Familial Hypercholesterolemia Drug Pipeline Analysis Report 2025

Familial hypercholesterolemia is a genetic disorder characterized by high cholesterol levels, leading to an increased risk of early cardiovascular diseases. It accounts for approximately 450,000 children born annually. However, only 2.1% of adults are diagnosed before age 18. There is a significant clinical need for better therapies, as current treatments, such as statins and LDL apheresis, are not always effective. The growing focus on gene therapy, novel lipid-lowering drugs, and advanced therapeutics is likely to support pipeline growth, offering promising familial hypercholesterolemia treatment options in the coming years.

Report Coverage

The Familial Hypercholesterolemia Drug Pipeline Insight Report by the publisher gives comprehensive insights into familial hypercholesterolemia therapeutics currently undergoing clinical trials. It covers various aspects related to the details of each of these drugs under development for familial hypercholesterolemia. The familial hypercholesterolemia report assessment includes the analysis of over 100 pipeline drugs and 50+ companies. The familial hypercholesterolemia pipeline landscape will include an analysis based on efficacy and safety measure outcomes published for the trials, including their adverse effects on patients suffering from the condition, and alignment with familial hypercholesterolemia treatment guidelines to ensure optimal care practices.

The assessment part will include a detailed analysis of each drug, drug class, clinical studies, phase type, drug type, route of administration, and ongoing product development activities related to familial hypercholesterolemia.

Familial Hypercholesterolemia Drug Pipeline Outlook

Familial hypercholesterolemia is a genetic disorder characterized by abnormally high levels of low-density lipoprotein cholesterol (LDL-C) in the blood. It occurs due to mutations in the LDL receptor gene, impairing the liver's ability to remove LDL-C. This leads to early plaque buildup in arteries, increasing the risk of heart disease.

Familial hypercholesterolemia treatment typically involves statins to lower LDL-C levels, PCSK9 inhibitors to further reduce cholesterol, and ezetimibe to block cholesterol absorption in the intestines. In severe cases, LDL apheresis, a procedure to remove excess cholesterol from the blood, may be necessary. Liver transplantation is an option for those with severe cases of unresponsiveness to medications.

Familial Hypercholesterolemia Epidemiology

Familial hypercholesterolemia affects approximately 450,000 children born globally each year, yet only 2.1% of adults are diagnosed before the age of 18. Around 1.3 million individuals in the United States have familial hypercholesterolemia, with only 10% being aware of their condition. The condition is estimated to impact about 270,000 people in the United Kingdom, 1 in 300 individuals in Japan, and 0.1% of the population in India.

Familial Hypercholesterolemia - Drug Pipeline Therapeutic Assessment

This section of the report covers the analysis of familial hypercholesterolemia drug candidates based on several segmentations, including:

By Phase

• Late-Stage Products (Phase 3 and Phase 4)
• Mid-Stage Products (Phase 2)
• Early-Stage Products (Phase I)
• Preclinical and Discovery Stage Products

By Drug Class

• Monoclonal Antibodies
• Gene Therapies
• Small Molecules
• RNA-Based Therapies
• Enzyme Inhibitors

By Route of Administration

• Oral
• Parenteral
• Others

Familial Hypercholesterolemia - Pipeline Assessment Segmentation, By Phases

The report covers phase I, phase II, phase III, phase IV, and early-phase drugs. The coverage includes an in-depth analysis of each drug across these phases. According to analysis, phase III covers a major share of the total familial hypercholesterolemia clinical trials.

Familial Hypercholesterolemia - Pipeline Assessment Segmentation, By Drug Classes

The drug molecule categories covered under the familial hypercholesterolemia pipeline analysis include monoclonal antibodies, gene therapies, small molecules, RNA-based therapies, and enzyme inhibitors. The familial hypercholesterolemia report provides a comparative analysis of the drug classes for each drug in various phases of clinical trials for familial hypercholesterolemia.

Familial Hypercholesterolemia Clinical Trials Therapeutic Assessment - Competitive Dynamics

The report for the familial hypercholesterolemia drug pipeline covers the profile of key companies involved in clinical trials and their drugs under development. It provides a detailed familial hypercholesterolemia therapeutic assessment, analyzing the competitive dynamics of the clinical trial landscape. Below is the list of a few players involved in familial hypercholesterolemia clinical trials:

• Novartis Pharmaceuticals
• Merck Sharp & Dohme LLC
• Beijing Suncadia Pharmaceuticals Co., Ltd.
• Arrowhead Pharmaceuticals
• Visirna Therapeutics HK Ltd.
• Verve Therapeutics, Inc.
• Esperion Therapeutics, Inc.
• LIB Therapeutics LLC
• Hasten Biopharmaceutical Co., Ltd.
• Qilu Pharmaceutical Co., Ltd.
• Ultragenyx Pharmaceutical Inc.
• Medpace, Inc.

Familial Hypercholesterolemia Emerging Drugs Profile

This section covers the detailed analysis of each drug under multiple phases, including phase I, phase II, phase III, phase IV, and emerging drugs for familial hypercholesterolemia. It includes product description, trial ID, study type, drug class, mode of administration, and recruitment status of familial hypercholesterolemia drug candidates.

Drug: Enlicitide Decanoate

Enlicitide Decanoate (MK-0616), an oral PCSK9 inhibitor, is being evaluated in the Phase III CORALreef study. The study, sponsored by Merck Sharp & Dohme LLC, aims to assess its efficacy, safety, and tolerability in adults with heterozygous familial hypercholesterolemia.

Drug: ARO-ANG 3 Injection

ARO-ANG3 is an investigational RNAi therapeutic targeting ANGPTL3, a protein regulating lipid metabolism, to potentially improve lipid clearance and lower cholesterol levels. The Phase II study of ARO-ANG3, sponsored by Arrowhead Pharmaceuticals, aims to evaluate the safety and efficacy of ARO-ANG3 in participants with homozygous familial hypercholesterolemia (HoFH). The study will involve up to 36 weeks of treatment with an optional 24-month extension.

Drug: VERVE-101

VERVE-101, developed by Verve Therapeutics, is undergoing a Phase I clinical trial to assess its safety in patients with heterozygous familial hypercholesterolemia (HeFH) and atherosclerotic cardiovascular disease (ASCVD). This single-dose, liver-base-editing drug targets the PCSK9 gene to reduce LDL-C levels, aiming to improve cardiovascular health in patients with uncontrolled hypercholesterolemia.

Reasons To Buy This Report

The Familial Hypercholesterolemia Drug Pipeline Insight Report provides a strategic overview of the latest and future landscape of treatments for familial hypercholesterolemia. It provides necessary information for making informed investment decisions along with research, development, and strategic planning efforts. The stakeholders will benefit from the essential insights into familial hypercholesterolemia collaborations, regulatory environments, and potential growth opportunities.

Key Questions Answered in the Familial Hypercholesterolemia - Pipeline Insight Report

• Which companies/institutions are leading the familial hypercholesterolemia drug development?
• What is the efficacy and safety profile of familial hypercholesterolemia pipeline drugs?
• Which company is leading the familial hypercholesterolemia pipeline development activities?
• What is the current familial hypercholesterolemia commercial assessment?
• What are the opportunities and challenges present in the familial hypercholesterolemia pipeline landscape?
• What is the efficacy and safety profile of familial hypercholesterolemia pipeline drugs?
• Which company is conducting major trials for familial hypercholesterolemia drugs?
• Which companies/institutions are involved in familial hypercholesterolemia collaborations aimed at providing enhanced therapeutic alternatives for patients?
• What are the geographies covered for clinical trials in familial hypercholesterolemia?

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