Familial Hypercholesterolemia Market

• According to DelveInsight’s estimates, in 2023, there were approximately 672 thousand diagnosed prevalent cases of Familial Hypercholesterolemia in the 7MM. Of these, the US accounted for approximately 54% of the cases, EU4 and the UK countries accounted for around 36%, followed by Japan which represented nearly 10%.
• The Familial Hypercholesterolemia Market is set for steady growth, with a robust compound annual growth rate (CAGR) of 12% anticipated from 2024 to 2034. This expansion in the 7MM is driven by the introduction of innovative therapies such as Zodasiran, VERVE-101, as well as growing awareness and diagnosis, and advancements in treatment options.
• A major Familial Hypercholesterolemia Unmet Needs is the development of more effective treatments that address LDL-C reduction while minimizing long-term cardiovascular risks. Despite available therapies, many patients fail to achieve target cholesterol levels, particularly those with homozygous FH, indicating a need for innovative, personalized approaches to optimize lipid management and improve outcomes.
• FDA-approved Familial Hypercholesterolemia Drugs like NEXLETOL (Esperion Therapeutics), EVKEEZA (Regeneron Pharmaceuticals), and PRALUENT (Sanofi) for Familial Hypercholesterolemia indicate strong market potential. 
• A significant market barrier for Familial Hypercholesterolemia treatments is the underdiagnosis of the condition. Despite its genetic nature and clear cardiovascular risks, many individuals remain undiagnosed due to a lack of awareness, limited genetic screening programs, and variable clinical presentation, reducing the potential market size and hindering timely therapeutic intervention.

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DelveInsight’s “Familial Hypercholesterolemia Market Insights, Epidemiology, and Market Forecast – 2034” report delivers an in-depth understanding of Familial Hypercholesterolemia, historical and forecasted epidemiology, as well as the Familial Hypercholesterolemia market trends in the United States, EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan.

The Familial Hypercholesterolemia Treatment Market Report provides current treatment practices, emerging drugs, market share of individual therapies, and current and forecasted 7MM Familial Hypercholesterolemia market size from 2020 to 2034. The report also covers Familial Hypercholesterolemia treatment market practices/algorithms and unmet medical needs to curate the best opportunities and assess the market’s potential.

Familial Hypercholesterolemia Treatment Market: Understanding and Algorithm

Familial Hypercholesterolemia is an inherited disorder characterized by elevated LDL cholesterol (LDL-C) levels from birth, leading to early-onset heart attacks. FH exists in two forms: Heterozygous Familial Hypercholesterolemia (HeFH), the more common type, and Homozygous Familial Hypercholesterolemia (HoFH), a rare, severe variant. Children with FH often exhibit LDL-C levels exceeding 160 mg/dL, while adults typically have levels above 190 mg/dL, resulting in atherosclerosis over time. FH is caused by mutations in genes such as the LDL receptor (LDLR), Apolipoprotein B (Apo B), PCSK9, and other rare genes. Signs and symptoms include high cholesterol, xanthomas, and early cardiovascular events. 

Familial Hypercholesterolemia diagnosis

The diagnosis of Familial Hypercholesterolemia is based on family history, physical examination, and lipid profile analysis. FH is suspected in adults with fasting LDL-C ≥190 mg/dL and in children with LDL-C ≥160 mg/dL, after ruling out secondary causes such as hypothyroidism, nephrotic syndrome, and liver disease. Physical signs like xanthomas, corneal arcus, and xanthelasmas, especially before age 60, strongly suggest FH, particularly in HoFH. Genetic testing for mutations in the LDLR, ApoB, PCSK9, or LDLRAP1 genes confirms the diagnosis, especially in compound heterozygotes. A family history of high LDL-C and premature coronary heart disease is crucial for assessing FH risk.

 Further details related to country-based variations are provided in the report…

Familial Hypercholesterolemia treatment

The treatment of Familial Hypercholesterolemia involves a combination of lipid-lowering therapies. Statins, ranging from low to high intensity, are the first-line treatment and are often combined with Ezetimibe for enhanced LDL-C reduction. Additional options include PCSK9 inhibitors such as REPATHA (evolocumab) and PRALUENT (alirocumab), and the MTP inhibitor JUXTAPID/LOJUXTA (Lomitapide) for more severe cases. NEXLETOL/NILEMDO (Bempedoic acid) and the combination of bempedoic acid with NEXLIZET/NUSTENDI (ezetimibe) are newer treatments. Other therapies include bile acid sequestrants, fibrates, stanol esters, and lipoprotein apheresis for severe, resistant cases. 

Familial Hypercholesterolemia Epidemiology

As the market is derived using a patient-based model, the Familial Hypercholesterolemia epidemiology chapter in the report provides historical as well as forecasted epidemiology segmented by total prevalent cases of Familial Hypercholesterolemia, total diagnosed prevalent cases of Familial Hypercholesterolemia, age-specific diagnosed prevalent cases of Familial Hypercholesterolemia, and mutation-specific diagnosed prevalent cases of Familial Hypercholesterolemia in the 7MM covering the United States, EU4 countries (Germany, France, Italy, and Spain) and the United Kingdom, and Japan from 2020 to 2034. 

• According to DelveInsight’s epidemiology model, in the US, the total diagnosed prevalent cases of Familial Hypercholesterolemia were approximately 361 thousand in 2023. This number is anticipated to rise during the forecast period (2024-2034), driven by increased awareness and screening, along with advancements in genetic testing.
• Among the EU4 and the UK, France accounted for the highest number of diagnosed prevalent cases of Familial Hypercholesterolemia, with approximately 58 thousand cases in 2023, followed by the UK with approximately 54 thousand cases, and Germany with nearly 53 thousand cases.
• Among the age-specific diagnosed prevalent cases of Familial Hypercholesterolemia in the US in 2023, the highest cases were observed in the 50–59 years age group with approximately 94 thousand whereas the lowest cases were for the 30–39 years of age group with nearly 27 thousand.
• In 2023, Japan had the second highest number of diagnosed prevalent cases of Familial Hypercholesterolemia among the 7MM, totaling approximately 69 thousand cases.
• In 2023, mutation-specific categories included LDL receptor (LDLR), Apolipoprotein B (Apo B), Proprotein Convertase Subtilisin/Kexin 9 (PCSK9), and other rare mutations such as SREBP2, STAP1 genes, and LDLRAP1 gene. Notably, LDL receptor (LDLR) mutations had the highest prevalence with around 61 thousand cases reported in Japan.

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Familial Hypercholesterolemia Marketed Drugs

• EVKEEZA (evinacumab-dgnb): Regeneron Pharmaceuticals

EVKEEZA (evinacumab) is a fully human monoclonal antibody that inhibits angiopoietin-like 3 (ANGPTL3), a protein crucial for lipid metabolism. Regeneron scientists discovered the angiopoietin gene family over 20 years ago. Research published in the New England Journal of Medicine (NEJM) in 2017 by the Regeneron Genetics Center revealed that patients with non-functional ANGPTL3 genes, due to "loss-of-function mutations," exhibit significantly lower levels of key blood lipids, including LDL cholesterol (LDL-C), correlating with a markedly reduced risk of coronary artery disease. Regeneron developed EVKEEZA and received FDA approval in February 2021. Additionally, EVKEEZA was approved in the European Economic Area in June 2021, with Regeneron collaborating with Ultragenyx for its commercialization outside the United States. 

• PRALUENT (alirocumab): Sanofi/ Regeneron Pharmaceuticals

PRALUENT (alirocumab) functions by inhibiting the binding of PCSK9 to LDL receptors, thereby enhancing the availability of LDL receptors on liver cell surfaces to facilitate the clearance of LDL cholesterol (LDL-C) from the bloodstream. Developed by Regeneron and Sanofi under a global collaboration agreement, Praluent received FDA approval in July 2015 as the first proprotein convertase subtilisin kexin 9 (PCSK9) inhibitor for the treatment of heterozygous familial hypercholesterolemia (HeFH). Subsequently, in September 2015, the EMA approved PRALUENT for treating primary hypercholesterolemia, encompassing both heterozygous familial and non-familial forms. In July 2016, Japan's Ministry of Health, Labor and Welfare granted marketing and manufacturing authorization for Praluent, indicating its use for adults with hypercholesterolemia at high cardiovascular risk, particularly when statin therapy is inadequate. 

• REPATHA (evolocumab): Amgen 

REPATHA (evolocumab) is a human monoclonal immunoglobulin G2 (IgG2) that targets proprotein convertase subtilisin kexin 9 (PCSK9), with a molecular weight of approximately 144 kDa. Produced in genetically engineered Chinese hamster ovary cells, REPATHA binds to PCSK9, inhibiting its attachment to low-density lipoprotein receptors (LDLR) and preventing LDLR degradation. This mechanism enhances LDLR recycling, leading to lower LDL cholesterol (LDL-C) levels. The Japanese Ministry of Health approved REPATHA for familial hypercholesterolemia (FH) in January 2016. The FDA granted approval in August 2015 for adults with HeFH and as an adjunct treatment for HoFH. The European Medicines Agency also authorized its use for adults with primary hypercholesterolemia and adolescents aged 12 and older with HoFH. In September 2021, the FDA expanded the indication to include pediatric patients aged 10 and older with HeFH, supported by the HAUSER-RCT Phase 3b study, which showed a 38% mean LDL-C reduction compared to placebo.

Note: Further marketed drugs and their details will be provided in the report…

Familial Hypercholesterolemia Emerging Drugs

• VERVE-101: Verve Therapeutics

VERVE-101 has demonstrated promising interim data, showcasing the first human proof-of-concept for in vivo base editing in patients with HeFH. Following a single infusion of VERVE-101 at potentially therapeutic doses, reductions in low-density lipoprotein cholesterol (LDL-C) of up to 55% and blood PCSK9 protein levels by as much as 84% were observed. The safety profile of VERVE-101 supports its continued development, with ongoing enrollment in the 0.45 mg/kg and 0.6 mg/kg cohorts. Additionally, plans are in place to initiate an expansion cohort in 2024. In October 2023, the US FDA lifted the clinical hold and cleared the Investigational New Drug (IND) application, allowing for a clinical trial in the United States to further evaluate VERVE-101 for treating HeFH. 

• Lerodalcibep: LIB Therapeutics

Lerodalcibep is an innovative, next-generation small-binding protein serving as an alternative to monoclonal antibodies in the PCSK9 inhibitor class, developed by LIB Therapeutics to address the limitations of existing LDL-lowering therapies. In the Phase III LIBerate-HeFH trial, monthly administration of lerodalcibep achieved both co-primary endpoints, demonstrating statistically significant (p < 0.0001) reductions in mean LDL cholesterol of 58.6% at Week 24 and 65.0% at the mean of Weeks 22 and 24 within the intent-to-treat population. Additionally, 68% of participants receiving lerodalcibep reached a reduction in LDL-C of at least 50% and met the recommended LDL-C targets set by the European Society of Cardiology (ESC) during the study. LIB Therapeutics has completed the final two registration-enabling Phase III trials and is now preparing a biologics license application (BLA) for lerodalcibep, with plans for regulatory submission.

• Zodasiran: Arrowhead Pharmaceuticals

Zodasiran, also known as ARO-ANG3, is engineered to inhibit the production of ANGPTL3, a liver-derived protein that serves as an inhibitor of lipoprotein lipase and endothelial lipase. The inhibition of ANGPTL3 has demonstrated significant potential in lowering serum low-density lipoprotein (LDL) levels and reducing serum and liver triglycerides, with genetic evidence supporting its role as a promising target for cardiovascular disease intervention. Notably, clinical findings indicate that Zodasiran has achieved mean reductions in LDL-C of 44-48% when administered alongside standard care. Phase III planning for the drug is currently underway.

Note: Further emerging therapies and their detailed assessment will be provided in the final report.

Familial Hypercholesterolemia Drugs Market Insights

The treatment of Familial Hypercholesterolemia encompasses several drug classes, each targeting lipid metabolism to effectively lower LDL cholesterol levels. Statins, including low, medium, and high-intensity variants, serve as first-line therapies by inhibiting HMG-CoA reductase, thereby reducing cholesterol synthesis in the liver. Ezetimibe complements statin therapy by inhibiting intestinal absorption of cholesterol. PCSK9 inhibitors, such as evolocumab and alirocumab, significantly enhance the clearance of LDL by preventing PCSK9 from degrading LDL receptors in liver cells. Additionally, bempedoic acid, a novel agent, works by inhibiting ATP-citrate lyase to further lower LDL-C levels. Other adjunct therapies include bile acid sequestrants, fibrates, and lipoprotein apheresis, which may be employed in severe cases to achieve optimal lipid control. This multi-faceted approach allows for personalized treatment regimens to address the unique challenges presented by FH. 

Familial Hypercholesterolemia Market Outlook

The Familial Hypercholesterolemia Market features a diverse array of treatment options across various drug classes, including statins (e.g., Atorvastatin, Rosuvastatin) either as monotherapy or in combination with Ezetimibe (Zetia). Additional therapeutic agents include MTP inhibitors such as Lomitapide (Juxtapid), PCSK9 inhibitors like PRALUENT (Alirocumab) and REPATHA (Evolocumab), and bempedoic acid formulations, including Bempedoic acid (Nexletol/Nilemdo) and NEXLIZET/NUSTENDI (Bempedoic acid/Ezetimibe). For advanced cases, lipoprotein apheresis is employed, while liver transplantation is considered in rare, severe instances. Statins remain the cornerstone of FH management by inhibiting HMG-CoA reductase, crucial for hepatic cholesterol synthesis, which reduces LDL production and promotes LDL receptor upregulation. However, in patients with homozygous FH (HoFH), the efficacy of statins is diminished due to the compensatory degradation of LDL receptors. Despite this limitation, statins continue to demonstrate cardiovascular mortality reduction benefits, even in receptor-negative individuals, highlighting their importance in FH treatment strategies.

Familial Hypercholesterolemia Drugs Uptake

This section focuses on the uptake rate of potential drugs expected to be launched in the market during 2020–2034. For example, Zodasiran is expected to enter the US market in 20XX and is projected to have a XX uptake during the forecast period.

Familial Hypercholesterolemia Pipeline Development Activities

The Familial Hypercholesterolemia therapeutics market report provides insights into different therapeutic candidates in Phase III, Phase II, and Phase I. It also analyzes key Familial Hypercholesterolemia Companies involved in developing targeted therapeutics. 

Pipeline development activities

The Familial Hypercholesterolemia therapeutics market report covers information on collaborations, acquisitions and mergers, licensing, and patent details for emerging therapies for Familial Hypercholesterolemia.

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KOL Views

To keep up with current market trends, we take KOLs and SMEs’ opinions working in the domain through primary research to fill the data gaps and validate our secondary research. Industry Experts contacted for insights on Familial Hypercholesterolemia evolving treatment landscape, patient reliance on conventional therapies, patient therapy switching acceptability, and drug uptake, along with challenges related to accessibility, including Medical/scientific writers, Medical Professionals, Professors, Directors, and Others.

DelveInsight’s analysts connected with 50+ KOLs to gather insights; however, interviews were conducted with 15+ KOLs in the 7MM. Centers like the Department of Family Medicine, Tulane University School of Medicine, New Orleans, USA; University of California, Los Angeles, CA, United States, among others, were contacted. Their opinion helps understand and validate current and emerging therapy treatment patterns or Familial Hypercholesterolemia market trends. This will support the clients in potential upcoming novel treatments by identifying the overall scenario of the market and the unmet needs.

Physician’s View

As per the KOLs from the US, apart from PCSK9 inhibitors, another potential class called ANGPTL3 antibody has emerged and is proving to be beneficial in the total FH population with its target towards the orphan HoHF.

Familial Hypercholesterolemia Drugs Market: Qualitative Analysis

We perform Qualitative and market Intelligence analysis using various approaches, such as SWOT and Conjoint Analysis. In the SWOT analysis, strengths, weaknesses, opportunities, and threats in terms of disease diagnosis, patient awareness, patient burden, competitive landscape, cost-effectiveness, and geographical accessibility of therapies are provided. These pointers are based on the Analyst’s discretion and assessment of the patient burden, cost analysis, and existing and evolving Familial Hypercholesterolemia treatment market landscape.

Conjoint Analysis analyzes multiple emerging therapies based on relevant attributes such as safety, efficacy, frequency of administration, route of administration, and order of entry. Scoring is given based on these parameters to analyze the effectiveness of therapy. To analyze the effectiveness of these therapies, have calculated their attributed analysis by giving them scores based on their ability to improve atrial and ventricular dimension/function and ability to regulate heart rate. 

Further, the therapies’ safety is evaluated wherein the adverse events are majorly observed, and it sets a clear understanding of the side effects posed by the drug in the trials, which directly affects the safety of the molecule in the upcoming trials. It sets a clear understanding of the side effects posed by the drug in the trials. In addition, the scoring is also based on the route of administration, order of entry and designation, probability of success, and the addressable patient pool for each therapy. According to these parameters, the final weightage score and the ranking of the emerging therapies are decided. 

Familial Hypercholesterolemia Therapeutics Market Access and Reimbursement

Esperion is also offering a Co-Pay Card for NEXLETOL and NEXLIZET, which are the most recent approvals for treating HeFH patients. With this card, patients may pay as little as USD 10 per fill for up to a 3-month supply each time the patients fill their prescriptions. A patient may be eligible for the NEXLETOL and NEXLIZET Co-Pay Card if they are at least 18 years of age, have a valid prescription for the drugs, possess a commercial prescription drug insurance, are a resident of the US, and are not enrolled in any state-, federal-, or government-funded healthcare program. 

Further details will be provided in the report.

Familial Hypercholesterolemia Treatment Market Report Scope

• The Familial Hypercholesterolemia treatment market report covers a segment of key events, an executive summary, and a descriptive overview explaining its causes, signs and symptoms, pathogenesis, and currently available therapies.
• Comprehensive insight into the epidemiology segments and forecasts, the future growth potential of diagnosis rate, disease progression, and treatment guidelines have been provided.
• Additionally, an all-inclusive account of the current and emerging therapies and the elaborative profiles of late-stage and prominent therapies will impact the current Familial Hypercholesterolemia treatment market landscape.
• A detailed review of the Familial Hypercholesterolemia treatment market, historical and forecasted Familial Hypercholesterolemia treatment market size, Familial Hypercholesterolemia drugs market share by therapies, detailed assumptions, and rationale behind our approach is included in the report, covering the 7MM drug outreach.
• The Familial Hypercholesterolemia treatment market report provides an edge while developing business strategies by understanding trends through SWOT analysis and expert insights/KOL views, patient journey, and treatment preferences that help shape and drive the 7MM Familial Hypercholesterolemia Drugs Market.

Familial Hypercholesterolemia Treatment Market Report Insights

• Patient-based Familial Hypercholesterolemia Market Forecasting
• Therapeutic Approaches
• Familial Hypercholesterolemia Pipeline Analysis
• Familial Hypercholesterolemia Market Size and Trends
• Existing and Future Familial Hypercholesterolemia Drugs Market Opportunity 

Familial Hypercholesterolemia Treatment Market Report Key Strengths

• 11 years Familial Hypercholesterolemia Market Forecast
• The 7MM Coverage 
• Familial Hypercholesterolemia  Epidemiology Segmentation
• Key Cross Competition 
• Conjoint analysis
• Familial Hypercholesterolemia Drugs Uptake
• Key Familial Hypercholesterolemia Market Forecast Assumptions

Familial Hypercholesterolemia Treatment Market Report Assessment

• Current Familial Hypercholesterolemia Treatment Market Practices
• Familial Hypercholesterolemia Unmet Needs
• Familial Hypercholesterolemia Pipeline Product Profiles
• Familial Hypercholesterolemia Drugs Market Attractiveness
• Qualitative Analysis (SWOT and Conjoint Analysis)

Key Questions

Familial Hypercholesterolemia Treatment Market Insights

• What was the total Familial Hypercholesterolemia Treatment Market Size, the Familial Hypercholesterolemia Market Size by therapies, and Familial Hypercholesterolemia Drugs Market Share (%) distribution in 2020, and what would it look like by 2034? What are the contributing factors for this growth?
• How will Zodasiran affect the treatment paradigm of Familial Hypercholesterolemia?
• How will EVKEEZA compete with the upcoming therapies?
• Which drug is going to be the largest contributor by 2034?
• What are the pricing variations among different geographies for approved and marketed therapies?
• How would future opportunities affect the Familial Hypercholesterolemia market dynamics and subsequent analysis of the associated trends?

Familial Hypercholesterolemia Epidemiology Insights

• What are the disease risks, burdens, and Familial Hypercholesterolemia Unmet Needs? What will be the growth opportunities across the 7MM with respect to the patient population pertaining to Familial Hypercholesterolemia?
• What is the historical and forecasted Familial Hypercholesterolemia patient pool in the United States, EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan?
• Out of the countries mentioned above, which country would have the highest diagnosed prevalent Familial Hypercholesterolemia population during the forecast period (2024–2034)?
• What factors are contributing to the growth of Familial Hypercholesterolemia cases?

Current Familial Hypercholesterolemia Treatment MarketScenario, Marketed Drugs, and Emerging Therapies

• What are the current options for the treatment of Familial Hypercholesterolemia? What are the current clinical and treatment guidelines for treating Familial Hypercholesterolemia?
• How many companies are developing therapies for the treatment of Familial Hypercholesterolemia?
• How many emerging therapies are in the mid-stage and late stage of development for treating Familial Hypercholesterolemia?
• What are the recent novel therapies, targets, mechanisms of action, and technologies developed to overcome the limitations of existing therapies? 
• What is the cost burden of current treatment on the patient?
• Patient acceptability in terms of preferred treatment options as per real-world scenarios?
• What are the accessibility issues of approved therapy in the US? 
• What is the 7MM historical and forecasted Familial Hypercholesterolemia Treatment Market?

Reasons to Buy

• The Familial Hypercholesterolemia treatment market report will help develop business strategies by understanding the latest trends and changing treatment dynamics driving the Familial Hypercholesterolemia Drugs Market.
• Insights on patient burden/disease Familial Hypercholesterolemia Prevalence, evolution in diagnosis, and factors contributing to the change in the epidemiology of the disease during the forecast years.
• Understand the existing Familial Hypercholesterolemia Drugs Market opportunities in varying geographies and the growth potential over the coming years.
• The distribution of historical and current patient share is based on real-world prescription data in the US, EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan.
• Identifying upcoming solid players in the Familial Hypercholesterolemia Drugs Market will help devise strategies to help get ahead of competitors.
• Detailed analysis and ranking of class-wise potential current and emerging therapies under the conjoint analysis section to provide visibility around leading classes.
• Highlights of Access and Reimbursement policies for Familial Hypercholesterolemia, barriers to accessibility of approved therapy, and patient assistance programs.
• To understand Key Opinion Leaders’ perspectives around the accessibility, acceptability, and compliance-related challenges of existing treatment to overcome barriers in the future.
• Detailed insights on the unmet needs of the existing Familial Hypercholesterolemia Drugs Market so that the upcoming players can strengthen their development and launch strategy.

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