Familial Hypercholesterolemia Pipeline

DelveInsight’s, “Familial Hypercholesterolemia - Pipeline Insight, 2025” report provides comprehensive insights about 8+ 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 genetic disorder marked by significantly elevated low-density lipoprotein (LDL) cholesterol levels due to inherited defects, most commonly transmitted in an autosomal dominant pattern, though codominant inheritance with over 90% penetrance is also observed. Classified primarily as type IIa hyperlipidemia under the Fredrickson system, FH predisposes patients to accelerated atherosclerosis, substantially increasing the risk of premature cardiovascular disease and early mortality. Early diagnosis and aggressive LDL-lowering interventions are critical to prevent or slow coronary artery disease progression, and screening of first-degree relatives is strongly recommended to identify carriers and initiate timely treatment.

Familial hypercholesterolemia (FH) is a genetic disorder characterized by elevated LDL cholesterol and increased risk of premature cardiovascular disease, and it is primarily classified into two types. Heterozygous FH (HeFH) results from a mutation in a single allele of genes such as LDLR, APOB, or PCSK9, leading to moderately elevated LDL levels and a heightened risk of early cardiovascular events. Homozygous FH (HoFH), a rare and more severe form, arises from mutations in both alleles of FH-related genes, causing extremely high LDL cholesterol levels and early-onset cardiovascular disease.

In homozygous familial hypercholesterolemia (FH), extremely high plasma LDL-C levels cause early and extensive cholesterol deposits, with signs often appearing within the first year of life. These include tendon xanthomas, most commonly on the Achilles tendons and hands, tuberous xanthomas over extensor surfaces such as the knees and elbows, and painful planar xanthomas on the palms and soles. Xanthelasmas, soft yellow cholesterol plaques, typically form on the eyelids, while corneal arcus, a white or gray ring around the cornea, is also common. In contrast, heterozygous FH presents with elevated serum cholesterol and increased risk of premature coronary artery disease, with tendon xanthomas and corneal arcus generally developing later, usually after the age of 20.

The pathophysiology of familial hypercholesterolemia (FH) involves impaired clearance of low-density lipoprotein (LDL) cholesterol from the plasma due to genetic defects affecting the LDL receptor (LDLR), apolipoprotein B-100 (apoB-100), or the proprotein convertase subtilisin/kexin type 9 (PCSK9) gene. Normally, LDL particles bind to LDLR via apoB-100 or apoE, undergo endocytosis, and are released into cells while the receptor recycles back to the membrane. PCSK9, secreted by the liver, binds to LDLR and prevents its recycling, thereby reducing receptor availability and increasing serum LDL levels. Mutations in apoB-100 disrupt its ability to bind LDLR, leading to reduced clearance and elevated plasma LDL, while gain-of-function mutations in PCSK9 increase receptor degradation and worsen hypercholesterolemia, whereas loss-of-function mutations in PCSK9 have protective effects by lowering LDL and reducing coronary heart disease risk. Overall, these genetic alterations impair LDL clearance, promote foam cell formation, and drive the development of atherosclerosis in FH.

The management of familial hypercholesterolemia (FH) involves lifestyle modification, including a healthy diet, smoking cessation, regular exercise, and weight control, alongside pharmacologic therapy to reduce cardiovascular risk. Statins are the first-line treatment, lowering LDL by about 50%, though many patients do not reach target levels. Ezetimibe is the preferred second-line therapy, adding a further 10–30% reduction, while PCSK9 inhibitors can lower LDL by 50–60% but are limited by high cost and access issues. For refractory cases, advanced options such as mipomersen, lomitapide, niacin, lipoprotein apheresis, or surgical approaches may be considered. LDL levels should be closely monitored, with targets set according to individual risk, typically below 70 mg/dL in high-risk patients and below 100 mg/dL in lower-risk patients.

 

"Familial Hypercholesterolemia- Pipeline Insight, 2025" report by DelveInsight 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

• Zodasiran: Arrowhead Pharmaceuticals

Zodasiran (formerly ARO-ANG3) is a first-in-class investigational RNAi therapeutic that targets angiopoietin-like protein 3 (ANGPTL3), a liver-expressed regulator of lipid and lipoprotein metabolism. By silencing ANGPTL3, it may act through multiple mechanisms, including inhibition of lipoprotein lipase (LPL) and endothelial lipase (EL).Currently, the drug is in Phase III stage of its clinical study for the treatment of Homozygous Familial Hypercholesterolemia.

 

• VERVE-102: Verve Therapeutics, Inc.

VERVE-102 is a first-in-class, investigational in vivo base-editing therapy designed as a one-time treatment to inactivate the PCSK9 gene in the liver, providing a sustained reduction in blood LDL-C levels. It consists of an mRNA encoding the base editor along with a guide RNA that precisely directs the editor to the PCSK9 gene sequence. It employs an internally developed GalNAc-LNP delivery system to introduce a base editor that targets the PCSK9 gene in the liver. Currently, the drug is in Phase Ib stage of its clinical study for the treatment of Heterozygous Familial Hypercholesterolemia.

 

• CTX310: CRISPR Therapeutics

CTX310 is designed to inhibit ANGPTL3, a key regulator of LDL and triglyceride levels, both major risk factors for atherosclerotic cardiovascular disease (ASCVD). Natural loss-of-function mutations in ANGPTL3 are linked to lower LDL and triglycerides and reduced ASCVD risk without adverse effects. With over 40 million people in the U.S. affected by elevated LDL, high triglycerides, or both, CTX310 addresses a significant unmet need, initially focusing on high-risk patients with limited treatment options. Currently, the drug is in Phase I stage of its clinical study for the treatment of Homozygous Familial Hypercholesterolemia and Heterozygous Familial Hypercholesterolemia.

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

 

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. 8+ 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, Arrowhead Pharmaceuticals

 

Phases

DelveInsight’s 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
• Intravenous
• Subcutaneous
• Parenteral
• Topical

 

Molecule Type

Products have been categorized under various Molecule types such as

• Recombinant fusion proteins
• Small molecule
• Monoclonal antibody
• Peptide
• Polymer
• 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?