Homozygous Familial Hypercholesterolemia Pipeline Drugs 2025 Report: Emerging Drugs, Innovative Therapies, Clinical Trial Updates, and Top Companies

DelveInsight's "Homozygous Familial Hypercholesterolemia Pipeline Insight 2025" report provides comprehensive insights about 3+ companies and 3+ pipeline drugs in the Homozygous Familial Hypercholesterolemia pipeline landscape. It covers the Homozygous Familial Hypercholesterolemia Pipeline drug profiles, including clinical and nonclinical stage products. It also covers the Homozygous Familial Hypercholesterolemia Pipeline Therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.

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Key Takeaways from the Homozygous Familial Hypercholesterolemia Pipeline Report

On 10 October 2025, Arrowhead Pharmaceuticals conducted a study will evaluate the efficacy and safety of zodasiran subcutaneous (SC) injection in subjects 12 years of age and older with genetically or clinically diagnosed Homozygous familial hypercholesterolemia (HoFH). After completion of the double blind (DB) treatment period subjects will be eligible to continue in the optional open-label extension (OLE) period of the study. All placebo subjects who opt to continue will transition to active drug during the OLE Period.

* DelveInsight's Homozygous Familial Hypercholesterolemia Pipeline report depicts a robust space with 3+ active players working to develop 3+ pipeline therapies for Homozygous Familial Hypercholesterolemia treatment.
* The leading Homozygous Familial Hypercholesterolemia Companies such as Arrowhead Pharmaceuticals, Innovent Biologics, Akeso Biopharma, LIB Therapeutics LLC and others.
* Promising Homozygous Familial Hypercholesterolemia Therapies such as Lomitapide, Inclisiran, Zodasiran Injection, SHR-1918, Evinacumab, IBI306 , and others.

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The Homozygous Familial Hypercholesterolemia Pipeline Report provides disease overview, pipeline scenario and therapeutic assessment of the key pipeline therapies in this domain. The Homozygous Familial Hypercholesterolemia Pipeline Report also highlights the unmet needs with respect to the Homozygous Familial Hypercholesterolemia.

Homozygous Familial Hypercholesterolemia Overview

Homozygous familial hypercholesterolaemia (HoFH) is a rare and life-threatening disease originally characterized clinically by plasma cholesterol levels >13 mmol/L (>500 mg/dL), extensive xanthomas, and marked premature and progressive atherosclerotic cardiovascular disease (ACVD). Studies in cultured fibroblasts from these patients showed a severe defect in the ability to bind and internalize LDL particles, subsequently shown to be caused by mutations in both alleles of the gene encoding the LDL receptor (LDLR). Recent genetic insights, however, indicate that mutations in alleles of other genes, including APOB, PCSK9, and LDLRAP1, may be present in some individuals with HoFH.

Homozygous Familial Hypercholesterolemia Emerging Drugs Profile

* ARO-ANG 3: Arrowhead Pharmaceuticals

ARO-ANG3 is designed to reduce production of angiopoietin-like protein 3 (ANGPTL3), a liver synthesized inhibitor of lipoprotein lipase and endothelial lipase. ANGPTL3 inhibition has been shown to lower serum LDL, serum and liver triglyceride and has genetic validation as a novel target for cardiovascular disease. Currently, it is in Phase II stage of clinical trial evaluation to treat Homozygous Familial Hypercholesterolemia.

* Tafolecimab: Innovent Biologics

Tafolecimab injection, developed by Innovent, is an lgG2 fully human monoclonal antibody that can specifically bind to PCSK-9 and reduce LDL-C level by inhibiting PCSK-9-mediated low-density lipoprotein receptor (LDLR) endocytosis, subsequently enhancing the clearance of LDL-C, resulting in a reduction in LDL-C level.

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The Homozygous Familial Hypercholesterolemia Pipeline report provides insights into:-

* The report provides detailed insights about companies that are developing therapies for the treatment of Homozygous Familial Hypercholesterolemia with aggregate therapies developed by each company for the same.
* It accesses the Different therapeutic candidates segmented into early-stage, mid-stage, and late-stage of development for Homozygous Familial Hypercholesterolemia Treatment.
* Homozygous Familial Hypercholesterolemia Companies are involved in targeted therapeutics development with respective active and inactive (dormant or discontinued) projects.
* Homozygous Familial Hypercholesterolemia Drugs under development based on the stage of development, route of administration, target receptor, monotherapy or combination therapy, a different mechanism of action, and molecular type.
* Detailed analysis of collaborations (company-company collaborations and company-academia collaborations), licensing agreement and financing details for future advancement of the Homozygous Familial Hypercholesterolemia market.

Homozygous Familial Hypercholesterolemia Companies

Arrowhead Pharmaceuticals, Innovent Biologics, Akeso Biopharma, LIB Therapeutics LLC and others.

Homozygous 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,

* Inhalation
* Inhalation/Intravenous/Oral
* Intranasal
* Intravenous
* Intravenous/ Subcutaneous
* NA
* Oral
* Oral/intranasal/subcutaneous
* Parenteral
* Subcutaneous

Homozygous Familial Hypercholesterolemia Products have been categorized under various Molecule types such as,

* Antibody
* Antisense oligonucleotides
* Immunotherapy
* Monoclonal antibody
* Peptides
* Protein
* Recombinant protein
* Small molecule
* Stem Cell
* Vaccine

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Scope of the Homozygous Familial Hypercholesterolemia Pipeline Report

* Coverage- Global
* Homozygous Familial Hypercholesterolemia Companies- Arrowhead Pharmaceuticals, Innovent Biologics, Akeso Biopharma, LIB Therapeutics LLC and others.
* Homozygous Familial Hypercholesterolemia Therapies- Lomitapide, Inclisiran, Zodasiran Injection, SHR-1918, Evinacumab, IBI306 , and others.
* Homozygous Familial Hypercholesterolemia Therapeutic Assessment by Product Type: Mono, Combination, Mono/Combination
* Homozygous Familial Hypercholesterolemia Therapeutic Assessment by Clinical Stages: Discovery, Pre-clinical, Phase I, Phase II, Phase III

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Table of Contents

* Introduction
* Executive Summary
* Homozygous Familial Hypercholesterolemia: Overview
* Pipeline Therapeutics
* Therapeutic Assessment
* Homozygous Familial Hypercholesterolemia - DelveInsight's Analytical Perspective
* Late Stage Products (Phase III)
* Drug name: Company name
* Drug profiles in the detailed report.....
* Mid Stage Products (Phase II)
* ARO-ANG 3: Arrowhead Pharmaceuticals
* Drug profiles in the detailed report.....
* Early Stage Products (Phase I)
* Drug name: Company name
* Drug profiles in the detailed report.....
* Preclinical and Discovery Stage Products
* Drug name: Company name
* Drug profiles in the detailed report.....
* Inactive Products
* Homozygous Familial Hypercholesterolemia Key Companies
* Homozygous Familial Hypercholesterolemia Key Products
* Homozygous Familial Hypercholesterolemia- Unmet Needs
* Homozygous Familial Hypercholesterolemia- Market Drivers and Barriers
* Homozygous Familial Hypercholesterolemia- Future Perspectives and Conclusion
* Homozygous Familial Hypercholesterolemia Analyst Views
* Homozygous Familial Hypercholesterolemia Key Companies
* Appendix

About Us

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