Homozygous Familial Hypercholesterolemia Market to Reach New Heights in Growth by 2034, DelveInsight Predicts
Key Homozygous Familial Hypercholesterolemia Companies in the market include - Arrowhead Pharmaceutical, Novartis, Alnylam Pharmaceutical, LIB Therapeutics, Amryt Pharma, Novartis, Ultragenyx Pharmaceutical, Arrowhead Pharmaceuticals, Sanofi, Cerenis Therapeutics, Aegerion Pharmaceuticals, The Medicines Company, Akeso, Aegerion Pharmaceuticals, and others.
DelveInsight's 'Homozygous Familial Hypercholesterolemia Market Insights, Epidemiology, and Market Forecast-2034″ report offers an in-depth understanding of the Homozygous Familial Hypercholesterolemia, historical and forecasted epidemiology as well as the Homozygous Familial Hypercholesterolemia market trends in the United States, EU4 (Germany, Spain, Italy, France) the United Kingdom and Japan.
To Know in detail about the Homozygous Familial Hypercholesterolemia market outlook, drug uptake, treatment scenario and epidemiology trends, Click here; Homozygous Familial Hypercholesterolemia Market Forecast
Some of the key facts of the Homozygous Familial Hypercholesterolemia Market Report:
The Homozygous Familial Hypercholesterolemia market size was valued approximately USD 108 million in 2022 and is anticipated to grow with a significant CAGR during the study period (2020-2034)
In March 2025, Esperion (NASDAQ: ESPR) has announced that, following discussions with the U.S. Food and Drug Administration (FDA), it has received regulatory alignment to proceed with Phase 3 clinical trials of bempedoic acid—both as a standalone therapy and in combination with ezetimibe—in pediatric patients with heterozygous and homozygous familial hypercholesterolemia (HeFH and HoFH). The company intends to launch these trials later this year. Notably, the FDA has previously granted orphan drug designation to bempedoic acid for the treatment of HoFH.
In January 2025, LIB Therapeutics Inc. (LIB), a privately held, late-stage biopharmaceutical company developing Lerodalcibep—a next-generation PCSK9 inhibitor—announced that results from its Phase 3 LIBerate-HoFH trial have been published in The Lancet Diabetes & Endocrinology (Raal, F.J. et al., 2025). The study evaluated Lerodalcibep in a globally diverse population with homozygous familial hypercholesterolemia (HoFH).
DelveInsight's 2022 analysis indicates that there were approximately 2,845 diagnosed prevalent cases of HoFH in the 7MM. This number is projected to rise significantly by 2034, with a notable CAGR throughout the study period (2020–2034).
Estimates from DelveInsight's epidemiology model suggest that there were around 1,349 diagnosed prevalent cases of HoFH in the US in 2022. This number is projected to increase by 2034 over the study period (2020–2034).
In the 7MM, the EU4 and the UK accounted for about 38% of the total diagnosed prevalent cases of HoFH in 2022, with expectations for this figure to rise further during the study period.
According to DelveInsight's analysis, the United States alone represented approximately 47% of all diagnosed prevalent cases of HoFH among the 7MM in 2022.
Key Homozygous Familial Hypercholesterolemia Companies: Arrowhead Pharmaceutical, Novartis, Alnylam Pharmaceutical, LIB Therapeutics, Amryt Pharma, Novartis, Ultragenyx Pharmaceutical, Arrowhead Pharmaceuticals, Sanofi, Cerenis Therapeutics, Aegerion Pharmaceuticals, The Medicines Company, Akeso, Aegerion Pharmaceuticals, and others
Key Homozygous Familial Hypercholesterolemia Therapies: ARO-ANG3, LEQVIO (inclisiran/KJX839), Lerodalcibep (LIB003), Lomitapide, Inclisiran, Evinacumab, ARO-ANG 3 Injection, Alirocumab, CER-001, lomitapide, ALN-PCSSC, AK102, AEGR-733, and others
The Homozygous Familial Hypercholesterolemia epidemiology based on mutation-specific cases analyzed that a mutation in the LDLR gene accounted for the highest diagnosed prevalent cases in the US
The Homozygous Familial Hypercholesterolemia market is expected to surge due to the disease's increasing prevalence and awareness during the forecast period. Furthermore, launching various multiple-stage Homozygous Familial Hypercholesterolemia pipeline products will significantly revolutionize the Homozygous Familial Hypercholesterolemia market dynamics.
Homozygous Familial Hypercholesterolemia Overview
Homozygous Familial Hypercholesterolemia (HoFH) is a rare and severe genetic disorder characterized by extremely high levels of low-density lipoprotein cholesterol (LDL-C) from birth. It is caused by mutations in both alleles of genes involved in the clearance of LDL-C from the bloodstream, typically the LDL receptor gene. This condition leads to early and aggressive atherosclerosis, increasing the risk of heart attacks, strokes, and other cardiovascular diseases at a young age.
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Homozygous Familial Hypercholesterolemia Epidemiology
The epidemiology section provides insights into the historical, current, and forecasted epidemiology trends in the seven major countries (7MM) from 2020 to 2034. It helps to recognize the causes of current and forecasted trends by exploring numerous studies and views of key opinion leaders. The epidemiology section also provides a detailed analysis of the diagnosed patient pool and future trends.
Homozygous Familial Hypercholesterolemia Epidemiology Segmentation:
The Homozygous Familial Hypercholesterolemia market report proffers epidemiological analysis for the study period 2020–2034 in the 7MM segmented into:
Total Diagnosed Prevalent Cases of HoFH in the 7MM
Mutation-specific Diagnosed Prevalent Cases of HoFHin the 7MM
Download the report to understand which factors are driving Homozygous Familial Hypercholesterolemia epidemiology trends @ Homozygous Familial Hypercholesterolemia Epidemiology Forecast
Homozygous Familial Hypercholesterolemia Drugs Uptake and Pipeline Development Activities
The drugs uptake section focuses on the rate of uptake of the potential drugs recently launched in the Homozygous Familial Hypercholesterolemia market or expected to get launched during the study period. The analysis covers Homozygous Familial Hypercholesterolemia market uptake by drugs, patient uptake by therapies, and sales of each drug.
Moreover, the therapeutics assessment section helps understand the drugs with the most rapid uptake and the reasons behind the maximal use of the drugs. Additionally, it compares the drugs based on market share.
The report also covers the Homozygous Familial Hypercholesterolemia Pipeline Development Activities. It provides valuable insights about different therapeutic candidates in various stages and the key companies involved in developing targeted therapeutics. It also analyzes recent developments such as collaborations, acquisitions, mergers, licensing patent details, and other information for emerging therapies.
Homozygous Familial Hypercholesterolemia Therapies and Key Companies
Homozygous Familial Hypercholesterolemia Market Strengths
The clarity in disease understanding and pathogenesis has driven the development of novel pharmacological options like PCSK9 inhibitors, ANGPTL3 inhibitors, and MTP inhibitor
Updated international diagnostic and clinical guidelines for FH and HoFH enable evidence-based therapeutic approaches and screening strategies for early identification
Advances in research have led to the discovery of novel molecules like siRNA and recombinant fusion protein that may offer novel options to lower LDL significantly
Homozygous Familial Hypercholesterolemia Market Opportunities
Preclinical studies have yielded gene therapy and CRISPR-based gene editing approaches; conducting further research and clinical trials may offer curative therapy.
Current therapies do not impart sufficient cholesterol-lowering allowing pharma players to bring newer, more potent, and better LDL-C lowering therapies and prevent ASCVD in HoFH patients.
Advancements in healthcare technologies and digital health solutions can potentially enhance early detection and personalized management of HoFH.
Scope of the Homozygous Familial Hypercholesterolemia Market Report
Study Period: 2020–2034
Coverage: 7MM [The United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom), and Japan]
Key Homozygous Familial Hypercholesterolemia Companies: Arrowhead Pharmaceutical, Novartis, Alnylam Pharmaceutical, LIB Therapeutics, Amryt Pharma, Novartis, Ultragenyx Pharmaceutical, Arrowhead Pharmaceuticals, Sanofi, Cerenis Therapeutics, Aegerion Pharmaceuticals, The Medicines Company, Akeso, Aegerion Pharmaceuticals, and others
Key Homozygous Familial Hypercholesterolemia Therapies: ARO-ANG3, LEQVIO (inclisiran/KJX839), Lerodalcibep (LIB003), Lomitapide, Inclisiran, Evinacumab, ARO-ANG 3 Injection, Alirocumab, CER-001, lomitapide, ALN-PCSSC, AK102, AEGR-733, and others
Homozygous Familial Hypercholesterolemia Therapeutic Assessment: Homozygous Familial Hypercholesterolemia current marketed and Homozygous Familial Hypercholesterolemia emerging therapies
Homozygous Familial Hypercholesterolemia Market Dynamics: Homozygous Familial Hypercholesterolemia market drivers and Homozygous Familial Hypercholesterolemia market barriers
Competitive Intelligence Analysis: SWOT analysis, PESTLE analysis, Porter's five forces, BCG Matrix, Market entry strategies
Homozygous Familial Hypercholesterolemia Unmet Needs, KOL's views, Analyst's views, Homozygous Familial Hypercholesterolemia Market Access and Reimbursement
Table of Contents
1. Homozygous Familial Hypercholesterolemia Market Report Introduction
2. Executive Summary for Homozygous Familial Hypercholesterolemia
3. SWOT analysis of Homozygous Familial Hypercholesterolemia
4. Homozygous Familial Hypercholesterolemia Patient Share (%) Overview at a Glance
5. Homozygous Familial Hypercholesterolemia Market Overview at a Glance
6. Homozygous Familial Hypercholesterolemia Disease Background and Overview
7. Homozygous Familial Hypercholesterolemia Epidemiology and Patient Population
8. Country-Specific Patient Population of Homozygous Familial Hypercholesterolemia
9. Homozygous Familial Hypercholesterolemia Current Treatment and Medical Practices
10. Homozygous Familial Hypercholesterolemia Unmet Needs
11. Homozygous Familial Hypercholesterolemia Emerging Therapies
12. Homozygous Familial Hypercholesterolemia Market Outlook
13. Country-Wise Homozygous Familial Hypercholesterolemia Market Analysis (2020–2034)
14. Homozygous Familial Hypercholesterolemia Market Access and Reimbursement of Therapies
15. Homozygous Familial Hypercholesterolemia Market Drivers
16. Homozygous Familial Hypercholesterolemia Market Barriers
17. Homozygous Familial Hypercholesterolemia Appendix
18. Homozygous Familial Hypercholesterolemia Report Methodology
19. DelveInsight Capabilities
20. Disclaimer
21. About DelveInsight
About DelveInsight
DelveInsight is a leading Healthcare Business Consultant, and Market Research firm focused exclusively on life sciences. It supports Pharma companies by providing comprehensive end-to-end solutions to improve their performance.
It also offers Healthcare Consulting Services, which benefits in market analysis to accelerate the business growth and overcome challenges with a practical approach.
Media Contact
Company Name: DelveInsight
Contact Person: Gaurav Bora
Email: Send Email
Phone: +14699457679
Address: 304 S. Jones Blvd #2432
City: Las Vegas
State: NV
Country: United States
Website: https://www.delveinsight.com/
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