Homozygous Familial Hypercholesterolemia Market Unlocking New Revenue Opportunities and Strategic Expansion by 2036 - DelveInsight | Arrowhead Pharma, Novartis, Alnylam Pharma, LIB Therapeutics

The 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-2036 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.

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Some of the key facts of the Homozygous Familial Hypercholesterolemia Market Report:

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The Homozygous Familial Hypercholesterolemia market size is anticipated to grow with a significant CAGR during the study period (2022-2036).

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In March 2026, Chiesi Global Rare Diseases announced that the US FDA approved Lomitapide (JUXTAPID) for pediatric patients with HoFH, expanding its indication to younger populations and addressing a significant unmet need in early disease management.

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In August 2025, Amgen reported that the US FDA expanded the label of Evolocumab (REPATHA) to include patients at increased cardiovascular risk and reinforced its use in genetic conditions such as HoFH, supporting broader clinical adoption.

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In September 2025, the US FDA expanded approval of Evinacumab (EVKEEZA) to include pediatric patients aged greater than or equal to 1 year with HoFH, highlighting its growing role as a key LDL-C lowering therapy independent of LDL receptor function.

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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.

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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).

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According to DelveInsight's estimates, the total diagnosed prevalent cases of HoFH in the 7MM were approximately 1,840 in 2025.

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According to estimates based on DelveInsight's epidemiology model, the total diagnosed prevalent cases of HoFH in the US were estimated to be approximately ~870 in 2025.

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Among the 7MM, the EU4 and the UK accounted for approximately 40% of the total diagnosed prevalent cases of HoFH in 2025, which is expected to increase further during the study period.

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According to DelveInsight's analysis in the US, nearly 80% of cases were reported due to mutation in the LDLR gene.

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Assessments as per DelveInsight's analysts showed that Germany accounted for the highest total diagnosed prevalent cases of HoFH in 2025 among EU4 and the UK.

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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

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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

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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

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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 2022 to 2036. 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 2022-2036 in the 7MM segmented into:

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Total Diagnosed Prevalent Cases of HoFH in the 7MM

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Mutation-specific Diagnosed Prevalent Cases of HoFHin the 7MM

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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

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Zodasiran (ARO-ANG3): Arrowhead Pharmaceuticals

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Lerodalcibep (LIB003) (LEROCHOL): LIB Therapeutics

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ARO-ANG3: Arrowhead Pharmaceutical

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LEQVIO (inclisiran/KJX839: Novartis/ Alnylam Pharmaceutical

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Lerodalcibep (LIB003): LIB Therapeutics

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Lomitapide: Amryt Pharma

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Inclisiran: Novartis

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Evinacumab: Ultragenyx Pharmaceutical

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ARO-ANG 3 Injection: Arrowhead Pharmaceuticals

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Alirocumab: Sanofi

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CER-001: Cerenis Therapeutics

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lomitapide: Aegerion Pharmaceuticals

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ALN-PCSSC: The Medicines Company

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AK102: Akeso

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AEGR-733: Aegerion Pharmaceuticals

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Homozygous Familial Hypercholesterolemia Market Strengths

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The clarity in disease understanding and pathogenesis has driven the development of novel pharmacological options like PCSK9 inhibitors, ANGPTL3 inhibitors, and MTP inhibitor

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Updated international diagnostic and clinical guidelines for FH and HoFH enable evidence-based therapeutic approaches and screening strategies for early identification

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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

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Preclinical studies have yielded gene therapy and CRISPR-based gene editing approaches; conducting further research and clinical trials may offer curative therapy.

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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.

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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

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Study Period: 2022-2036

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Coverage: 7MM [The United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom), and Japan]

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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

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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

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Homozygous Familial Hypercholesterolemia Therapeutic Assessment: Homozygous Familial Hypercholesterolemia current marketed and Homozygous Familial Hypercholesterolemia emerging therapies

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Homozygous Familial Hypercholesterolemia Market Dynamics: Homozygous Familial Hypercholesterolemia market drivers and Homozygous Familial Hypercholesterolemia market barriers

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Competitive Intelligence Analysis: SWOT analysis, PESTLE analysis, Porter's five forces, BCG Matrix, Market entry strategies

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Homozygous Familial Hypercholesterolemia Unmet Needs, KOL's views, Analyst's views, Homozygous Familial Hypercholesterolemia Market Access and Reimbursement

To know more about Homozygous Familial Hypercholesterolemia companies working in the treatment market, visit @ Homozygous Familial Hypercholesterolemia Clinical Trials and Treatments [https://www.delveinsight.com/sample-request/homozygous-familial-hypercholesterolemia-market?utm_source=naver&utm_medium=promotion&utm_campaign=gpr]

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 (2022-2036)

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.

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