Homozygous Familial Hypercholesterolemia Market Poised for Transformative Growth by 2034 - DelveInsight | Key Players: LIB Therapeutics, Innovent Biologics, Akeso Biopharma, Novartis, Alnylam Pharmaceuticals

Homozygous Familial Hypercholesterolemia (HoFH), a severe and ultra-rare genetic disorder, has long posed therapeutic challenges due to its aggressive cardiovascular complications and limited treatment options. However, with the development of novel lipid-lowering therapies-including PCSK9 inhibitors, ANGPTL3 inhibitors, and gene-based approaches-the market is witnessing a surge in innovation and hope for improved long-term outcomes.

DelveInsight's report, "Homozygous Familial Hypercholesterolemia - Market Insight, Epidemiology, and Market Forecast - 2034," offers a granular view of the HoFH market across the 7MM. The analysis includes in-depth epidemiology, patient segmentation, treatment gaps, and future outlook. As pioneering companies advance therapies that target the genetic and molecular root causes of HoFH, the market is expected to evolve toward personalized and more effective care solutions, with substantial growth anticipated through 2034.

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Some of the key insights of the Homozygous Familial Hypercholesterolemia Market Report:
• The homozygous familial hypercholesterolemia market is expected to witness significant growth at a notable CAGR throughout the forecast period, 2024-2034.
• According to DelveInsight's 2024 analysis, there were approximately 3,000 diagnosed prevalent cases of HoFH across the 7MM, projected to grow at a significant CAGR through 2034.
• The US accounted for nearly 50% of these cases in 2024, highlighting its dominant share in the HoFH patient population among the 7MM.
• In the US, around 1,250 cases were linked to LDLR mutations, followed by ~70 from APOB, ~55 from other mutations (LDLRAP1, APOE, SREBP2, STAP1), and ~15 from PCSK9.
• Japan held the second-highest prevalence, with about 400 diagnosed cases in 2024, which are expected to decline by 2034.
• PCSK9 inhibitors, including REPATHA® (evolocumab) and PRALUENT® (alirocumab), are approved third-line treatments for HoFH, targeting LDL-C reduction in severe and statin-intolerant cases.
• Despite therapeutic advances, achieving optimal LDL-C reduction in HoFH remains difficult, emphasizing the need for more effective, better-tolerated treatments to prevent ASCVD in these patients.
• Esperion (NASDAQ: ESPR) announced in March 2025 that it has secured FDA alignment for initiating Phase III studies of bempedoic acid, alone and with ezetimibe, in pediatric patients with heterozygous and homozygous familial hypercholesterolemia (HeFH and HoFH). Phase III trials are expected to begin later this year. Bempedoic acid previously received orphan drug designation for HoFH.
• LIB Therapeutics Inc. submitted a Biologics License Application (BLA) to the FDA in December 2024 for Lerodalcibep, a novel third-generation PCSK9 inhibitor aimed at reducing LDL-C in patients with ASCVD, primary hyperlipidemia, and HeFH/HoFH. Lerodalcibep is a more patient-friendly, once-monthly, subcutaneous injection.
• In January 2025, LIB Therapeutics announced the publication of its Phase III LIBerate-HoFH study, which evaluates Lerodalcibep in a global HoFH patient population.
• In February 2025, LIB Therapeutics revealed that the FDA has accepted the BLA for Lerodalcibep for the treatment of LDL-C reduction in ASCVD and HeFH/HoFH patients, including those aged 10 and older.
• Emerging therapies for homozygous familial hypercholesterolemia include ARO-ANG 3, Tafolecimab, AK102, Lerodalcibep, LEQVIO, and others.
• Key companies involved in the treatment of LIB Therapeutics, Innovent Biologics, Akeso Biopharma, Novartis, Alnylam Pharmaceuticals, Arrowhead Pharmaceuticals, and others.

To know in detail about the homozygous familial hypercholesterolemia market outlook, drug uptake, treatment scenario, and epidemiology trends, click here: https://www.delveinsight.com/report-store/food-allergy-market-insights?utm_source=openpr&utm_medium=pressrelease&utm_campaign=jpr

Homozygous Familial Hypercholesterolemia Overview
Homozygous familial hypercholesterolemia (HoFH) is a rare, severe genetic disorder affecting lipid metabolism, resulting in extremely high LDL cholesterol (LDL-C) levels. It is inherited in an autosomal recessive pattern and caused by mutations in both copies of the LDLR gene, which impairs the removal of LDL-C from the bloodstream. This leads to cholesterol accumulation in arteries and tissues, causing early-onset, aggressive atherosclerotic cardiovascular disease.
Patients typically present with LDL-C levels over 13 mmol/L (>500 mg/dL), along with corneal arcus, xanthomas, and early cardiovascular complications. The disease is diagnosed early and, if untreated, can cause severe cardiac issues by mid-life. In the US, cholesterol levels are measured in mg/dL, while in Europe, they are measured in mmol/L. Without treatment, HoFH significantly increases the risk of heart attacks and strokes due to rapid arterial narrowing and blockages.

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Homozygous Familial Hypercholesterolemia Epidemiology
The epidemiology section offers an overview of historical, current, and projected trends in the seven major countries (7MM) from 2020 to 2034. It helps identify the factors influencing these trends by examining various studies and perspectives from key opinion leaders. Additionally, the section provides an in-depth analysis of the diagnosed patient population 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 HoFH in the 7MM

Homozygous Familial Hypercholesterolemia Drugs Uptake and Pipeline Development Activities
The drug uptake section examines the adoption rates of newly launched and upcoming homozygous familial hypercholesterolemia drugs over the study period. It analyzes the uptake of these treatments, evaluating how patients adopt these therapies and the sales performance of each drug. This section provides a comprehensive overview of the factors that influence the acceptance and success of homozygous familial hypercholesterolemia treatments on the market.

In addition, the therapeutics assessment section highlights the homozygous familial hypercholesterolemia drugs that have experienced the fastest uptake. It delves into the key drivers behind their widespread use and provides a comparison of market share among these drugs. This section helps identify which therapies are gaining traction and the reasons behind their rapid adoption.

The report further explores the homozygous familial hypercholesterolemia pipeline, providing insights into therapeutic candidates at different stages of development. It identifies the key companies involved in creating targeted homozygous familial hypercholesterolemia treatments. The report also covers recent developments in the field, including collaborations, mergers, acquisitions, licensing agreements, and other significant updates on emerging therapies for Homozygous Familial Hypercholesterolemia.

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Homozygous Familial Hypercholesterolemia Market Outlook
Homozygous Familial Hypercholesterolemia (HoFH) presents a significant therapeutic challenge due to its rarity, genetic complexity, and resistance to standard lipid-lowering therapies. The disorder is primarily driven by biallelic mutations in the LDLR gene, found in over 90% of patients, and is associated with extremely elevated LDL-C levels (typically exceeding 400 mg/dL). As a result, the treatment landscape targets LDL-receptor pathways and complementary mechanisms to achieve adequate cholesterol control.

Initial treatment typically begins with high-intensity statins, such as simvastatin, rosuvastatin, or atorvastatin-many of which are available as cost-effective generics. Recently, CMP Pharma's ATORVALIQ (oral suspension formulation of atorvastatin) received FDA approval in June 2023, offering an alternative for patients with swallowing difficulties or pediatric populations.
For better outcomes, statins are often combined with ezetimibe, both of which rely on functional LDL receptors. However, due to the limited LDLR activity in HoFH, many patients require additional therapies.

• PCSK9 inhibitors such as REPATHA (evolocumab, Amgen) and PRALUENT (alirocumab, Regeneron/Sanofi) are now widely used in patients who respond poorly to traditional therapies or have very high ASCVD risk. These agents increase LDLR recycling and clearance of LDL-C and have been approved in the US and globally since 2015.

• EVKEEZA (evinacumab, Regeneron), approved in 2021 by the US FDA and EMA, offers a unique mechanism as an ANGPTL3 inhibitor, effective even in patients with minimal or no LDLR function. It is approved for both adult and pediatric patients aged five years and older.

• Lomitapide, marketed as JUXTAPID/LOJUXTA by Chiesi Farmaceutici/Recordati, is a microsomal triglyceride transfer protein (MTP) inhibitor approved in the US (2012), EU (2013), and Japan (2016). It bypasses LDLR pathways by directly inhibiting ApoB lipoprotein assembly and secretion, significantly lowering LDL-C levels in patients with defective LDLR function.

The HoFH treatment market is being shaped by increasing awareness, improved genetic screening, and a growing arsenal of innovative lipid-lowering agents. The emergence of monoclonal antibodies and oral therapies targeting non-LDLR pathways offers promise for broader efficacy across the genetically diverse HoFH patient population. However, accessibility, cost, and long-term safety remain key concerns influencing therapy adoption and global market expansion.

Homozygous Familial Hypercholesterolemia Market Drivers
• Emergence of novel treatments like ANGPTL3 inhibitors and gene therapies addressing unmet needs in LDLR-deficient patients.
• Growth in genetic screening and awareness is leading to earlier and more accurate HoFH diagnosis.
Homozygous Familial Hypercholesterolemia Market Barriers
• Advanced therapies, especially monoclonal antibodies and MTP inhibitors, remain expensive and limit widespread access.
• As a rare disease, the small number of diagnosed HoFH patients can restrict commercial viability and investment incentives.

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: LIB Therapeutics, Innovent Biologics, Akeso Biopharma, Novartis, Alnylam Pharmaceuticals, Arrowhead Pharmaceuticals, and others.
• Key Homozygous Familial Hypercholesterolemia Therapies: ARO-ANG 3, Tafolecimab, AK102, Lerodalcibep, LEQVIO, and others.
• Homozygous Familial Hypercholesterolemia Therapeutic Assessment: Homozygous Familial Hypercholesterolemia currently 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

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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

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