Familial Lipoprotein Lipase Deficiency: A Rare Inherited Condition, in Which the Normal Breakdown of Fats in the Body is Affected - Global Pipeline Insight Report 2020 - ResearchAndMarkets.com

DUBLIN--(BUSINESS WIRE)--The "Familial Lipoprotein Lipase Deficiency - Pipeline Insight, 2020" drug pipelines has been added to ResearchAndMarkets.com's offering.

This "Familial Lipoprotein Lipase Deficiency - Pipeline Insight, 2020," report provides comprehensive insights about 8+ companies and 8+ pipeline drugs in Familial Lipoprotein Lipase Deficiency 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.

Familial Lipoprotein Lipase Deficiency: Overview

Familial Lipoprotein Lipase Deficiency (LPLD) is a rare inherited condition, in which the normal breakdown of fats in the body is affected. The condition is inherited in an autosomal recessive pattern. This deficiency is usually caused by a defect in the LPL gene, which encodes for an enzyme called lipoprotein lipase.

Lipoprotein lipase (LPL) is a key enzyme that ensures triglycerides are unloaded from chylomicrons. Absence of this enzyme leads to increased blood triglyceride and chylomicron levels. Persons with LPLD cannot break down fat from digested food and therefore fat particles called chylomicrons build up in the blood.

Diagnosis

A diagnosis of Familial Lipoprotein Lipase Deficiency may be done based upon identification of characteristic symptoms, a detailed patient history, and a thorough clinical evaluation. Blood tests can reveal reduced activity of the lipoprotein lipase enzyme in the plasma. Molecular genetic testing can confirm the diagnosis of Familial Lipoprotein Lipase Deficiency.

Treatment

Treatment for Familial Lipoprotein Lipase Deficiency aims to control the symptoms and blood triglyceride levels with a very low-fat diet. Drugs that lower lipid levels in the body are not effective in reducing fat levels. Alcohol and drugs that increase triglyceride levels should be avoided. Gene Therapy is approved for the treatment, specifically for patients who have not responded to other treatments

Familial Lipoprotein Lipase Deficiency Emerging Drugs Chapters

This segment of the Familial Lipoprotein Lipase Deficiency 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 Lipoprotein Lipase Deficiency Emerging Drugs

ARO-APOC3: Arrowhead Pharmaceuticals

ARO-APOC3 is a subcutaneously administered RNAi therapeutic that targets apolipoprotein C-III (apoC-III), and reduces VLDL synthesis and assembly, enhances the breakdown of triglyceride rich lipoproteins, and improve clearance of VLDL and chylomicron remnants.

Vupanorsen: Ionis Pharmaceuticals

Vupanorsen is an investigational antisense therapy to reduce the production of angiopoietin-like 3 (ANGPTL3) protein, a key regulator of triglyceride and cholesterol metabolism, in the liver. The therapy was developed using Ionis' advanced LIgand Conjugated Antisense (LICA) technology platform.

Familial Lipoprotein Lipase Deficiency: Therapeutic Assessment

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

Major Players in Familial Lipoprotein Lipase Deficiency

There are approx. 8+ key companies which are developing the therapies for Familial Lipoprotein Lipase Deficiency. The companies which have their Familial Lipoprotein Lipase Deficiency drug candidates in the most advanced stage, i.e. phase III include, Ionis Pharmaceuticals.

Report Highlights

• The companies and academics are working to assess challenges and seek opportunities that could influence Familial Lipoprotein Lipase Deficiency R&D. The therapies under development are focused on novel approaches to treat/improve Familial Lipoprotein Lipase Deficiency.
• In August, 2020 Ionis Pharmaceuticals and its affiliate Akcea Therapeutics announced that they have entered into a definitive agreement under which Ionis will acquire all of the outstanding shares of Akcea common stock. In November 2019, Akcea and Ionis announced the closing of a worldwide exclusive licensing agreement with Pfizer Inc. for vupanorsen development. Pfizer is responsible for all development and regulatory activities and costs beyond those associated with the Phase 2 study.

Familial Lipoprotein Lipase Deficiency Report Insights

• Familial Lipoprotein Lipase Deficiency Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

Familial Lipoprotein Lipase Deficiency Report Assessment

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

Key Players

• Arrowhead Pharmaceuticals
• Novartis
• Ionis Pharmaceuticals
• Catabasis Pharmaceuticals
• iMetabolic Biopharma
• Precision Biosciences

Key Products

• ARO-APOC3
• Pradigastat
• Vupanorsen
• CAT-2003
• iMBP-001
• ApoC3 gene editing therapeutic
• IONIS APOCIII LRx

For more information about this drug pipelines report visit https://www.researchandmarkets.com/r/k10swe