Familial Chylomicronemia Syndrome - Pipeline Insight, 2024

This “Familial Chylomicronemia Syndrome - Pipeline Insight, 2024,” report provides comprehensive insights about 5+ companies and 5+ 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 hyperchylomicronemia syndrome is a rare autosomal recessive metabolic disorder caused by the mutation in lipoprotein lipase. It correlates with significant morbidity and distress amongst the patients. This article describes the background, pathophysiology, history, and physical and evaluation of type 1 familial hyperlipidemia. The pathophysiology behindfamilial hyperchylomicronemia syndrome is a mutation in the enzyme lipoprotein lipase. Lipoprotein lipase is an enzyme that is present in the vascular endothelial surface. It degrades circulating triglycerides found in chylomicrons and other triglycerides-rich lipoproteins (TGRL) in the bloodstream.

Familial Chylomicronemia Syndrome - Pipeline Insight, 2024 report 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, 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 in Familial Chylomicronemia Syndrome R&D. The therapies under development are focused on novel approaches to treat/improve in Familial Chylomicronemia Syndrome.

This segment of the Familial Chylomicronemia Syndrome 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.

Familial Chylomicronemia Syndrome Emerging Drugs

Olezarsen: Ionis Pharmaceuticals Olezarsen, formerly known as IONIS-APOCIII-LRx and AKCEA-APOCIII-LRx, is a ligand-conjugated (LICA) investigational antisense medicine designed to inhibit the production of apoC-III in the liver. ApoC-III is a protein that regulates triglyceride metabolism in the blood by inhibiting lipoprotein lipase that breakdown triglycerides and by preventing clearance of triglyceride-rich lipoproteins. ApoC-III is an independent risk factor for both pancreatitis and cardiovascular disease.

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. 5+ 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

This report covers around 5+ 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
• Intravenous
• Subcutaneous
• 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 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?
• 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?

Key Players

• Ionis Pharmaceuticals
• Pfizer
• Arrowhead Pharmaceuticals
• Lipigon Pharmaceuticals

Key Products

• Olezarsen
• Vupanorsen
• ARO-APOC3
• Lipisense

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