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Familial Hypercholesterolemia - Market Insight, Epidemiology and Market Forecast -2030

  • ID: 5017396
  • Drug Pipelines
  • April 2020
  • Region: Global
  • 150 pages
  • DelveInsight

FEATURED COMPANIES

  • Amgen
  • Arrowhead Pharmaceutica
  • Esperion Therapeutics
  • LIB Therapeutics
  • Madrigal Pharmaceuticals
  • NeuroBo Pharmaceuticals
  • MORE
‘Familial Hypercholesterolemia (FH) -Market Insights, Epidemiology and Market Forecast- 2030' report delivers an in-depth understanding of the Familial Hypercholesterolemia (FH), historical and forecasted epidemiology as well as the Familial Hypercholesterolemia (FH) market trends in the United States, EU5 (Germany, Spain, Italy, France, and United Kingdom) and Japan.

The Familial Hypercholesterolemia (FH) market report provides current treatment practices, emerging drugs, and market share of the individual therapies, current and forecasted 7MM Familial Hypercholesterolemia (FH) market size from 2017 to 2030. The report also covers current Familial Hypercholesterolemia (FH) treatment practice/algorithm, market drivers, market barriers and unmet medical needs to curate the best of the opportunities and assesses the underlying potential of the market.

Geography Covered
  • The United States
  • EU5 (Germany, France, Italy, Spain, and the United Kingdom)
  • Japan
Study Period: 2017-2030

Familial Hypercholesterolemia (FH) Disease Understanding and Treatment Algorithm

Familial Hypercholesterolemia (FH) Overview

Familial Hypercholesterolemia (FH) is a genetic and hereditary disorder, which leads to a high level of LDL (bad) cholesterol. The condition begins at birth and can cause heart attacks at an early age.

FH is an autosomal-dominant disorder associated with mutations in the LDL receptor gene resulting in elevated plasma low-density lipoprotein cholesterol levels and premature atherosclerotic cardiovascular disease (ASCVD). FH is significantly under-recognized with as many as 1 in 300 having the heterozygous form and one in a million having the homozygous form of the disease. Patients with FH are characterized by a decreased clearance of LDL from the circulation and an increase in LDL synthesis, with changes in homozygotes being more marked than in heterozygotes, consistent with a gene dosage effect.

Heterozygous FH (HeFH) (mutation in one allele) is related with plasma LDL-C levels >190 mg/dL, whereas homozygous FH (HoFH) (mutation in both alleles) is associated with plasma LDL-C levels >500 mg/dL. As a result, there is a 20-fold increase in the risk of premature coronary heart disease (CHD) in untreated patients compared to control. HeFH patients usually develop CHD, without treatment, before age 55 and 60 for men and women, respectively. HoFH patients, however, develop CHD very early in life and can die before age 20 if untreated.

Familial Hypercholesterolemia (FH) Diagnosis

In general, an estimated 20 million people worldwide have FH. Despite the high prevalence and increased risk of premature ASCVD in untreated patients, less than 1% are diagnosed with FH worldwide.

Family history, physical examination and a lipid profile are essential to establishing a diagnosis of FH. FH should be suspected with fasting LDL-C ≥190 mg/dL in adults and ≥160 mg/dL in children if secondary causes of hypercholesterolemia, such as hypothyroidism, nephritic syndrome, and liver disease are ruled out. The presence of xanthomas, corneal arcus, and xanthelasmas before the age of 60 are highly suggestive of FH, more specifically homozygous FH (HoFH), although sitosterolemia should be ruled out as a cause. Individuals affected by homozygous FH possess two mutant alleles at the LDLR, ApoB, PCSK9, or LDLRAP1 gene loci. Individuals may also be genetically compound heterozygotes but may phenotypically look homozygous with severely elevated LDL-C and physical symptoms. Assessment of family history of high LDL-C and premature coronary heart disease is crucial for HoFH diagnosis.

Familial Hypercholesterolemia (FH) Treatment

Early diagnosis and treatment of FH are vital to reduce the risk of premature atherosclerotic cardiovascular disease and death. The goal of treatment is to reduce LDL-C by 50 % from baseline levels with lifestyle modification, pharmacologic lipid-lowering therapy, and LDL apheresis and in rare cases, liver transplantation. People who get only one copy of the defective gene from their parents may do well with diet changes and statin drugs. Pharmacologic treatment ranges from statin medications to newer agents such as lomitapide, mipomersin, and PCSK9 inhibitors. Combination therapy is frequently required to accomplish goal lipoprotein level reductions and prevent complications.

Familial Hypercholesterolemia (FH) Epidemiology

The disease epidemiology covered in the report provides historical as well as forecasted epidemiology segmented by Prevalent Population of Familial hypercholesterolemia, Diagnosed Prevalent Population of Familial hypercholesterolemia, Mutation-specific Diagnosed Prevalence of Familial hypercholesterolemia and Diagnosed Prevalence of Coronary Heart Disease due to Familial hypercholesterolemia in the 7MM market covering the United States, EU5 countries (Germany, France, Italy, Spain, and United Kingdom) and Japan from 2017 to 2030.

Key Findings

This section provides glimpse of the Familial Hypercholesterolemia (FH) epidemiology in the 7MM.
  • The total prevalent population of Familial Hypercholesterolemia (FH) in the seven major markets was found to be 2,367,466 in 2017. However, total diagnosed prevalent patient population of Familial Hypercholesterolemia (FH) was 965,405 in 2017, which is anticipated to increase by 2030 in the seven major markets.
  • The diagnosed prevalence of FH was found to be 244,214 in the United States in 2017.
  • In the EU5 Countries, France had the maximum diagnosed prevalent population of Familial Hypercholesterolemia (FH) in 2017, followed by Italy .
  • Japan accounted for 221,519 cases of diagnosed prevalent population Familial Hypercholesterolemia (FH) in 2017.
Country Wise- Familial Hypercholesterolemia (FH) Epidemiology

The epidemiology segment also provides the Familial Hypercholesterolemia (FH) epidemiology data and findings across the United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom) and Japan.

Familial Hypercholesterolemia (FH) Drug Chapters

The drug chapter segment of the Familial Hypercholesterolemia (FH) report encloses the detailed analysis of Familial Hypercholesterolemia (FH) marketed drugs and mid and late stage pipeline drugs. It also helps to understand the Familial Hypercholesterolemia (FH) clinical trial details, expressive pharmacological action, agreements and collaborations, approval and patent details of each included drug and the latest news and press releases.

Familial Hypercholesterolemia (FH) Marketed Drugs

Praluent (alirocumab): Sanofi/ Regeneron Pharmaceuticals
Alirocumab is marketed by brand name Praluent, which is a human monoclonal antibody that binds to proprotein convertase subtilisin kexin type 9 (PCSK9). PCSK9 binds to the low-density lipoprotein receptors (LDLR) on the surface of hepatocytes to promote LDLR degradation within the liver. LDLR is the primary receptor that clears circulating LDL, therefore the decrease in LDLR levels by PCSK9 results in higher blood levels of LDL-C. By inhibiting the binding of PCSK9 to LDLR, alirocumab increases the number of LDLRs available to clear LDL, thereby lowering LDL-C levels.

Products detail in the report.
Repatha (evolocumab): Amgen
Evolocumab is a human monoclonal immunoglobulin G2 (IgG2) directed against human proprotein convertase subtilisin kexin 9 (PCSK9). Evolocumab has an approximate molecular weight (MW) of 144 kDa and is produced in genetically engineered mammalian (Chinese hamster ovary) cells.

Evolocumab binds to PCSK9 and inhibits circulating PCSK9 from binding to the low-density lipoprotein (LDL) receptor (LDLR), preventing PCSK9-mediated LDLR degradation and permitting LDLR to recycle back to the liver cell surface. By inhibiting the binding of PCSK9 to LDLR, evolocumab increases the number of LDLRs available to clear LDL from the blood, thereby lowering LDL-C levels.

Products detail in the report.
Nustendi (bempedoic acid / ezetimibe): Esperion Therapeutics
Nustendi is a combination of bempedoic acid and ezetimibe developed by Esperion Therapeutics. Bempedoic acid has been designed as a once-daily, oral therapy that can significantly reduce low-density lipoprotein cholesterol (LDL-C) levels when added to other lipid-lowering therapies in clinical trials of patients with primary hyperlipidemia who need additional LDL-C lowering.

Products detail in the report.

Familial Hypercholesterolemia (FH) Emerging Drugs

Evinacumab (REGN1500): Regeneron Pharmaceuticals
Evinacumab (REGN1500) is being investigated by Regeneron Pharmaceuticals, which is a fully human monoclonal antibody (mAb) to angiopoietin-like protein 3 (ANGPTL3) that acts as an inhibitor of lipoprotein lipase (LPL) and endothelial lipase (EL) administered intravenously. It plays a central role in lipoprotein metabolism by binding to ANGPTL3 with high affinity and specificity, and to completely reverse its inhibitory activity on LPL and EL both in vitro and in vivo thereby reducing the serum triglycerides and VLDL-C levels in the patient (Creative Biolabs, n.d.). ANGPTL3 inhibition increases activity of endothelial lipase and, therefore, decreases HDL-C levels.

Products detail in the report.
Inclisiran (ALN-PCSSC): Novartis
Inclisiran by Novartis is a long-acting, synthetic siRNA directed against PCSK9 and it has been shown to significantly decrease hepatic production of PCSK9 and cause a marked reduction in LDL-C levels. Inclisiran is conjugated to triantennary N-acetylgalactosamine carbohydrates administered via subcutaneous injection. These carbohydrates bind to abundant liver-expressed asialoglycoprotein receptors, leading to the uptake of inclisiran specifically into the hepatocytes.

Products detail in the report.
LIB003 + Evolocumab: LIB Therapeutics
LIB003 is a PCSK9 Inhibitor being developed by LIB Therapeutics, which is administered subcutaneously. The PCSK9 protein is an important regulator of circulating LDL‐C levels, through its inhibitory action on recycling of the LDL receptor (LDLR). LDLR on the liver cell surface binds to LDL and the LDLR-LDL complex is then internalized, after which the LDLR is normally recycled back to the cell surface up to 150 times. Secreted PCSK9 binds to the LDLR on the surface of the hepatocyte, leading to the internalization and degradation of the LDLR in the lysosomes, and reducing the number of LDLRs on the cell surface. Inhibition of secreted PCSK9 should therefore increase the number of available LDLRs on the cell surface and increase uptake of LDL‐C into the cell. PCSK9 inhibition thus offers a novel therapeutic mechanism for the lowering of LDL‐C levels.

Products detail in the report.
Gemcabene: NeuroBo Pharmaceuticals
Gemcabene, a small molecule and is the monocalcium salt of a dialkyl ether dicarboxylic acid [6,6′-oxybis (2,2-dimethylhexanoic acid)] in late-stage clinical development, lowers pro-inflammatory acute-phase protein, C-reactive protein (CRP). It inhibits both cholesterol and fatty acid synthesis as determined by the C-acetate incorporation in hepatocytes. In addition to LDL lowering, gemcabene reduces plasma levels of CRP in patients by 53.5% in monotherapy and by 71% in combination with statins, indicating that this compound may have anti-inflammatory properties.

Products detail in the report.
Resmetirom (MGL-3196): Madrigal Pharmaceuticals
Resmetirom (MGL-3196) is a human body, thyroid hormone. Through activation of its beta receptor it plays a central role in controlling lipid metabolism, impacting a range of health parameters from levels of serum cholesterol and triglycerides to the pathological buildup of fat in the liver. MGL-3196 attempts to exploit this pathway for therapeutic purposes in cardio-metabolic and liver diseases have been hampered by the lack of selectivity of older compounds for the thyroid hormone receptor (THR)-β, chemically-related toxicities and undesirable distribution in the body. Madrigal recognized that greater selectivity for thyroid hormone receptor (THR)-β and liver targeting might overcome these challenges and deliver the full therapeutic potential of THR-β agonism. Madrigal believes that MGL-3196 is the first orally administered, small-molecule, liverdirected, truly β-selective THR agonist that has demonstrated the potential for a broad array of therapeutically beneficial effects, improving components of both metabolic syndrome, such as insulin resistance and dyslipidemia, and fatty liver disease, including lipotoxicity and inflammation.

Products detail in the report.

Familial Hypercholesterolemia (FH) Market Outlook

This section of the report includes the dynamics of market scenario for FH in the 7MM, i.e., the United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom), and Japan.

FH is the most common autosomal dominant genetic disease. It is characterized by extremely elevated levels of low-density lipoprotein cholesterol (LDL-C) and a tendency to early-onset atherosclerotic cardiovascular disease. In general, homozygotes manifest the disease at a much earlier age than heterozygotes, and the condition is more severe. It is important to note that if diagnosed, it can be treated with medicines and a healthy lifestyle.

The current market of familial hypercholesterolemia comprises of several treatment options lying in variable classes, such as Statins, Cholesterol absorption inhibitors (Zetia), MTP inhibitors (Juxtapid), APOB antisense inhibitors (Kynamro; mipomersen sodium), PCSK9 inhibitors (Praluent and Repatha), and others (bile-acid-binding resins). In the case of advanced treatment options, Lipoprotein apheresis can also be opted to treat the patients with FH. While in rare and severe cases, patients can also go for liver transplantation.

The pipeline of FH holds potential products by several key players, such as Regeneron Pharmaceuticals (Evinacumab), Novartis (Inclisiran), LIB Therapeutics (LIB003 with Evolocumab), NeuroBo Pharmaceuticals (Gemcabene), Madrigal Pharmaceuticals (Resmetirom), and Arrowhead Pharmaceuticals (ARO-ANG3). 

Key Findings

This section includes a glimpse of the Familial Hypercholesterolemia (FH) 7MM market.
  • The market size of Familial Hypercholesterolemia (FH) in the seven major markets is 1,069.27 USD Million in 2017.
  • The United States accounts for the largest market size of Familial Hypercholesterolemia (FH), in comparison to EU5 (the United Kingdom, Germany, Italy, France, and Spain) and Japan.
  • Among the EU5 countries, Germany had the highest market size with 84.82 USD Million in 2017. While Spain had the lowest market size of Familial Hypercholesterolemia (FH).
The United States Market Outlook

This section provides the total Familial Hypercholesterolemia (FH) market size and market size by therapies in the United States.

EU-5 Market Outlook

The total aspregillosis market size and market size by therapies in Germany, France, Italy, Spain, and the United Kingdom are provided in this section.

Japan Market Outlook

The total Familial Hypercholesterolemia (FH) market size and market size by therapies in Japan are provided.

Familial Hypercholesterolemia (FH) Drugs Uptake

This section focusses on the rate of uptake of the potential drugs recently launched in the Familial Hypercholesterolemia (FH) market or expected to get launched in the market during the study period 2017-2030. The analysis covers Familial Hypercholesterolemia (FH) market uptake by drugs; patient uptake by therapies; and sales of each drug.

This helps in understanding the drugs with the most rapid uptake, reasons behind the maximal use of new drugs and allow the comparison of the drugs on the basis of market share and size which again will be useful in investigating factors important in market uptake and in making financial and regulatory decisions.

Familial Hypercholesterolemia (FH) Development Activities

The report provides insights into different therapeutic candidates in phase II, and phase III stage. It also analyzes key players involved in developing targeted therapeutics.

Pipeline Development Activities

The report covers the detailed information of collaborations, acquisition and merger, licensing and patent details for Familial Hypercholesterolemia (FH) emerging therapies.

Competitive Intelligence Analysis

The publisher performs competitive and market Intelligence analysis of the Familial Hypercholesterolemia (FH) market by using various competitive intelligence tools that include-SWOT analysis, PESTLE analysis, Porter's five forces, BCG Matrix, Market entry strategies, etc. The inclusion of the analysis entirely depends upon the data availability.

Scope of the Report
  • The report covers the descriptive overview of Familial Hypercholesterolemia (FH), explaining its causes, signs and symptoms, pathogenesis and currently available therapies.
  • Comprehensive insight has been provided into the Familial Hypercholesterolemia (FH) epidemiology and treatment.
  • Additionally, an all-inclusive account of both the current and emerging therapies for Familial Hypercholesterolemia (FH) are provided, along with the assessment of new therapies, which will have an impact on the current treatment landscape.
  • A detailed review of Familial Hypercholesterolemia (FH) market; historical and forecasted is included in the report, covering the 7MM drug outreach.
  • The report provides an edge while developing business strategies, by understanding trends shaping and driving the 7MM Familial Hypercholesterolemia (FH) market.
Report Highlights
  • In the coming years, Familial Hypercholesterolemia (FH) market is set to change due to the rising awareness of the disease, and incremental healthcare spending across the world; which would expand the size of the market to enable the drug manufacturers to penetrate more into the market.
  • The companies and academics are working to assess challenges and seek opportunities that could influence Familial Hypercholesterolemia (FH) R&D. The therapies under development are focused on novel approaches to treat/improve the disease condition.
  • Report also covers Mutation-specific diagnosed prevalence of FH, including several mutations such as LDL receptor (LDLR) Mutations, Proprotein Convertase Subtilin/Kexin 9 (PCSK9), Apolipoprotein B (Apo B) and Other rare mutations (SREBP2, and STAP1 genes, LDLRAP1 gene).
  • Deleveinsight has segregated prevalence of FH on the basis of genetic types, i.e., homozygous familial hypercholesterolemia (HoFH) and heterozygous hypercholesterolemia (HeFH).
  • Expected Launch of potential therapies may increase market size in the coming years, assisted by an increase in diagnosed prevalent population of Familial Hypercholesterolemia (FH). Owing to the positive outcomes of the upcoming products during the developmental stage by key players such as Regeneron Pharmaceuticals, Novartis, LIB Therapeutics, NeuroBo Pharmaceuticals, Madrigal Pharmaceuticals and Arrowhead Pharmaceuticals have a potential to create a significant positive shift in the Familial Hypercholesterolemia (FH) Market Size.
  • The publisher has also estimated the diagnosed prevalent population of Coronary Heart Disease (CHD) due to FH.
Familial Hypercholesterolemia (FH) Report Insights
  • Patient Population
  • Therapeutic Approaches
  • Familial Hypercholesterolemia (FH) Pipeline Analysis
  • Familial Hypercholesterolemia (FH) Market Size and Trends
  • Market Opportunities
  • Impact of upcoming Therapies
Familial Hypercholesterolemia (FH) Report Key Strengths
  • Eleven Years Forecast
  • 7MM Coverage
- Familial Hypercholesterolemia (FH) Epidemiology Segmentation
  • Key Cross Competition
  • Highly Analyzed Market
  • Drugs Uptake
Familial Hypercholesterolemia (FH) Report Assessment
  • Current Treatment Practices
  • Unmet Needs
  • Pipeline Product Profiles
  • Market Attractiveness
  • Market Drivers and Barriers
Key Questions

Market Insights:
  • What was the Familial Hypercholesterolemia (FH) market share (%) distribution in 2017 and how it would look like in 2030?
  • What would be the Familial Hypercholesterolemia (FH) total market size as well as market size by therapies across the 7MM during the forecast period (2020-2030)?
  • What are the key findings pertaining to the market across the 7MM and which country will have the largest Familial Hypercholesterolemia (FH) market size during the forecast period (2020-2030)?
  • At what CAGR, the Familial Hypercholesterolemia (FH) market is expected to grow at the 7MM level during the forecast period (2020-2030)?
  • What would be the Familial Hypercholesterolemia (FH) market outlook across the 7MM during the forecast period (2020-2030)?
  • What would be the Familial Hypercholesterolemia (FH) market growth till 2030 and what will be the resultant market size in the year 2030?
  • How would the market drivers, barriers and future opportunities affect the market dynamics and subsequent analysis of the associated trends?
Epidemiology Insights:
  • What is the disease risk, burden and unmet needs of Familial Hypercholesterolemia (FH)?
  • What is the historical Familial Hypercholesterolemia (FH) patient pool in the United States, EU5 (Germany, France, Italy, Spain, and the UK) and Japan?
  • What would be the forecasted patient pool of Familial Hypercholesterolemia (FH) at the 7MM level?
  • What will be the growth opportunities across the 7MM with respect to the patient population pertaining to Familial Hypercholesterolemia (FH)?
  • Out of the above-mentioned countries, which country would have the highest prevalent population of Familial Hypercholesterolemia (FH) during the forecast period (2020-2030)?
  • At what CAGR the population is expected to grow across the 7MM during the forecast period (2020-2030)?
Current Treatment Scenario, Marketed Drugs and Emerging Therapies:
  • What are the current options for the treatment of Familial Hypercholesterolemia (FH) along with the approved therapy?
  • What are the current treatment guidelines for the treatment of Familial Hypercholesterolemia (FH) in the US and Europe?
  • What are the Familial Hypercholesterolemia (FH) marketed drugs and their MOA, regulatory milestones, product development activities, advantages, disadvantages, safety and efficacy, etc.?
  • How many companies are developing therapies for the treatment of Familial Hypercholesterolemia (FH)?
  • How many therapies are developed by each company for the treatment of Familial Hypercholesterolemia (FH)?
  • How many emerging therapies are in the mid-stage and late stage of development for the treatment of Familial Hypercholesterolemia (FH)?
  • What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the Familial Hypercholesterolemia (FH) therapies?
  • What are the recent novel therapies, targets, mechanisms of action and technologies developed to overcome the limitation of existing therapies?
  • What are the clinical studies going on for Familial Hypercholesterolemia (FH) and their status?
  • What are the key designations that have been granted for the emerging therapies for Familial Hypercholesterolemia (FH)?
  • What are the 7MM historical and forecasted market of Familial Hypercholesterolemia (FH)?
Reasons to Buy
  • The report will help in developing business strategies by understanding trends shaping and driving the Familial Hypercholesterolemia (FH).
  • To understand the future market competition in the asprgillosis market and Insightful review of the key market drivers and barriers.
  • Organize sales and marketing efforts by identifying the best opportunities for Familial Hypercholesterolemia (FH) in the US, Europe (Germany, Spain, Italy, France, and the United Kingdom) and Japan.
  • Identification of strong upcoming players in the market will help in devising strategies that will help in getting ahead of competitors.
  • Organize sales and marketing efforts by identifying the best opportunities for Familial Hypercholesterolemia (FH) market.
  • To understand the future market competition in the Familial Hypercholesterolemia (FH) market.
Note: Product cover images may vary from those shown

FEATURED COMPANIES

  • Amgen
  • Arrowhead Pharmaceutica
  • Esperion Therapeutics
  • LIB Therapeutics
  • Madrigal Pharmaceuticals
  • NeuroBo Pharmaceuticals
  • MORE
1 Key Insights

2 Familial Hypercholesterolemia Market Overview at a Glance
2.1 Market Share (%) Distribution of Familial Hypercholesterolemia in 2017
2.2 Market Share (%) Distribution of Familial Hypercholesterolemia in 2030

3 Disease Background and Overview: Familial Hypercholesterolemia (FH)
3.1 Introduction
3.2 Types of Familial Hypercholesterolemia
3.2.1 Homozygous Familial Hypercholesterolemia (HoFH)
3.2.2 Heterozygous FH Familial Hypercholesterolemia (HeFH)
3.3 FH Causative Genes
3.3.1 LDL Receptor
3.3.2 Apo B-100
3.3.3 PCSK9
3.3.4 LDL Receptor Adapter Protein 1 (LDLRAP1)
3.4 Symptoms
3.5 Clinical Features of FH
3.5.1 Hyper-LDL-Cholesterolemia
3.5.2 Premature Coronary Artery Disease
3.5.3 Tendon and Skin Xanthomas
3.5.4 Corneal Arcus
3.5.5 Other Risk Factors in FH
3.6 Diagnosis
3.6.1 Detection of FH through Genetic Screening
3.6.2 Detection of FH through Cascade Screening
3.6.3 Achilles tendon radiography
3.6.4 Diagnosis of Pediatric FH
3.6.5 Establishing the Diagnosis
3.7 Differential diagnosis

4 Diagnostic guidelines for Familial Hypercholesterolemia by the American Heart Association (AHA)

5 Diagnostic guidelines for Familial Hypercholesterolemia by the National Institute for Health and Care Excellence (NICE)

6 Recognized Establishments
6.1 United States
6.2 Europe
6.3 Japan

7 Epidemiology and Patient Population: Key Findings
7.1 7MM Total Prevalent Population of Familial Hypercholesterolemia
7.2 7MM Total Diagnosed Prevalent Population of Familial Hypercholesterolemia

8 Country Wise-Epidemiology of Familial Hypercholesterolemia (FH)
8.1 United States
8.1.1 Assumptions and Rationale
8.1.2 Prevalent Population of Familial Hypercholesterolemia in the United States
8.1.3 Diagnosed Prevalent Population of Familial Hypercholesterolemia in the United States
8.1.4 Mutation-specific Diagnosed Prevalence of Familial Hypercholesterolemia in the United States
8.1.5 Diagnosed Prevalence of Coronary Heart Disease due to Familial Hypercholesterolemia in the United States
8.2 EU5 Countries
8.3 Germany
8.3.1 Assumptions and Rationale
8.3.2 Prevalent Population of Familial Hypercholesterolemia in Germany
8.3.3 Diagnosed Prevalent Population of Familial Hypercholesterolemia in Germany
8.3.4 Mutation-specific Diagnosed Prevalence of Familial Hypercholesterolemia in Germany
8.3.5 Diagnosed Prevalence of Coronary Heart Disease due to Familial Hypercholesterolemia in Germany
8.4 France
8.4.1 Assumptions and Rationale
8.4.2 Prevalent Population of Familial Hypercholesterolemia in France
8.4.3 Diagnosed Prevalent Population of Familial Hypercholesterolemia in France
8.4.4 Mutation-specific Diagnosed Prevalence of Familial Hypercholesterolemia in France
8.4.5 Diagnosed Prevalence of Coronary Heart Disease due to Familial Hypercholesterolemia in France
8.5 Italy
8.5.1 Assumptions and Rationale
8.5.2 Prevalent Population of Familial Hypercholesterolemia in Italy
8.5.3 Diagnosed Prevalent Population of Familial Hypercholesterolemia in Italy
8.5.4 Mutation-specific Diagnosed Prevalence of Familial Hypercholesterolemia in Italy
8.5.5 Diagnosed Prevalence of Coronary Heart Disease due to Familial Hypercholesterolemia in Italy
8.6 Spain
8.6.1 Assumptions and Rationale
8.6.2 Prevalent Population of Familial Hypercholesterolemia in Spain
8.6.3 Diagnosed Prevalent Population of Familial Hypercholesterolemia in Spain
8.6.4 Mutation-specific Diagnosed Prevalence of Familial Hypercholesterolemia in Spain
8.6.5 Diagnosed Prevalence of Coronary Heart Disease due to Familial Hypercholesterolemia in Spain
8.7 United Kingdom
8.7.1 Assumptions and Rationale
8.7.2 Prevalent Population of Familial Hypercholesterolemia in the United Kingdom
8.7.3 Diagnosed Prevalent Population of Familial Hypercholesterolemia in the United Kingdom
8.7.4 Mutation-specific Diagnosed Prevalence of Familial Hypercholesterolemia in the United Kingdom
8.7.5 Diagnosed Prevalence of Coronary Heart Disease due to Familial Hypercholesterolemia in the United Kingdom
8.8 Japan
8.8.1 Assumptions and Rationale
8.8.2 Prevalent Population of Familial Hypercholesterolemia in Japan
8.8.3 Diagnosed Prevalent Population of Familial Hypercholesterolemia in Japan
8.8.4 Mutation-specific Diagnosed Prevalence of Familial Hypercholesterolemia in Japan
8.8.5 Diagnosed Prevalence of Coronary Heart Disease due to Familial Hypercholesterolemia in Japan

9 Treatment
9.1 Lifestyle Modifications
9.2 Pharmacologic Treatment
9.2.1 Statins
9.2.2 Cholesterol Absorption Inhibitors
9.2.3 Bile Acid Sequestrants
9.2.4 Kynamro (Mipomersen)
9.2.5 Juxtapid (Lomitapide)
9.2.6 PCSK9 Inhibitors
9.3 Lipoprotein Apheresis
9.4 Liver Transplantation

10 Treatment guidelines for Familial Hypercholesterolemia by the American Heart Association (AHA)

11 Treatment guidelines by the National Institute for Health and Care Excellence (NICE)
11.1 Management of FH

12 Treatment guidelines for Familial Hypercholestrolemia by the Japan Atherosclerosis Society and the Asian Pacific Society of Atherosclerosis and Vascular Diseases

13 Unmet Needs

15 Marketed Drugs
15.1 Marketed Drugs Key-cross Competition
15.2 Praluent (alirocumab): Sanofi/ Regeneron Pharmaceuticals
15.2.1 Drug Description
15.2.2 Regulatory Milestones
15.2.3 Other Development Activities
15.2.4 Safety and Efficacy
15.2.5 Current Pipeline Activity
15.2.6 Product Profile
15.3 Repatha (evolocumab): Amgen
15.3.1 Drug Description
15.3.2 Regulatory Milestones
15.3.3 Other Development Activities
15.3.4 Safety and Efficacy
15.3.5 Product Profile
15.4 Nustendi (bempedoic acid / ezetimibe): Esperion Therapeutics
15.4.1 Drug Description
15.4.2 Regulatory Milestones
15.4.3 Other Development Activities
15.4.4 Current Pipeline Activity
15.4.5 Product Profile

16 Emerging Drugs
16.1 Key Cross Competition
16.2 Evinacumab (REGN1500): Regeneron Pharmaceuticals
16.2.1 Product Description
16.2.2 Other Developmental Activities
16.2.3 Clinical Development
16.2.4 Product Profile
16.3 Inclisiran (ALN-PCSSC): Novartis
16.3.1 Product Description
16.3.2 Other Developmental Activities
16.3.3 Clinical Development
16.3.4 Product Profile
16.4 LIB003 + Evolocumab: LIB Therapeutics
16.4.1 Product Description
16.4.2 Clinical Development
16.4.3 Product Profile
16.5 Gemcabene: NeuroBo Pharmaceuticals
16.5.1 Product Description
16.5.2 Other Developmental Activities
16.5.3 Clinical Development
16.5.4 Product Profile
16.6 Resmetirom (MGL-3196): Madrigal Pharmaceuticals
16.6.1 Product Description
16.6.2 Other Developmental Activities
16.6.3 Clinical Development
16.6.4 Product Profile
16.7 ARO-ANG3: Arrowhead Pharmaceuticals
16.7.1 Product Description
16.7.2 Other Developmental Activities
16.7.3 Clinical Development
16.7.4 Product Profile

17 Familial Hypercholesterolemia (FH): 7 Major Market Analysis
17.1 Key Findings
17.2 Market Size of Familial Hypercholesterolemia in the 7MM
17.3 7MM Market Outlook
17.4 United States Market Size
17.4.1 Total Market size of Familial Hypercholesterolemia
17.4.2 Current and Emerging Market Size by Therapies
17.5 Germany Market Size
17.5.1 Total Market size of Familial Hypercholesterolemia in Germany
17.5.2 Current and Emerging Market Size by Therapies
17.6 France Market Size
17.6.1 Total Market size of Familial Hypercholesterolemia in France
17.6.2 Current and Emerging Market Size by Therapies
17.7 Italy Market Size
17.7.1 Total Market size of Familial Hypercholesterolemia in Italy
17.7.2 Current and Emerging Market Size by Therapies
17.8 Spain Market Size
17.8.1 Total Market size of Familial Hypercholesterolemia in Spain
17.8.2 Current and Emerging Market Size by Therapies
17.9 United Kingdom Market Size
17.9.1 Total Market size of Familial Hypercholesterolemia in the United Kingdom
17.9.2 Current and Emerging Market Size by Therapies
17.1 Japan market Size
17.10.1 Total Market Size of Familial Hypercholesterolemia
17.10.2 Current and Emerging Market Size by Therapies

18 Case Reports
18.1 A 72-year-old Patient with Longstanding, Untreated Familial Hypercholesterolemia but no Coronary Artery Calcification: A Case Report (United States)
18.2 Management of homozygous familial hypercholesterolemia in two brothers (Spain)
18.3 A Japanese case of familial hypercholesterolemia with a novel mutation in the LDLR gene

19 Market Drivers

20 Market Barriers

21 SWOT Analysis for Familial Hypercholesterolemia

22 Appendix
22.1 Bibliography
22.2 Report Methodology

23 Capabilities

24 Disclaimer

25 About the Publisher
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  • Sanofi/ Regeneron Pharmaceuticals
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  • Esperion Therapeutics
  • Regeneron Pharmaceuticals
  • Novartis
  • LIB Therapeutics
  • NeuroBo Pharmaceuticals
  • Madrigal Pharmaceuticals
  • Arrowhead Pharmaceutica
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