Familial Chylomicronemia Syndrome Pipeline Insight

DelveInsight’s, “Familial Chylomicronemia Syndrome - Pipeline Insight, 2021,” report provides comprehensive insights about 6+ companies and 6+ pipeline drugs in Familial Chylomicronemia Syndrome 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

Familial Chylomicronemia Syndrome Understanding

Familial Chylomicronemia Syndrome: Overview

Familial chylomicronemia syndrome (FCS), formerly known as type 1 hyperlipoproteinemia, is a rare autosomal recessive monogenic disease caused by mutations in the LPL gene. The disease is characterized by hyperchylomicronemia and recurrent episodes of abdominal pain and/or acute, potentially fatal, pancreatitis. The most serious complication of FCS is acute, and often recurrent, pancreatitis secondary to severe hypertriglyceridemia. FCS is diagnosed by a combination of clinical signs and symptoms, and extreme elevations in triglyceride levels. Treatment for FCS aims to control blood triglyceride levels with a very low-fat diet. Traditional medications used to lower triglycerides are often ineffective in patients with FCS. Avoidance of alcohol and simple, refined carbohydrates, such as those found in sodas and candy, are also very important to prevent further rise in triglycerides.


"Familial Chylomicronemia Syndrome - Pipeline Insight, 2021" report by DelveInsight outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Familial Chylomicronemia Syndrome pipeline landscape is provided which includes the disease overview and Familial Chylomicronemia Syndrome treatment guidelines. The assessment part of the report embraces, in depth Familial Chylomicronemia Syndrome 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, Familial Chylomicronemia Syndrome 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 Familial Chylomicronemia Syndrome R&D. The therapies under development are focused on novel approaches to treat/improve Familial Chylomicronemia Syndrome.

Familial Chylomicronemia Syndrome Emerging Drugs Chapters

This segment of the Familial Chylomicronemia Syndrome 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.


Familial Chylomicronemia Syndrome Emerging Drugs


Vupanorsen: Pfizer

Vupanorsen (PF-07285557), formerly known as IONIS-ANGPTL3-LRx and AKCEA-ANGPTL3-LRx is a ligand-conjugated (LICA) investigational antisense medicine designed to reduce angiopoietin-like 3 protein, or ANGPTL3. This antisense therapy was developed using Ionis' advanced LIgand Conjugated Antisense (LICA) technology platform. Vupanorsen was discovered by Ionis and has been co-developed by Akcea and Ionis. In November 2019, Akcea and Ionis announced the closing of a worldwide exclusive licensing agreement with Pfizer Inc. for vupanorsen. Pfizer is responsible for all development and regulatory activities and costs beyond those associated with the Phase 2a study.


IONIS-APOCIII-LRx: Ionis Pharmaceuticals

IONIS-APOCIII-LRx, formerly known as AKCEA-APOCIII-LRx, is a ligand-conjugated (LICA) investigational antisense medicine designed to inhibit the production of apoC-III, for patients who are at risk of disease due to elevated triglyceride levels. AKCEA-APOCIII-LRx is designed using Ionis' proprietary Ligand Conjugated Antisense (LICA) technology platform to inhibit production of apolipoprotein C-III (apoC-III). The drug is in Phase III clinical development for the treatment of FCS.

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

Familial Chylomicronemia Syndrome: Therapeutic Assessment

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

  • Major Players in Familial Chylomicronemia Syndrome

There are approx. 6+ key companies which are developing the therapies for Familial Chylomicronemia Syndrome. The companies which have their Familial Chylomicronemia Syndrome drug candidates in the most advanced stage, i.e. Phase III include, Ionis Pharmaceuticals.

Phases

DelveInsight’s report covers around 6+ 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


Familial Chylomicronemia Syndrome 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.

Familial Chylomicronemia Syndrome: Pipeline Development Activities

The report provides insights into different therapeutic candidates in phase III, II, I, preclinical and discovery stage. It also analyses Familial Chylomicronemia Syndrome 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 Familial Chylomicronemia Syndrome drugs.

Familial Chylomicronemia Syndrome Report Insights

  • Familial Chylomicronemia Syndrome Pipeline Analysis
  • Therapeutic Assessment
  • Unmet Needs
  • Impact of Drugs

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

Introduction

Executive Summary

Familial Chylomicronemia Syndrome: 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

Familial Chylomicronemia Syndrome – DelveInsight’s Analytical Perspective

In-depth Commercial Assessment

  • Familial Chylomicronemia Syndrome companies’ collaborations, Licensing, Acquisition -Deal Value Trends

Familial Chylomicronemia Syndrome Collaboration Deals

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

Late Stage Products (Phase III)

  • Comparative Analysis

IONIS-APOCIII-LRx: Ionis Pharmaceuticals

  • Product Description
  • Research and Development
  • Product Development Activities

Drug profiles in the detailed report…..

Mid Stage Products (Phase II)

  • Comparative Analysis

Vupanorsen: Pfizer

  • Product Description
  • Research and Development
  • Product Development Activities

Drug profiles in the detailed report…..

Early Stage Products (Phase I)

  • Comparative Analysis

ARO-APOC3: Arrowhead Pharmaceuticals

  • Product Description
  • Research and Development
  • Product Development Activities

Drug profiles in the detailed report…..

Preclinical/Discovery Stage Products

  • Comparative Analysis

iMBP-001: iMetabolic Biopharma

  • Product Description
  • Research and Development
  • Product Development Activities

Drug profiles in the detailed report…..

Inactive Products

  • Comparative Analysis

Familial Chylomicronemia Syndrome Key Companies

Familial Chylomicronemia Syndrome Key Products

Familial Chylomicronemia Syndrome- Unmet Needs

Familial Chylomicronemia Syndrome- Market Drivers and Barriers

Familial Chylomicronemia Syndrome- Future Perspectives and Conclusion

Familial Chylomicronemia Syndrome Analyst Views

Familial Chylomicronemia Syndrome Key Companies

Appendix

List of Table

Table 1: Total Products for Familial Chylomicronemia Syndrome

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 Familial Chylomicronemia Syndrome

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

• Pfizer

• Ionis Pharmaceuticals

• iMetabolic Biopharma

• Arrowhead Pharmaceuticals

• Lipigon Pharmaceuticals

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