Familial Hypercholesterolemia (Type II Hyperlipoproteinemia - Pipeline Insight, 2024

This “Familial Hypercholesterolemia - Pipeline Insight, 2024,” report provides comprehensive insights about 10+ companies and 10+ pipeline drugs in Familial Hypercholesterolemia 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 Hypercholesterolemia Understanding

Familial Hypercholesterolemia: Overview

Familial hypercholesterolemia (FH) is a common, inherited disorder of cholesterol metabolism characterized by very high plasma concentrations of low-density lipoprotein cholesterol. The disease, if left untreated can leads to early heart attacks and heart disease. The diagnosis of both homozygous and heterozygous FH is based primarily on the finding of severe low-density lipoprotein cholesterol (LDL-C) elevations in the absence of secondary causes of hypercholesterolemia. Treatment of FH is focused on reducing the LDL-C levels in order to decrease the risk for heart disease. Pharmacotherapy involves medicines like statin drug, and other cholesterol-lowering medications such as ezetimibe, bile acid sequestrants, bempedoic acid and icosapent ethyl or PCSK9 inhibitors like Praluent may be used. Lifestyle intervention and exercise are also helpful along with medication.

Familial Hypercholesterolemia - Pipeline Insight, 2024 report outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Familial Hypercholesterolemia pipeline landscape is provided which includes the disease overview and Familial Hypercholesterolemia treatment guidelines. The assessment part of the report embraces, in depth Familial Hypercholesterolemia 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 Hypercholesterolemia 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 Hypercholesterolemia R&D. The therapies under development are focused on novel approaches to treat/improve Familial Hypercholesterolemia.

Familial Hypercholesterolemia Emerging Drugs Chapters

This segment of the Familial Hypercholesterolemia 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 Hypercholesterolemia Emerging Drugs

Ebronucimab: Akeso Biopharma Ebronucimab (AK-102) is a monoclonal antibody based therapy targeting proprotein convertase subtilisin/kexin type 9 (PCSK9). Ebronucimab is being developed for the treatment of primary hyperlipidemias, including homozygous familial hypercholesterolemia (Ho FH), heterozygous familial hypercholesterolemia (HeFH) and other primaryhypercholesterolemiapatients.

Tafolecimab: Innovent Biologics Tafolecimab (IBI306) is fully human monoclonal antibody targeting proprotein convertase subtilisin/kexin type 9. The drug is currently in Phase III clinical trials for patients with homozygous or heterozygous familial hypercholesterolemia. PCSK9 is a protein that targets LDL receptors for degradation and thereby reduces the liver’s ability to remove LDL-C, or “bad” cholesterol, from theblood.

Familial Hypercholesterolemia: Therapeutic Assessment

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

Major Players in Familial Hypercholesterolemia

There are approx. 10+ key companies which are developing the therapies for Familial Hypercholesterolemia. The companies which have their Familial Hypercholesterolemia drug candidates in the most advanced stage, i.e. Phase III include, Innovent Biologics.

Phases

This report covers around 10+ 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 Hypercholesterolemia 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 Hypercholesterolemia: Pipeline Development Activities

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

Familial Hypercholesterolemia Report Insights

• Familial Hypercholesterolemia Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

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

Key Players

• Akeso Biopharma
• Innovent Biologics
• Jiangsu Hengrui Medicine
• LIB Therapeutics
• Arrowhead Pharmaceuticals
• Precision BioSciences
• Verve Therapeutics
• SalioGen Therapeutics

Key Products

• Ebronucimab
• Tafolecimab
• SHR-1209
• Lerodalcibep
• ARO-ANG3
• PCSK9 gene editing therapeutic
• VERVE-101

Research programme: gene therapies


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