Fabry Disease Pipeline Insight

DelveInsight’s, “Fabry Disease – Pipeline Insight, 2021,” report provides comprehensive insights about 15+ companies and 15+ pipeline drugs in Fabry Disease 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. 

Geography Covered

  • Global coverage

Fabry Disease Understanding

Fabry Disease: Overview

Fabry Disease is a rare genetic disorder that prevents the body from making an enzyme called alpha-galactosidase A. This enzyme is responsible for breaking down a type of fat called globotriaosylceramide (Gb3 or GL-3) into building blocks that the body’s cells can use. Fabry Disease can have many symptoms because it affects cells throughout the body. The symptoms include: Chronic pain — usually a burning or tingling sensation — in the hands and feet, Clusters of small, dark red spots in various locations on the skin, Opaque corneas, resulting in cloudy-looking eyes and problems with eyesight, Ringing in the ears, and hearing loss. Fabry Disease is caused by a mutation of the GLA gene, which encodes for the alpha-galactosidase A enzyme. The mutation usually makes the enzyme unable to function, although in mild cases, it may function to some degree. The disease is inherited in an X-linked dominant manner, which means that the gene involved is situated on the X chromosome.


"Fabry Disease - Pipeline Insight, 2021" report by DelveInsight outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Fabry Disease pipeline landscape is provided which includes the disease overview and Fabry Disease treatment guidelines. The assessment part of the report embraces, in depth Fabry Disease commercial assessment and clinical assessment of the pipeline products under development. In the report, detailed description of the drug is given which includes mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, Fabry Disease collaborations, licensing, mergers and acquisition, funding, designations and other product related details.

Report Highlights

The companies and academics are working to assess challenges and seek opportunities that could influence Fabry Disease R&D. The therapies under development are focused on novel approaches to treat/improve Fabry Disease.

Fabry Disease Emerging Drugs Chapters

This segment of the Fabry Disease report encloses its detailed analysis of various drugs in different stages of clinical development, including phase 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.


Fabry Disease Emerging Drugs


Pegunigalsidase alfa: Protalix Biotherapeutics

Pegunigalsidase alfa is designed to be a plant cell culture-expressed, and a chemically modified version of, the recombinant alpha-Galactosidase-A protein. Protein sub-units are covalently bound via chemical cross-linking using PEG chains. In October 2017, Protalix entered into a license and collaboration agreement with Chiesi Farmaceutici S.p.A., with respect to the commercialization of pegunigalsidase alfa (PRX-102) for the treatment of Fabry Disease.


Venglustat: Sanofi

Venglustat is an oral inhibitor of an enzyme called glucosylceramide synthase (GCS). GCS turns its substrate, ceramide, into glucosylceramide (GL-1) during lipid metabolism, a series of biochemical reactions that degrade and generate lipids. GL-1 acts as a substrate to other enzymes and is turned into globosides, a subclass of lipids where Gb3 belongs. The U.S. Food and Drug Administration granted venglustat fast-track designation in 2015 for the treatment of Fabry Disease. The drug is currently in Phase II stage of development for Fabry Disease.


4D 310: 4D Molecular Therapeutics

4D-310 is a novel adeno-associated virus (AAV) gene therapy comprised of two active components: the capsid (4D-C102) and the transgene cassette, which encodes a codon-optimized full length human GLA transgene driven by the CAG promoter. 4D-310 has been engineered so that it cannot replicate. The drug is being investiaged in Phase I/II stage of development for the treatment of patients with Fabry Disease.

Further product details are provided in the report……..

Fabry Disease: Therapeutic Assessment

This segment of the report provides insights about the different Fabry Disease drugs segregated based on following parameters that define the scope of the report, such as:

  • Major Players in Fabry Disease

There are approx. 15+ key companies which are developing the therapies for Fabry Disease. The companies which have their Fabry Disease drug candidates in the most advanced stage, i.e. preregistration include Protalix Biotherapeutics.

Phases

DelveInsight’s report covers around 15+ products under different phases of clinical development like

  • Late stage products (Phase III)
  • Mid-stage products (Phase II)
  • Early-stage product (Phase I) along with the details of
  • Pre-clinical and Discovery stage candidates
  • Discontinued & Inactive candidates
  • Route of Administration


Fabry Disease pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as

  • Oral
  • Parenteral
  • Intravitreal
  • Subretinal
  • Topical.
  • Molecule Type


Products have been categorized under various Molecule types such as

  • Monoclonal Antibody
  • Peptides
  • Polymer
  • Small molecule
  • Gene therapy
  • Product Type

Drugs have been categorized under various product types like Mono, Combination and Mono/Combination.

Fabry Disease: Pipeline Development Activities

The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Fabry Disease 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 Fabry Disease drugs.

Fabry Disease Report Insights

  • Fabry Disease Pipeline Analysis
  • Therapeutic Assessment
  • Unmet Needs
  • Impact of Drugs

Fabry Disease Report Assessment

  • Pipeline Product Profiles
  • Therapeutic Assessment
  • Pipeline Assessment
  • Inactive drugs assessment
  • Unmet Needs

Key Questions

Current Treatment Scenario and Emerging Therapies:

  • How many companies are developing Fabry Disease drugs?
  • How many Fabry Disease drugs are developed by each company?
  • How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Fabry Disease?
  • What are the key collaborations (Industry–Industry, Industry–Academia), Mergers and acquisitions, licensing activities related to the Fabry Disease 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 Fabry Disease and their status?
  • What are the key designations that have been granted to the emerging drugs?


Introduction

Executive Summary

Fabry Disease: Overview

  • Causes
  • Mechanism of Action
  • Signs and Symptoms
  • Diagnosis
  • Disease Management

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

Fabry Disease – DelveInsight’s Analytical Perspective

In-depth Commercial Assessment

  • Fabry Disease companies’ collaborations, Licensing, Acquisition -Deal Value Trends

Fabry Disease Collaboration Deals

  • Company-Company Collaborations (Licensing / Partnering) Analysis
  • Company-University Collaborations (Licensing / Partnering) Analysis

Late Stage Products (Preregistration)

  • Comparative Analysis

Pegunigalsidase alfa: Protalix Biotherapeutics

  • Product Description
  • Research and Development
  • Product Development Activities

Drug profiles in the detailed report…..

Mid Stage Products (Phase II)

  • Comparative Analysis

Venglustat: Sanofi

  • Product Description
  • Research and Development
  • Product Development Activities

Drug profiles in the detailed report…..

Early Stage Products (Phase I/II)

  • Comparative Analysis

4D 310: 4D Molecular Therapeutics

  • Product Description
  • Research and Development
  • Product Development Activities

Drug profiles in the detailed report…..

Inactive Products

  • Comparative Analysis

Fabry Disease Key Companies

Fabry Disease Key Products

Fabry Disease- Unmet Needs

Fabry Disease- Market Drivers and Barriers

Fabry Disease- Future Perspectives and Conclusion

Fabry Disease Analyst Views

Fabry Disease Key Companies

Appendix

List of Table

Table 1: Total Products for Fabry Disease

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 Products

List of Figures

Figure 1: Total Products for Fabry Disease

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      

• Protalix Biotherapeutics

• Sanofi

• 4D Molecular therapeutics

• Ozmosis Research Inc.

• AvroBio

• Freeline Therapeutics

• Sangamo Therapeutics

• Translate Bio

• Abeona Therapeutics

• AVROBIO

• Sangamo Therapeutics

  • Tags:
  • Fabry Disease Pipeline
  • Fabry Disease clinical trials
  • Fabry Disease companies
  • Fabry Disease drugs
  • Fabry Disease therapies
  • Fabry Disease treatment algorithm
  • Fabry Disease pipeline drugs
  • Fabry Disease pipeline companies

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