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Homozygous Familial Hypercholesterolemia Market

DelveInsight’s ‘Homozygous Familial Hypercholesterolemia (HoFH) - Market Insights, Epidemiology and Market Forecast– 2030’ report delivers an in-depth understanding of the HoFH , historical and forecasted epidemiology as well as the HoFH market trends in the United States, EU5 (Germany, Spain, Italy, France, and United Kingdom) and Japan.


The HoFH market report provides current treatment practices, emerging drugs, and market share of the individual therapies, current and forecasted 7MM HoFH market size from 2017 to 2030. The report also covers current HoFH 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

Homozygous Familial Hypercholesterolemia (HoFH) Disease Understanding and Treatment Algorithm

Homozygous Familial Hypercholesterolemia (HoFH) Overview

Homozygous Familial Hypercholesterolemia (HoFH) is the more serious form of familial hypercholesterolemia (FH). FH is an autosomal dominant genetic disorder, meaning that only one parent needs to have the condition for his or her children to inherit it. HoFH is a rare and life-threatening disease originally characterized clinically by plasma cholesterol levels >13 mmol/L (>500 mg/dL), extensive xanthomas, and marked premature and progressive atherosclerotic cardiovascular disease (ACVD). It is defined as having two pathogenic mutations in two alleles of the causative genes. According to the National Institutes of Health, patients with HoFH have LDL cholesterol (LDL-C) levels three to six times higher than normal.


The physical signs of HoFH are generally more severe and occur at an earlier age than in patients with HeFH. Untreated patients with HoFH generally present with atherosclerosis, aortic valve disease, and coronary heart disease before the age of 20 years. The deposition of cholesterol in vascular and extravascular compartments, such as tendons and skin, results in xanthomas (yellow, waxy patches on your skin over the elbows, knees, and buttocks due to subcutaneous lipid depositions usually caused by derangement of lipoprotein metabolism) and the deposition of lipid in the cornea of the eyes causes corneal arcus and an increase in intraocular pressure. Some symptoms of heart disease include chest pain (angina), rapid heartbeat, and shortness of breath.


Most patients with genetically confirmed HoFH have two mutant alleles of the LDLR gene, and their parents have HeFH each. Mutations in alleles of three other genes were also identified as causal in some cases with a severe phenotype resembling HoFH. These secondary genes are ApoB encoding apolipoprotein (apo) B, PCSK9 encoding proprotein convertase subtilisin/kexin type 9 (PCSK9), and LDLRAP1 encoding LDL receptor adapter protein 1, which uniquely causes a recessive phenotype since carrier parents have normal lipid profiles. Patients are homozygotes, with the same mutation in both alleles of the same gene, or more commonly, compound heterozygotes with different mutations in each allele of the same gene, or double heterozygotes with mutations in two different genes affecting LDL receptor function. Irrespective of the underlying genetic defect, the severity of the HoFH phenotype depends on residual LDL receptor activity.

Homozygous Familial Hypercholesterolemia (HoFH) Diagnosis

Diagnosis of HoFH can be made based on genetic or clinical criteria. While genetic testing may provide a definitive diagnosis of HoFH, it is recognized that in some patients genetic confirmation remains elusive, despite exhaustive investigation; indeed, the existence of additional FH genes cannot be excluded. Historically, HoFH has been most commonly diagnosed on the basis of an untreated LDL-C plasma concentration >13 mmol/L (>500 mg/dL), or a treated LDL-C concentration of ≥8 mmol/L (≥300 mg/dL), and the presence of cutaneous or tendon xanthomas before the age of 10 years, or the presence of untreated, elevated LDL-C levels consistent with HeFH in both parents. HoFH can be diagnosed based on clinical features: total serum cholesterol of 600 mg/dL or more, cutaneous xanthomas and ASCVD from childhood and parents’ family history of HeFH. Cutaneous and tendon xanthomas frequently occur in parts subjected to mechanical stimulation, such as the finger joints, elbow joints, knee joints and so on. If it is difficult to distinguish between HoFH and severe HeFH, genetic analysis is useful.

Homozygous Familial Hypercholesterolemia (HoFH) Treatment

Given the severity of hypercholesterolemia with increased CV risk, HoFH requires intensive therapy. However, HoFH is often unresponsive to traditional treatment. The foundation of treatment for HoFH is an aggressive lowering of LDL-C levels in an attempt to bring levels to goal. This is often difficult to achieve despite multidrug treatment, given the markedly elevated levels of cholesterol at baseline and the limited response to available treatments. Statin treatment has been shown to affect CVD mortality in HoFH and statins are typically first-line treatment started at the time of diagnosis, including in very young children.


In HoFH, to prevent the incidence and progression of CAD, intensive lipid-lowering therapy should be initiated as early as possible at an early age. The major mechanisms of action of statins, bile acid adsorbing resins and PCSK9 inhibitors are to enhance expression (activation) of LDL receptors. For the defective type, in which only a small amount of LDL receptor activity remains, slight efficacy is observed, but in the negative type in which LDL receptor activity is completely absent, no LDL-C lowering effect is observed. However, as the frequencies of the adverse events of fatty liver and diarrhea are high, it is essential to control the fat and alcohol intake strictly. Probucol reportedly exerts a certain LDL-C lowering effect on HoFH and may cause the regression or disappearance of xanthoma in the skin or Achilles tendon). Nevertheless, for LDL-C control, LDL apheresis therapy once every 1–2 weeks is still required in many cases. When patients are resistant to all of the above treatments or show intolerance, liver transplantation may be considered.


Furthermore, in patients with HoFH, it is difficult to decrease the LDL-C level sufficiently using existing drug therapies, and many patients require continued LDL apheresis with extracorporeal circulation starting in childhood. Considering the inhibition of the progression of CAD, the earlier LDL apheresis is initiated, the better; however, it is difficult to perform LDL apheresis until the affected child can be kept in bed during apheresis. Realistically, the timing of treatment initiation is 4–6 years of age, when children can lie on the bed, and extracorporeal circulation can be performed; however, it is recommended that the treatment be initiated as early as possible.


Homozygous Familial Hypercholesterolemia (HoFH) Epidemiology

The disease epidemiology covered in the report provides historical as well as forecasted epidemiology segmented by Prevalence of Homozygous Familial Hypercholesterolemia (HoFH), Diagnosed Prevalence of Homozygous Familial Hypercholesterolemia (HoFH) and Mutation-specific Distribution of Homozygous Familial Hypercholesterolemia (HoFH) 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 HoFH epidemiology in the 7MM.

  • The prevalent population of HoFH in the seven major markets is estimated to be 3,882 in 2020.
  • The diagnosed prevalent cases of HoFH, in the United States, is estimated to be 698 in 2020.
  • HoFH can be divided into LDL Receptor (LDLR), Apolipoprotein B (Apo B), Proprotein Convertase Subtilin/Kexin 9 (PCSK9), and other rare mutations (SREBP2, and STAP1 genes, LDLRAP1 gene) based on the types of mutations causing the condition. In the United States, the number of cases of HoFH caused by LDLR, Apo B, PCSK9, and other rare mutations (SREBP2, and STAP1 genes, LDLRAP1 gene) was 587, 20, 13, and 33 respectively, in 2017.
  • In the EU5 countries, the diagnosed prevalence of HoFH was maximum in Germany with 231 cases, followed by the France with 189 cases in 2017. While, the least number of cases were in Spain, with 131 cases in 2017.
  • In Japan, the diagnosed prevalence of HoFH is estimated to be 215 in 2020.


Country-wise Homozygous Familial Hypercholesterolemia (HoFH) Epidemiology

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


Homozygous Familial Hypercholesterolemia (HoFH) Drug Chapters

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

Homozygous Familial Hypercholesterolemia (HoFH): Marketed Drugs

Repatha (evolocumab): Amgen

Repatha (evolocumab) is a human monoclonal IgG2 antibody that inhibits proprotein convertase subtilisin/kexin type 9 (PCSK9). The drug 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, Repatha increases the number of LDLRs available to clear LDL from the blood, thereby lowering LDL-C levels. The drug has an approximate molecular weight (MW) of 144 kDa and is produced in genetically engineered mammalian

Product details in the report…


Juxtapid (Lomitapide): Aegerion Pharmaceutical

Juxtapid (AEGR-733) is a novel oral therapeutic agent for hypercholesterolemia. Its mechanism involves inhibition of microsomal triglyceride transfer protein, resulting in a reduction of LDL cholesterol. The drug directly binds and inhibits microsomal triglyceride transfer protein (MTP), which resides in the lumen of the endoplasmic reticulum, thereby preventing the assembly of apo B containing lipoproteins in enterocytes and hepatocytes. This inhibits the synthesis of chylomicrons and VLDL. The inhibition of the synthesis of VLDL leads to reduced levels of plasma LDL-C.

Product details in the report…

Homozygous Familial Hypercholesterolemia (HoFH): Emerging Drugs

Evinacumab (REGN1500): Regeneron Pharmaceuticals

Evinacumab (REGN1500) is an investigational fully-human monoclonal antibody that binds to and blocks the function of ANGPTL3. ANGPTL3 plays a key role in regulating plasma lipid levels, including triglycerides, LDL cholesterol, and HDL cholesterol, through inhibition of lipase enzymes (lipoprotein lipase and endothelial lipase). It acts as an inhibitor of lipoprotein lipase (LPL) and endothelial lipase (EL) and is administered intravenously. Regeneron invented evinacumab using the company’s VelocImmune technology, a proprietary genetically-engineered mouse platform endowed with a genetically-humanized immune system to produce optimized fully-human monoclonal antibodies.

Product details in the report…


Inclisiran: Alnylam Pharmaceuticals/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.

Product details in the report…


LIB003: LIB Therapeutics

LIB003, developed by LIB Therapeutics, is a PCSK9 Inhibitor being which is administered subcutaneously. The PCSK9 protein is an essential 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.

Product details in the report…


Praluent (Alirocumab): Sanofi/Regeneron Pharmaceuticals

Praluent (alirocumab) is a human monoclonal antibody (mAb) that blocks the interaction of proprotein convertase subtilisin/kexin type 9 (PCSK9) with low-density lipoprotein (LDL) receptors, increasing the recycling of LDL receptors and reducing LDL cholesterol levels. 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. Praluent is being developed by Regeneron and Sanofi under a global collaboration agreement. In the US, Praluent is approved for use as an adjunct to diet and maximally tolerated statin therapy for the treatment of adults with HeFH and has currently completed phase III clinical developmental trial for treating HoFH patients.

Product details in the report…


Gemcabene: NeuroBo Pharmaceuticals

Gemcabene is a novel lipid-lowering agent administered orally with a mechanism of action (MOA) independent of the LDLR, which has previously demonstrated the ability to reduce levels of LDL-C on top of maximally tolerated statins. Gemcabene’s MOA is designed to enhance the clearance of very-low-density lipoproteins (VLDLs) in the plasma and inhibition of the production of cholesterol and triglycerides in the liver. The combined effect of these mechanisms has been clinically observed to result in a reduction of plasma non-HDL-C, VLDL-C, LDL-C, apolipoprotein B and triglycerides. In addition, gemcabene has been shown to markedly lower C-reactive protein in humans and improve insulin sensitization. Gemcabene’s MOA is liver-directed involving downregulation of hepatic apolipoprotein C-III (apoC-III) mRNA expression and decrease of plasma apoC-III levels


ARO-ANG3: Arrowhead Pharmaceuticals

ARO-ANG3 is designed to reduce the production of angiopoietin-like protein 3 (ANGPTL3), a liver synthesized inhibitor of lipoprotein lipase and endothelial lipase by Arrowhead Pharmaceuticals. ANGPTL3 inhibition has been shown to lower serum LDL, serum and liver triglyceride and has genetic validation as a novel target for cardiovascular diseases and FH. It is being tested in the patients via a subcutaneous mode of administration.

Product details in the report…


Homozygous Familial Hypercholesterolemia (HoFH): Market Outlook

According to the FH Foundation, Homozygous Familial Hypercholesterolemia (HoFH) is a rare but serious autosomal dominant genetic condition that can lead to aggressive and premature heart disease, heart attacks, and strokes even in patients below 18 years. The indication has an estimated prevalence of 1 in 160,000 to 1 in 300,000 individuals.


The current market of HoFH comprises of several classes of treatment options, namely, Statins, MTP inhibitors (Lomitapide), PCSK9 inhibitors, and others (bile-acid-binding resins). In the case of advanced treatment options, Lipoprotein apheresis can also be opted to treat the patients with HoFH. While in rare and severe cases, liver transplantation can also be advised.


Out of these therapies, statins remain the first-line therapy for LDL-C lowering in HoFH, including in very young children, whereas, lomitapide remains the most powerful drug approved for HoFH with studies showing good response and tolerability for lomitapide. Common statin drugs that are usually prescribed for treating HoFH patients include Lipitor, Lescol, Pravachol, Crestor, Zocor, and others. Furthermore, statin treatment has been shown to affect CVD mortality in patients suffering from HoFH. Though this therapy is usually prescribed in combination with ezetimibe; however, this combination is far from adequate in achieving the required goal LDL-C levels in HoFH patients.


Since HoFH patients face a much more intensive challenge in managing their condition than an average person with high cholesterol. Therefore, to manage this condition, currently, there are two approved therapies available in the 7MM market, namely, Repatha (Amgen) and Juxtapid (Aegerion Pharmaceuticals).


Even after the availability of so many therapeutic options, HoFH is highly underdiagnosed and undertreated. This fact has also been highlighted by the European Atherosclerosis Society (EAS) Consensus Panel. Additionally, the organization also indicated that even at the highest doses of the most efficacious statins, only modest reductions in LDL C plasma levels, of 10–25%, are generally observed in HoFH patients. The EAS Consensus Panel suggests that the combination of statin plus ezetimibe can produce a further decrease in LDL-C levels of 10–15%. Furthermore, combination with other lipid-lowering treatments may also be considered, depending on their availability and tolerability.

Key Findings

This section includes a glimpse of the HoFH 7MM market.

  • The market size of HoFH in the seven major markets is estimated to be USD 159.5 Million in 2020.
  • The United States accounts for the largest market size of HoFH in comparison to EU5 (the United Kingdom, Germany, France, Italy, and Spain) and Japan.
  • Among the EU5 countries, Germany had the highest market size with USD 9.9 Million in 2017, while Spain had the smallest market size of HoFH with USD 5.6 Million in 2017, which is expected to rise during the study period 2017–2030.
  • The Japan HoFH market accounts for USD 16.8 Million in 2020.


The United States Market Outlook

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


EU-5 Market Outlook

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


Homozygous Familial Hypercholesterolemia (HoFH) Drugs Uptake

This section focusses on the rate of uptake of the potential drugs recently launched in the Homozygous Familial Hypercholesterolemia (HoFH) market or expected to get launched in the market during the study period 2017–2030. The analysis covers Homozygous Familial Hypercholesterolemia (HoFH) 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.


Homozygous Familial Hypercholesterolemia (HoFH) 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 Homozygous Familial Hypercholesterolemia (HoFH) emerging therapies.


Competitive Intelligence Analysis

We perform competitive and market Intelligence analysis of the Homozygous Familial Hypercholesterolemia (HoFH) 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 Homozygous Familial Hypercholesterolemia (HoFH) , explaining its causes, signs and symptoms, pathogenesis and currently available therapies.
  • Comprehensive insight has been provided into the Homozygous Familial Hypercholesterolemia (HoFH) epidemiology and treatment.
  • Additionally, an all-inclusive account of both the current and emerging therapies for Homozygous Familial Hypercholesterolemia (HoFH) are provided, along with the assessment of new therapies, which will have an impact on the current treatment landscape.
  • A detailed review of Homozygous Familial Hypercholesterolemia (HoFH) 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 Homozygous Familial Hypercholesterolemia (HoFH) market.

Report Highlights

  • In the coming years, HoFH 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 HoFH R&D. The therapies under development are focused on novel approaches to treat/improve the disease condition.
  • Delvelnsight has also analysed mutation-specific data of HoFH, which suggested that mutations in the LDLR gene is the most common causes of HoFH as compared to Apo B, PCSK9, and other rare mutations (SREBP2, and STAP1 genes, LDLRAP1 gene).
  • Currently, the drugs used for the treatment of HoFH in the 7MM include Statins (mostly high intensity) alone or in combination with Ezetimibe, MTP inhibitor (lomitapide, Juxtapid), PCSK9 Inhibitor (Repatha), others (Bile acid sequestrants (cholestyramine, colesevelam), Stanol esters, Fibrates, Binders) along with Lipoprotein Apheresis.
  • Expected Launch of potential therapies, Evinacumab/ REGN1500 (Regeneron Pharmaceuticals), LIB003 (LIB Therapeutics), ARO-ANG3 (Arrowhead Pharmaceuticals) and Inclisiran (Novartis), Alirocumab/ Praluent (Regeneron Pharmaceuticals/ Sanofi), may increase the market size in the coming years, assisted by an increase in the diagnosed prevalent population of HoFH.


Homozygous Familial Hypercholesterolemia (HoFH) Report Insights

  • Patient Population
  • Therapeutic Approaches
  • Homozygous Familial Hypercholesterolemia (HoFH) Pipeline Analysis
  • Homozygous Familial Hypercholesterolemia (HoFH) Market Size and Trends
  • Market Opportunities
  • Impact of upcoming Therapies


Homozygous Familial Hypercholesterolemia (HoFH) Report Key Strengths

  • Eleven Years Forecast
  • 7MM Coverage
  • Homozygous Familial Hypercholesterolemia (HoFH) Epidemiology Segmentation
  • Key Cross Competition
  • Highly Analyzed Market
  • Drugs Uptake


Homozygous Familial Hypercholesterolemia (HoFH) Report Assessment

  • Current Treatment Practices
  • Unmet Needs
  • Pipeline Product Profiles
  • Market Attractiveness
  • Market Drivers and Barriers

Key Questions

Market Insights:

  • What was the Homozygous Familial Hypercholesterolemia (HoFH) market share (%) distribution in 2017 and how it would look like in 2030?
  • What would be the Homozygous Familial Hypercholesterolemia (HoFH) 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 Homozygous Familial Hypercholesterolemia (HoFH) market size during the forecast period (2020–2030)?
  • At what CAGR, the Homozygous Familial Hypercholesterolemia (HoFH) market is expected to grow at the 7MM level during the forecast period (2020–2030)?
  • What would be the Homozygous Familial Hypercholesterolemia (HoFH) market outlook across the 7MM during the forecast period (2020–2030)?
  • What would be the Homozygous Familial Hypercholesterolemia (HoFH) 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 Homozygous Familial Hypercholesterolemia (HoFH)?
  • What is the historical Homozygous Familial Hypercholesterolemia (HoFH) patient pool in the United States, EU5 (Germany, France, Italy, Spain, and the UK) and Japan?
  • What would be the forecasted patient pool of Homozygous Familial Hypercholesterolemia (HoFH) at the 7MM level?
  • What will be the growth opportunities across the 7MM with respect to the patient population pertaining to Homozygous Familial Hypercholesterolemia (HoFH)?
  • Out of the above-mentioned countries, which country would have the highest prevalent population of Homozygous Familial Hypercholesterolemia (HoFH) 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 Homozygous Familial Hypercholesterolemia (HoFH) along with the approved therapy?
  • What are the current treatment guidelines for the treatment of Homozygous Familial Hypercholesterolemia (HoFH) in the US and Europe?
  • What are the Homozygous Familial Hypercholesterolemia (HoFH) 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 Homozygous Familial Hypercholesterolemia (HoFH) ?
  • How many therapies are developed by each company for the treatment of Homozygous Familial Hypercholesterolemia (HoFH) ?
  • How many emerging therapies are in the mid-stage and late stage of development for the treatment of Homozygous Familial Hypercholesterolemia (HoFH) ?
  • What are the key collaborations (Industry–Industry, Industry–Academia), Mergers and acquisitions, licensing activities related to the Homozygous Familial Hypercholesterolemia (HoFH) 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 Homozygous Familial Hypercholesterolemia (HoFH) and their status?
  • What are the key designations that have been granted for the emerging therapies for Homozygous Familial Hypercholesterolemia (HoFH) ?
  • What are the 7MM historical and forecasted market of Homozygous Familial Hypercholesterolemia (HoFH) ?

Reasons to buy

  • The report will help in developing business strategies by understanding trends shaping and driving the Homozygous Familial Hypercholesterolemia (HoFH).
  • To understand the future market competition in the Homozygous Familial Hypercholesterolemia (HoFH) market and Insightful review of the key market drivers and barriers.
  • Organize sales and marketing efforts by identifying the best opportunities for Homozygous Familial Hypercholesterolemia (HoFH) 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 Homozygous Familial Hypercholesterolemia (HoFH) market.
  • To understand the future market competition in the Homozygous Familial Hypercholesterolemia (HoFH) market.

1 KEY INSIGHTS

2 EXECUTIVE SUMMARY

3 HOMOZYGOUS FAMILIAL HYPERCHOLESTEROLEMIA (HOFH) MARKET OVERVIEW AT A GLANCE

3.1 MARKET SHARE (%) DISTRIBUTION IN 2017

3.2 MARKET SHARE (%) DISTRIBUTION IN 2030

4 DISEASE BACKGROUND AND OVERVIEW

4.1 INTRODUCTION

4.1.1 SYMPTOMS OF HOFH

4.2 GENETICS OF HOFH

4.2.1 Genetic heterogeneity translates to phenotypic variability

4.2.2 Metabolic characteristics of HoFH

4.3 DIAGNOSIS OF HOFH

5 RECOGNIZED ESTABLISHMENTS

6 TREATMENT AND MANAGEMENT

6.1.1 Lifestyle Modifications

6.1.2 Medication

6.1.3 LDL Apheresis

6.1.4 Liver Transplantation

6.1.5 Possible future treatments for HoFH

6.1.6 Treatment for Pediatric HoFH

7 HOMOZYGOUS FAMILIAL HYPERCHOLESTEROLEMIA: GUIDELINES BY EUROPEAN ATHEROSCLEROSIS SOCIETY (EAS) (2014)

7.1 DIAGNOSIS OF HOFH

7.1.1 Plasma low-density lipoprotein cholesterol levels

7.1.2 Xanthomas and arcus corneae

7.1.3 Family history

7.1.4 Differentiation from sitosterolaemia

7.1.5 Cardiovascular complications and natural history

7.1.6 Screening for subclinical atherosclerosis

7.2 CURRENT TREATMENT FOR HOFH

7.2.1 Pharmacotherapy

7.2.2 Lipoprotein apheresis

7.2.3 Liver transplantation and other surgical approaches

7.2.4 Other issues

7.2.5 Recommendations

8 JAPAN ATHEROSCLEROSIS SOCIETY (JAS) GUIDELINES FOR PREVENTION OF ATHEROSCLEROTIC CARDIOVASCULAR DISEASES 2017

8.1 DIAGNOSIS OF HOFH

8.1.1 Diagnostic Criteria

8.2 TREATMENT OF HOFH

8.2.1 Target Levels for Management

8.2.2 Lifestyle Modification

8.2.3 Drug Therapy

8.2.4 LDL Apheresis in HoFH

8.2.5 Pregnancy and Delivery of Patients with HoFH

8.2.6 HoFH Designated as an Intractable Disease

9 GUIDANCE FOR PEDIATRIC FAMILIAL HYPERCHOLESTEROLEMIA (2017)

9.1 DIAGNOSIS OF PEDIATRIC HOFH

9.1.1 Clinical Features of Pediatric HoFH

9.1.2 Genetic Diagnosis of HoFH

9.1.3 Important Notes for Diagnosis of HoFH

9.1.4 Differential Diagnosis

9.2 EVALUATION FOR ATHEROSCLEROTIC CARDIOVASCULAR DISEASES IN PEDIATRIC HOFH

9.2.1 Medical Questionnaire

9.2.2 Physical Findings

9.2.3 Biochemical Examination

9.2.4 Morphological Examinations

9.3 TREATMENT OF PEDIATRIC HOFH

9.3.1 Drug Therapy for Pediatric HoFH

10 TREATMENT ALGORITHM FOR HOFH

11 EPIDEMIOLOGY AND PATIENT POPULATION

11.1 KEY FINDINGS

11.2 7MM PREVALENT POPULATION OF HOMOZYGOUS FAMILIAL HYPERCHOLESTEROLEMIA

11.3 7MM DIAGNOSED PREVALENT POPULATION OF HOMOZYGOUS FAMILIAL HYPERCHOLESTEROLEMIA

12 7MM EPIDEMIOLOGY OF HOMOZYGOUS FAMILIAL HYPERCHOLESTEROLEMIA (HOFH)

12.1 ASSUMPTIONS AND RATIONALE

12.2 UNITED STATES

12.2.1 Prevalence of Homozygous Familial Hypercholesterolemia (HoFH) in the United States

12.2.2 Diagnosed Prevalence of Homozygous Familial Hypercholesterolemia in the United States

12.2.3 Mutation-specific Distribution of Homozygous Familial Hypercholesterolemia in the United States

12.3 EU5 COUNTRIES

12.4 GERMANY

12.4.1 Prevalence of Homozygous Familial Hypercholesterolemia in Germany

12.4.2 Diagnosed Prevalence of Homozygous Familial Hypercholesterolemia in Germany

12.4.3 Mutation-specific Distribution of Homozygous Familial Hypercholesterolemia in Germany

12.5 FRANCE

12.5.1 Prevalence of Homozygous Familial Hypercholesterolemia in France

12.5.2 Diagnosed Prevalence of Homozygous Familial Hypercholesterolemia in France

12.5.3 Mutation-specific Distribution of Homozygous Familial Hypercholesterolemia in France

12.6 ITALY

12.6.1 Prevalence of Homozygous Familial Hypercholesterolemia in Italy

12.6.2 Diagnosed Prevalence of Homozygous Familial Hypercholesterolemia in Italy

12.6.3 Mutation-specific Distribution of Homozygous Familial Hypercholesterolemia in Italy

12.7 SPAIN

12.7.1 Prevalence of Homozygous Familial Hypercholesterolemia in Spain

12.7.2 Diagnosed Prevalence of Homozygous Familial Hypercholesterolemia in Spain

12.7.3 Mutation-specific Distribution of Homozygous Familial Hypercholesterolemia in Spain

12.8 UNITED KINGDOM

12.8.1 Prevalence of Homozygous Familial Hypercholesterolemia in the United Kingdom

12.8.2 Diagnosed Prevalence of Homozygous Familial Hypercholesterolemia in the United Kingdom

12.8.3 Mutation-specific Distribution of Homozygous Familial Hypercholesterolemia in the United Kingdom

12.9 JAPAN

12.9.1 Prevalence of Homozygous Familial Hypercholesterolemia in Japan

12.9.2 Diagnosed Prevalence of Homozygous Familial Hypercholesterolemia in Japan

12.9.3 Mutation-specific Distribution of Homozygous Familial Hypercholesterolemia in Japan

13 UNMET NEEDS

14 MARKETED DRUGS

14.1 REPATHA (EVOLOCUMAB/AMG 145): AMGEN

14.1.1 Drug Description

14.1.2 Regulatory Milestones

14.1.3 Other Development Activities

14.1.4 Clinical Development

14.1.5 Clinical Trials Information

14.1.6 Safety and Efficacy

14.1.7 Product Profile

14.2 JUXTAPID (LOMITAPIDE): AEGERION PHARMACEUTICAL

14.2.1 Drug Description

14.2.2 Regulatory Milestones

14.2.3 Other Development Activities

14.2.4 Clinical Trials Information

14.2.5 Safety and Efficacy

14.2.6 Product Profile

15 EMERGING DRUGS

15.1 KEY COMPETITIORS

15.2 EVINACUMAB (REGN1500): REGENERON PHARMACEUTICALS

15.2.1 Drug Description

15.2.2 Other Development Activities

15.2.3 Clinical Development

15.2.4 Safety and Efficacy

15.2.5 Product Profile

15.3 INCLISIRAN: ALNYLAM PHARMACEUTICALS/NOVARTIS

15.3.1 Drug Description

15.3.2 Other Development Activities

15.3.3 Clinical Development

15.3.4 Clinical Trials Information

15.3.5 Safety and Efficacy

15.3.6 Product Profile

15.4 LIB003: LIB THERAPEUTICS

15.4.1 Drug Description

15.4.2 Clinical Development

15.4.3 Product Profile

15.5 PRALUENT (ALIROCUMAB): SANOFI/REGENERON PHARMACEUTICALS

15.5.1 Product Description

15.5.2 Other Developmental Activities

15.5.3 Clinical Development

15.5.4 Safety and Efficacy

15.5.5 Product Profile

15.6 GEMCABENE: NEUROBO PHARMACEUTICALS

15.6.1 Product Description

15.6.2 Other Developmental Activities

15.6.3 Clinical Development

15.6.4 Safety and Efficacy

15.6.5 Product Profile

15.7 ARO-ANG3: ARROWHEAD PHARMACEUTICALS

15.7.1 Product Description

15.7.2 Other Developmental Activities

15.7.3 Clinical Development

15.7.4 Safety and Efficacy

15.7.5 Product Profile

16 MARKET ACCESS AND REIMBURSEMENT

17 HOMOZYGOUS FAMILIAL HYPERCHOLESTROLEMIA (HOFH): SEVEN MAJOR MARKET ANALYSIS

17.1 KEY FINDINGS

17.2 MARKET SIZE OF HOMOZYGOUS FAMILIAL HYPERCHOLESTEROLEMIA (HOFH) IN THE 7MM

18 7MM MARKET OUTLOOK

18.1 UNITED STATES MARKET SIZE

18.1.1 Total Market size of Homozygous Familial Hypercholesterolemia

18.1.2 Market Size by Current Therapies

18.1.3 Market Size by Emerging Therapies

18.2 EU5 MARKET SIZE

18.3 GERMANY

18.3.1 Total Market size of Homozygous Familial Hypercholesterolemia

18.3.2 Market Size by Current Therapies

18.3.3 Market Size by Emerging Therapies

18.4 FRANCE

18.4.1 Total Market size of Homozygous Familial Hypercholesterolemia

18.4.2 Market Size by Current Therapies

18.4.3 Market Size by Emerging Therapies

18.5 ITALY

18.5.1 Total Market size of Homozygous Familial Hypercholesterolemia

18.5.2 Market Size by Current Therapies

18.5.3 Market Size by Emerging Therapies

18.6 SPAIN

18.6.1 Total Market size of Homozygous Familial Hypercholesterolemia

18.6.2 Market Size by Current Therapies

18.6.3 Market Size by EmergingTherapies

18.7 UNITED KINGDOM

18.7.1 Total Market size of Homozygous Familial Hypercholesterolemia

18.7.2 Market Size by Current Therapies

18.7.3 Market Size by Emerging Therapies

18.8 JAPAN MARKET SIZE

18.8.1 Total Market size of Homozygous Familial Hypercholesterolemia

18.8.2 Market Size by Current Therapies

18.8.3 Market Size by Emerging Therapies

19 CASE REPORTS

20 MARKET DRIVERS

21 MARKET BARRIERS

22 SWOT ANALYSIS

23 APPENDIX

23.1 BIBLIOGRAPHY

23.2 REPORT METHODOLOGY

24 DELVEINSIGHT CAPABILITIES

25 DISCLAIMER

26 ABOUT DELVEINSIGHT

List of Table

TABLE 1: SUMMARY OF HOFH, MARKET, EPIDEMIOLOGY AND KEY EVENTS (2017-2030)

TABLE 2: RECOGNIZED ESTABLISHMENTS

TABLE 3: CRITERIA FOR THE DIAGNOSIS OF HOFH

TABLE 4: CARDIOVASCULAR COMPLICATIONS OF HOFH

TABLE 5: SUMMARY OF EAS CONSENSUS PANEL RECOMMENDATIONS.

TABLE 6: DIAGNOSTIC CRITERIA FOR HOFH IN ADULTS (15 YEARS OF AGE OR OLDER)

TABLE 7: PREVALENT POPULATION OF HOFH IN THE 7MM (2017-2030)

TABLE 8: DIAGNOSED PREVALENT POPULATION OF HOFH IN THE 7MM (2017-2030)

TABLE 9: PREVALENCE OF HOFH IN THE US (2017-2030)

TABLE 10: DIAGNOSED PREVALENCE OF HOFH IN THE US (2017-2030)

TABLE 11: MUTATION-SPECIFIC DISTRIBUTION OF HOFH IN THE US (2017-2030)

TABLE 12: PREVALENCE OF HOFH IN GERMANY (2017-2030)

TABLE 13: DIAGNOSED PREVALENCE OF HOFH IN GERMANY (2017-2030)

TABLE 14: MUTATION-SPECIFIC DISTRIBUTION OF HOFH IN GERMANY (2017-2030)

TABLE 15: PREVALENCE OF HOFH IN FRANCE (2017-2030)

TABLE 16: DIAGNOSED PREVALENCE OF HOFH IN FRANCE (2017-2030)

TABLE 17: MUTATION-SPECIFIC DISTRIBUTION OF HOFH IN FRANCE (2017-2030)

TABLE 18: PREVALENCE OF HOFH IN ITALY (2017-2030)

TABLE 19: DIAGNOSED PREVALENCE OF HOFH IN ITALY (2017-2030)

TABLE 20: MUTATION-SPECIFIC DISTRIBUTION OF HOFH IN ITALY (2017-2030)

TABLE 21: PREVALENCE OF HOFH IN SPAIN (2017-2030)

TABLE 22: DIAGNOSED PREVALENCE OF HOFH IN SPAIN (2017-2030)

TABLE 23: MUTATION-SPECIFIC DISTRIBUTION OF HOFH IN SPAIN (2017-2030)

TABLE 24: PREVALENCE OF HOFH IN THE UK (2017-2030)

TABLE 25: DIAGNOSED PREVALENCE OF HOFH IN THE UK (2017-2030)

TABLE 26: MUTATION-SPECIFIC DISTRIBUTION OF HOFH IN THE UK (2017-2030)

TABLE 27: PREVALENCE OF HOFH IN JAPAN (2017-2030)

TABLE 28: DIAGNOSED PREVALENCE OF HOFH IN JAPAN (2017-2030)

TABLE 29: MUTATION-SPECIFIC DISTRIBUTION OF HOFH IN JAPAN (2017-2030)

TABLE 30: REPATHA (EVOLOCUMAB/AMG 145), CLINICAL TRIAL DESCRIPTION, 2020

TABLE 31: JUXTAPID (LOMITAPIDE), CLINICAL TRIAL DESCRIPTION, 2020

TABLE 32: COMPARISON OF EMERGING DRUGS UNDER DEVELOPMENT

TABLE 33: EVINACUMAB (REGN1500), CLINICAL TRIAL DESCRIPTION

TABLE 34: INCLISIRAN, CLINICAL TRIAL DESCRIPTION, 2020

TABLE 35: LIB003, CLINICAL TRIAL DESCRIPTION, 2020

TABLE 36: PRALUENT, CLINICAL TRIAL DESCRIPTION, 2020

TABLE 37: GEMCABENE, CLINICAL TRIAL DESCRIPTION, 2020

TABLE 38: ARO-ANG3, CLINICAL TRIAL DESCRIPTION

TABLE 39: SEVEN MAJOR MARKET SIZE OF HOFH IN USD MILLION (2017-2030)

TABLE 40: TOTAL MARKET SIZE OF HOFH IN THE UNITED STATES, IN USD MILLION (2017-2030)

TABLE 41: MARKET SIZE OF HOFH BY CURRENT THERAPIES IN THE UNITED STATES, IN USD MILLION (2017-2030)

TABLE 42: MARKET SIZE OF HOFH BY EMERGING THERAPIES IN THE UNITED STATES, IN USD MILLION (2017-2030)

TABLE 43: TOTAL MARKET SIZE OF HOFH IN GERMANY, IN USD MILLION (2017-2030)

TABLE 44: MARKET SIZE OF HOFH BY CURRENT THERAPIES IN GERMANY, IN USD MILLION (2017-2030)

TABLE 45: MARKET SIZE OF HOFH BY EMERGING THERAPIES IN GERMANY, IN USD MILLION (2017-2030)

TABLE 46: TOTAL MARKET SIZE OF HOFH IN FRANCE, IN USD MILLION (2017-2030)

TABLE 47: MARKET SIZE OF HOFH BY CURRENT THERAPIES IN FRANCE, IN USD MILLION (2017-2030)

TABLE 48: MARKET SIZE OF HOFH BY EMERGING THERAPIES IN FRANCE, IN USD MILLION (2017-2030)

TABLE 49: TOTAL MARKET SIZE OF HOFH IN ITALY, IN USD MILLION (2017-2030)

TABLE 50: MARKET SIZE OF HOFH BY CURRENT THERAPIES IN ITALY, IN USD MILLION (2017-2030)

TABLE 51: MARKET SIZE OF HOFH BY EMERGING THERAPIES IN ITALY, IN USD MILLION (2017-2030)

TABLE 52: TOTAL MARKET SIZE OF HOFH IN SPAIN, IN USD MILLION (2017-2030)

TABLE 53: MARKET SIZE OF HOFH BY CURRENT THERAPIES IN SPAIN, IN USD MILLION (2017-2030)

TABLE 54: MARKET SIZE OF HOFH BY EMERGING THERAPIES IN SPAIN, IN USD MILLION (2017-2030)

TABLE 55: TOTAL MARKET SIZE OF HOFH IN THE UNITED KINGDOM, IN USD MILLION (2017-2030)

TABLE 56: MARKET SIZE OF HOFH BY CURRENT THERAPIES IN THE UNITED KINGDOM, IN USD MILLION (2017-2030)

TABLE 57: MARKET SIZE OF HOFH BY EMERGING THERAPIES IN THE UNITED KINGDOM, IN USD MILLION (2017-2030)

TABLE 58: TOTAL MARKET SIZE OF HOFH IN JAPAN, IN USD MILLION (2017-2030)

TABLE 59: MARKET SIZE OF HOFH BY CURRENT THERAPIES IN JAPAN, IN USD MILLION (2017-2030)

TABLE 60: MARKET SIZE OF HOFH BY EMERGING THERAPIES IN JAPAN, IN USD MILLION (2017-2030)

List of Figures

FIGURE 1: PROTEINS AFFECTING LOW-DENSITY LIPOPROTEIN RECEPTOR FUNCTION.

FIGURE 2: GENETICS AND GENETIC HETEROGENEITY OF HOFH

FIGURE 3: MECHANISMS OF ACTIONS OF LIPID-LOWERING THERAPIES

FIGURE 4: CLINICAL GENETIC TESTING FOR FAMILIAL HYPERCHOLESTEROLEMIA

FIGURE 5: ALGORITHM OF TREATMENT OF PEDIATRIC HOFH

FIGURE 6: TREATMENT FLOW CHART FOR ADULT (15 YEARS OR OVER) HOFH

FIGURE 7: PREVALENT POPULATION OF HOFH IN THE 7MM (2017-2030)

FIGURE 8: DIAGNOSED PREVALENT POPULATION OF HOFH IN THE 7MM (2017-2030)

FIGURE 9: PREVALENCE OF HOFH IN THE US (2017-2030)

FIGURE 10: DIAGNOSED PREVALENCE OF HOFH IN THE US (2017-2030)

FIGURE 11: MUTATION-SPECIFIC DISTRIBUTION OF HOFH IN THE US (2017-2030)

FIGURE 12: PREVALENCE OF HOFH IN GERMANY (2017-2030)

FIGURE 13: DIAGNOSED PREVALENCE OF HOFH IN GERMANY (2017-2030)

FIGURE 14: MUTATION-SPECIFIC DISTRIBUTION OF HOFH IN GERMANY (2017-2030)

FIGURE 15: PREVALENCE OF HOFH IN FRANCE (2017-2030)

FIGURE 16: DIAGNOSED PREVALENCE OF HOFH IN FRANCE (2017-2030)

FIGURE 17: MUTATION-SPECIFIC DISTRIBUTION OF HOFH IN FRANCE (2017-2030)

FIGURE 18: PREVALENCE OF HOFH IN ITALY (2017-2030)

FIGURE 19: DIAGNOSED PREVALENCE OF HOFH IN ITALY (2017-2030)

FIGURE 20: MUTATION-SPECIFIC DISTRIBUTION OF HOFH IN ITALY (2017-2030)

FIGURE 21: PREVALENCE OF HOFH IN SPAIN (2017-2030)

FIGURE 22: DIAGNOSED PREVALENCE OF HOFH IN SPAIN (2017-2030)

FIGURE 23: MUTATION-SPECIFIC DISTRIBUTION OF HOFH IN SPAIN (2017-2030)

FIGURE 24: PREVALENCE OF HOFH IN THE UK (2017-2030)

FIGURE 25: DIAGNOSED PREVALENCE OF HOFH IN THE UK (2017-2030)

FIGURE 26: MUTATION-SPECIFIC DISTRIBUTION OF HOFH IN THE UK (2017-2030)

FIGURE 27: PREVALENCE OF HOFH IN JAPAN (2017-2030)

FIGURE 28: DIAGNOSED PREVALENCE OF HOFH IN JAPAN (2017-2030)

FIGURE 29: MUTATION-SPECIFIC DISTRIBUTION OF HOFH IN JAPAN (2017-2030)

FIGURE 30: UNMET NEEDS OF HOFH

FIGURE 31: SEVEN MAJOR MARKET SIZE OF HOFH IN USD MILLION (2017-2030)

FIGURE 32: TOTAL MARKET SIZE OF HOFH IN THE UNITED STATES, USD MILLIONS (2017-2030)

FIGURE 33: MARKET SIZE OF HOFH BY CURRENT THERAPIES IN THE UNITED STATES, IN USD MILLION (2017-2030)

FIGURE 34: MARKET SIZE OF HOFH BY EMERGING THERAPIES IN THE UNITED STATES, IN USD MILLION (2017-2030)

FIGURE 35: TOTAL MARKET SIZE OF HOFH IN GERMANY, USD MILLIONS (2017-2030)

FIGURE 36: MARKET SIZE OF HOFH BY CURRENT THERAPIES IN GERMANY, IN USD MILLION (2017-2030)

FIGURE 37: MARKET SIZE OF HOFH BY EMERGING THERAPIES IN GERMANY, IN USD MILLION (2017-2030)

FIGURE 38: TOTAL MARKET SIZE OF HOFH IN FRANCE, USD MILLIONS (2017-2030)

FIGURE 39: MARKET SIZE OF HOFH BY CURRENT THERAPIES IN FRANCE, IN USD MILLION (2017-2030)

FIGURE 40: MARKET SIZE OF HOFH BY EMERGING THERAPIES IN FRANCE, IN USD MILLION (2017-2030)

FIGURE 41: TOTAL MARKET SIZE OF HOFH IN ITALY, USD MILLIONS (2017-2030)

FIGURE 42: MARKET SIZE OF HOFH BY CURRENT THERAPIES IN ITALY, IN USD MILLION (2017-2030)

FIGURE 43: MARKET SIZE OF HOFH BY EMERGING THERAPIES IN ITALY, IN USD MILLION (2017-2030)

FIGURE 44: TOTAL MARKET SIZE OF HOFH IN SPAIN, USD MILLIONS (2017-2030)

FIGURE 45: MARKET SIZE OF HOFH BY CURRENT THERAPIES IN SPAIN, IN USD MILLION (2017-2030)

FIGURE 46: MARKET SIZE OF HOFH BY EMERGING THERAPIES IN SPAIN, IN USD MILLION (2017-2030)

FIGURE 47: TOTAL MARKET SIZE OF HOFH IN THE UNITED KINGDOM, USD MILLIONS (2017-2030)

FIGURE 48: MARKET SIZE OF HOFH BY CURRENT THERAPIES IN THE UNITED KINGDOM, IN USD MILLION (2017-2030)

FIGURE 49: MARKET SIZE OF HOFH BY EMERGING THERAPIES IN THE UNITED KINGDOM, IN USD MILLION (2017-2030)

FIGURE 50: TOTAL MARKET SIZE OF HOFH IN JAPAN, USD MILLIONS (2017-2030)

FIGURE 51: MARKET SIZE OF HOFH BY CURRENT THERAPIES IN JAPAN, IN USD MILLION (2017-2030)

FIGURE 52: MARKET SIZE OF HOFH BY EMERGING THERAPIES IN JAPAN, IN USD MILLION (2017-2030)

FIGURE 53: MARKET DRIVERS

FIGURE 54: MARKET BARRIERS

FIGURE 55: SWOT ANALYSIS OF HOFH

Amgen

Aegerion Pharmaceutical

Regeneron Pharmaceuticals

Alnylam Pharmaceuticals/Novartis

LIB Therapeutics

Sanofi/Regeneron Pharmaceuticals

Neurobo Pharmaceuticals

Arrowhead Pharmaceuticals

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