products. It also covers the therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.
Glucosylceramide synthase inhibitors Understanding
Glucosylceramide synthase (GCS) is a glucosyltranserase that processes the sphingolipid ceramide. The product, glucosylceramide, can be further elaborated with a variety of oligosaccharides to become glycosphingolipids called gangliosides (most notably GM1 and GM3). Excess glycosphingolipids have been correlated with a variety of human diseases. The pathologies associated with lysosomal storage disorders (Gaucher, Fabry, Tay-Sachs, and Sandhoff) stem from improper disposal of glucosylceramide and other glycosphingolipids. Gaucher disease (GD) is caused by a deficiency of glucocerebrosidase and the consequent lysosomal accumulation of unmetabolized glycolipid substrates. Substrate reduction therapy through inhibition of glucosylceramide synthase (GCS) has also been shown to effectively treat the visceral disease.
Report Highlights
This segment of the Glucosylceramide synthase inhibitors report encloses its detailed analysis of various drugs in different stages of clinical development, including phase III, II, I, preclinical and Discovery. It also helps to understand clinical trial details, expressive pharmacological action, agreements and collaborations, and the latest news and press releases.
Glucosylceramide synthase inhibitors Emerging Drugs
Lucerastat: Idorsia Pharmaceuticals
Lucerastat inhibits glycolipid biosynthesis. Lucerastat is an orally bioavailable inhibitor of glucosylceramide synthase. Lucerastat is being developed by the biopharmaceutical company Idorsia for the treatment of Fabry disease. It is currently in Phase III stage of development. It acts as Glucosylceramide synthase inhibitor. In 2018, Lucerastat has received Orphan Drug designation in the US and in the EU for Fabry disease.
Venglustat: Sanofi
Venglustat is an investigational oral glucosylceramide synthase inhibitor in development for multiple diseases with disruptions in the GSL metabolic pathway. These diseases include three lysosomal storage disorders: Gaucher disease type 3, Fabry disease and GM2 gangliosidosis as well as autosomal dominant polycystic kidney (ADPKD) disease and GBA-associated Parkinson's disease. The FDA has granted Fast Track designation for the development of venglustat as an investigational therapy for the treatment of Gaucher disease type 3, Fabry disease and GM2 gangliosidosis. Sanofi has been granted Orphan Drug Designation for venglustat in Europe and the U.S. for treatment of patients with Fabry disease, ADPKD, Gaucher disease and GM2 gangliosidosis.
This segment of the report provides insights about the different Glucosylceramide synthase inhibitors drugs segregated based on following parameters that define the scope of the report.
There are approx. 5+ key companies which are developing the Glucosylceramide synthase inhibitors. The companies which have their Glucosylceramide synthase inhibitors drug candidates in the most advanced stage, i.e. Phase III include, Idorsia Pharmaceuticals.
The drugs have been categorized under various product types like Mono, Combination and Mono/Combination.
Glucosylceramide synthase inhibitors: Pipeline Development Activities
The report provides insights into different therapeutic candidates in phase III, II, I, preclinical and discovery stage. It also analyses Glucosylceramide synthase inhibitors therapeutic drugs key players involved in developing key drugs.
Pipeline Development Activities
The report covers the detailed information of collaborations, acquisition and merger, licensing along with a thorough therapeutic assessment of emerging Glucosylceramide synthase inhibitors drugs.
Geography Covered
- Global coverage
Glucosylceramide synthase inhibitors Understanding
Glucosylceramide synthase inhibitors: Overview
Glucosylceramide synthase (GCS) is a glucosyltranserase that processes the sphingolipid ceramide. The product, glucosylceramide, can be further elaborated with a variety of oligosaccharides to become glycosphingolipids called gangliosides (most notably GM1 and GM3). Excess glycosphingolipids have been correlated with a variety of human diseases. The pathologies associated with lysosomal storage disorders (Gaucher, Fabry, Tay-Sachs, and Sandhoff) stem from improper disposal of glucosylceramide and other glycosphingolipids. Gaucher disease (GD) is caused by a deficiency of glucocerebrosidase and the consequent lysosomal accumulation of unmetabolized glycolipid substrates. Substrate reduction therapy through inhibition of glucosylceramide synthase (GCS) has also been shown to effectively treat the visceral disease.
Report Highlights
- The companies and academics are working to assess challenges and seek opportunities that could influence Glucosylceramide synthase inhibitors R&D. The therapies under development are focused on novel approaches for Glucosylceramide synthase inhibitors.
Glucosylceramide synthase inhibitors Emerging Drugs Chapters
This segment of the Glucosylceramide synthase inhibitors report encloses its detailed analysis of various drugs in different stages of clinical development, including phase III, II, I, preclinical and Discovery. It also helps to understand clinical trial details, expressive pharmacological action, agreements and collaborations, and the latest news and press releases.
Glucosylceramide synthase inhibitors Emerging Drugs
Lucerastat: Idorsia Pharmaceuticals
Lucerastat inhibits glycolipid biosynthesis. Lucerastat is an orally bioavailable inhibitor of glucosylceramide synthase. Lucerastat is being developed by the biopharmaceutical company Idorsia for the treatment of Fabry disease. It is currently in Phase III stage of development. It acts as Glucosylceramide synthase inhibitor. In 2018, Lucerastat has received Orphan Drug designation in the US and in the EU for Fabry disease.
Venglustat: Sanofi
Venglustat is an investigational oral glucosylceramide synthase inhibitor in development for multiple diseases with disruptions in the GSL metabolic pathway. These diseases include three lysosomal storage disorders: Gaucher disease type 3, Fabry disease and GM2 gangliosidosis as well as autosomal dominant polycystic kidney (ADPKD) disease and GBA-associated Parkinson's disease. The FDA has granted Fast Track designation for the development of venglustat as an investigational therapy for the treatment of Gaucher disease type 3, Fabry disease and GM2 gangliosidosis. Sanofi has been granted Orphan Drug Designation for venglustat in Europe and the U.S. for treatment of patients with Fabry disease, ADPKD, Gaucher disease and GM2 gangliosidosis.
Glucosylceramide synthase inhibitors: Therapeutic Assessment
This segment of the report provides insights about the different Glucosylceramide synthase inhibitors drugs segregated based on following parameters that define the scope of the report.
Major Players working on Glucosylceramide synthase inhibitors
There are approx. 5+ key companies which are developing the Glucosylceramide synthase inhibitors. The companies which have their Glucosylceramide synthase inhibitors drug candidates in the most advanced stage, i.e. Phase III include, Idorsia Pharmaceuticals.
Phases
This report covers around 5+ products under different phases of clinical development like
- Late-stage products (Phase III and
- Mid-stage products (Phase II and
- Early-stage products (Phase I/II and Phase I) along with the details of
- Pre-clinical and Discovery stage candidates
- Discontinued & Inactive candidates
- Route of Administration
Glucosylceramide synthase inhibitors pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as
- Infusion
- Intradermal
- Intramuscular
- Intranasal
- Intravaginal
- Oral
- Parenteral
- Subcutaneous
- Topical
- Molecule Type
Products have been categorized under various Molecule types such as
- Vaccines
- Monoclonal Antibody
- Peptides
- Polymer
- Small molecule
- Product Type
The drugs have been categorized under various product types like Mono, Combination and Mono/Combination.
Glucosylceramide synthase inhibitors: Pipeline Development Activities
The report provides insights into different therapeutic candidates in phase III, II, I, preclinical and discovery stage. It also analyses Glucosylceramide synthase inhibitors therapeutic drugs key players involved in developing key drugs.
Pipeline Development Activities
The report covers the detailed information of collaborations, acquisition and merger, licensing along with a thorough therapeutic assessment of emerging Glucosylceramide synthase inhibitors drugs.
Glucosylceramide synthase inhibitors Report Insights
- Glucosylceramide synthase inhibitors Pipeline Analysis
- Therapeutic Assessment
- Unmet Needs
- Impact of Drugs
Glucosylceramide synthase inhibitors Report Assessment
- Pipeline Product Profiles
- Therapeutic Assessment
- Pipeline Assessment
- Inactive drugs assessment
- Unmet Needs
Key Questions
Current Scenario and Emerging Therapies:
- How many companies are developing Glucosylceramide synthase inhibitors drugs?
- How many Glucosylceramide synthase inhibitors drugs are developed by each company?
- How many emerging drugs are in mid-stage, and late-stage of development for Glucosylceramide synthase inhibitors?
- What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the Glucosylceramide synthase inhibitors therapeutics?
- What are the recent trends, drug types and novel technologies developed to overcome the limitation of existing therapies?
- What are the clinical studies going on for Glucosylceramide synthase inhibitors and their status?
- What are the key designations that have been granted to the emerging drugs?
Key Players
- Idorsia Pharmaceuticals
- Sanofi
Key Products
- Lucerastat
- Venglustat
- Sinbaglustat
Table of Contents
IntroductionExecutive Summary
Glucosylceramide synthase inhibitors: Overview
- Structure
- Mechanism of Action
Pipeline Therapeutics
- Comparative Analysis
Therapeutic Assessment
- Assessment by Product Type
- Assessment by Stage and Product Type
- Assessment by Route of Administration
- Assessment by Stage and Route of Administration
- Assessment by Molecule Type
- Assessment by Stage and Molecule Type
Glucosylceramide synthase inhibitors- Analytical Perspective
In-depth Commercial Assessment
- Glucosylceramide synthase inhibitors companies’ collaborations, Licensing, Acquisition -Deal Value Trends
Glucosylceramide synthase inhibitors Collaboration Deals
- Company-Company Collaborations (Licensing / Partnering) Analysis
- Company-University Collaborations (Licensing / Partnering) Analysis
Late Stage Products (Phase III)
- Comparative Analysis
Lucerastat: Idorsia Pharmaceuticals
- Product Description
- Research and Development
- Product Development Activities
Mid Stage Products (Phase II)
- Comparative Analysis
Drug Name: Company Name
- Product Description
- Research and Development
- Product Development Activities
Early Stage Products (Phase I)
- Comparative Analysis
Sinbaglustat: Idorsia Pharmaceuticals
- Product Description
- Research and Development
- Product Development Activities
Pre-clinical and Discovery Stage Products
- Comparative Analysis
Drug Name: Company Name
- Product Description
- Research and Development
- Product Development Activities
Inactive Products
- Comparative Analysis
Glucosylceramide synthase inhibitors Key Companies
Glucosylceramide synthase inhibitors Key Products
Glucosylceramide synthase inhibitors- Unmet Needs
Glucosylceramide synthase inhibitors- Market Drivers and Barriers
Glucosylceramide synthase inhibitors- Future Perspectives and Conclusion
Glucosylceramide synthase inhibitors Analyst Views
Glucosylceramide synthase inhibitors Key Companies
AppendixList of Tables
Table 1 Total Products for Glucosylceramide synthase inhibitors
Table 2 Late Stage Products
Table 3 Mid Stage Products
Table 4 Early Stage Products
Table 5 Pre-clinical & Discovery Stage Products
Table 6 Assessment by Product Type
Table 7 Assessment by Stage and Product Type
Table 8 Assessment by Route of Administration
Table 9 Assessment by Stage and Route of Administration
Table 10 Assessment by Molecule Type
Table 11 Assessment by Stage and Molecule Type
Table 12 Inactive ProductsList of Figures
Figure 1 Total Products for Glucosylceramide synthase inhibitors
Figure 2 Late Stage Products
Figure 3 Mid Stage Products
Figure 4 Early Stage Products
Figure 5 Preclinical and Discovery Stage Products
Figure 6 Assessment by Product Type
Figure 7 Assessment by Stage and Product Type
Figure 8 Assessment by Route of Administration
Figure 9 Assessment by Stage and Route of Administration
Figure 10 Assessment by Molecule Type
Figure 11 Assessment by Stage and Molecule Type
Figure 12 Inactive Products
Companies Mentioned
A selection of companies mentioned in this report includes:
- Idorsia Pharmaceuticals
- Sanofi
