家族性カイロミクロン血症症候群(FCS)治療薬の世界市場:インサイト・疫学・市場予測

◆英語タイトル:Familial Chylomicronemia Syndrome (FCS) - Market Insights, Epidemiology and Market Forecast - 2030
◆商品コード:DELV20JU157
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◆発行日:2020年5月
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【レポートの概要】

DelveInsight’s ‘Familial Chylomicronemia Syndrome (FCS) Market Insights, Epidemiology and Market Forecast-2030’ report delivers an in-depth understanding of the FCS, historical and forecasted epidemiology as well as the FCS market trends in the United States, EU5 (Germany, France, Italy, Spain, and United Kingdom), and Japan.
The FCS market report provides current treatment practices, emerging drugs, FCS market share of the individual therapies, current and forecasted FCS market size from 2017 to 2030 segmented by seven major markets. The Report also covers current FCS treatment practice/algorithm, market drivers, market barriers and unmet medical needs to curate best of the opportunities and assesses 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 Chylomicronemia Syndrome (FCS): Disease Understanding and Treatment Algorithm
Familial Chylomicronemia Syndrome Overview

Familial Chylomicronemia Syndrome (FCS) is a rare genetic lipid disorder that is associated with the buildup of chylomicrons (a large lipoprotein particle that transports dietary fat and cholesterol in the body). It is also known as lipoprotein lipase deficiency (LPLD), Type 1 hyperlipoproteinemia, endogenous hypertriglyceridemia, familial fat-induced hypertriglyceridemia, familial hyperchylomicronemia, and familial LPL deficiency, hyperlipidemia Type I (fredrickson), hyperlipoproteinemia Type IA, lipase D deficiency, and Burger-Grutz syndrome.

The normal triglyceride levels are <150 mg/dL (or 1.7 mmol/L), but as the patients with FSC are incapable of breaking down chylomicrons (CM) that are rich in triglycerides, the levels can be as high as 10–20 times the normal threshold. FCS patients are at risk of severe recurrent abdominal pain and potentially fatal pancreatitis, long-term complications from pancreatic damage, and symptoms that can interfere with daily living. The leading cause behind the increase in the CM is the reduction or absence of LPL, which is an enzyme that helps to break down CM in the body. The patients with FCS encounter several severe symptoms before it is diagnosed.

The signs and symptoms that patients may experience include xanthomas (fatty deposits in the skin, usually on buttocks, knees, and arms), hepatosplenomegaly (swelling of liver and spleen), lipemia retinalis (milky appearance of retinal veins and arteries), and neurological symptoms such as depression and memory loss.

Familial Chylomicronemia Syndrome Diagnosis
The clinical progression of FCS is unclear, in some cases, the patient may develop symptoms, including abdominal pain, quiet early in life, but may not be diagnosed until they are in their teens or 20’s when the pancreatitis attacks become more frequent. Due to disease rarity and lack of understanding, FCS is often misdiagnosed or undiagnosed; however, the most number of patients have their diagnosis confirmed by a genetic test. Recently FCS score is developed to combat with misdiagnosis of FCS with multifactorial chylomicronemia syndrome (MCS).

Familial Chylomicronemia Syndrome Treatment
Currently, there is no US Food and Drug Administration (FDA) approved therapy, however, the European Medicines Agency (EMA) had granted conditional marketing authorization to Waylivra in the European Union (EU) in 2019.

The current standard of care is mainly focused on reducing triglyceride levels by severely restricting dietary intake of fat allied with triglycerides lowering agents. Many guidelines suggest that adults with FCS should limit dietary intake of total fat to levels as low as 15 g/day.

Familial Chylomicronemia Syndrome (FCS) Epidemiology
The FCS epidemiology division provide the insights about historical and current FCS patient pool and forecasted trend for each seven major countries. It helps to recognize the causes of current and forecasted trends by exploring numerous studies and views of key opinion leaders. This part of the

DelveInsight report also provides the diagnosed patient pool and their trends along with assumptions undertaken.

Key Findings

The total prevalent cases of FCS in the 7MM were found to be 5,801 in 2017 which is expected grow during the study period, i.e., 2017–2030.

The disease epidemiology covered in the report provides historical as well as forecasted FCS epidemiology [segmented as Total Prevalence of FCS, Total Diagnosed Prevalence of FCS, Age-specific Diagnosed Prevalence of FCS, Severity- Specific Diagnosed Prevalence of FCS, and Treated Patient Pool of FCS] scenario of FCS in the 7MM covering United States, EU5 countries (Germany, France, Italy , Spain, and United Kingdom), and Japan from 2017 to 2030.

Country Wise- Familial Chylomicronemia Syndrome Epidemiology

Estimates show that the highest prevalent population of FCS is in the United States, followed by Japan, Germany, France, and the United Kingdom in 2017.

Familial Chylomicronemia Syndrome Drug Chapters
Drug chapter segment of the Familial Chylomicronemia Syndrome report encloses the detailed analysis of Familial Chylomicronemia Syndrome marketed drugs and late stage (Phase-III and Phase-II) pipeline drugs. It also helps to understand the Familial Chylomicronemia Syndrome clinical trial details, expressive pharmacological action, agreements and collaborations, approval and patent details, advantages and disadvantages of each included drug and the latest news and press releases.

Familial Chylomicronemia Syndrome Marketed Drugs

Waylivra (volanesorsen): Ionis Pharmaceuticals/Akcea Therapeutic

Waylivra (Volanesorsen) is a 2’-O-(2-methoxyethyl) antisense oligonucleotide (ASO) inhibitor of the molecular target apolipoprotein C-III (APOC-III). Volanesorsen is delivered as a subcutaneous injection of 280 mg/1.5 mL weekly in a single-use pre-filled syringe.

Waylivra is indicated as an adjunct to diet for the treatment of patients with FCS. In May 2019, Akcea and Ionis have received conditional marketing authorization from the European Commission (EC) for the treatment of FCS for Waylivra. The company has launched Waylivra in Germany, however, launch in other European countries is expected in 2020. The company has also initiated ATU (Autorisation Temporaire d’Utilisation | Temporary Use Authorization) in France. This drug is not approved by the US FDA. Also recently in January 2020, NICE rejected Waylivra for the treatment of FCS.

Waylivra is a product of Ionis’ proprietary antisense technology. Antisense technology is an innovative platform for discovering first-in-class and/or best-in-class medicines for treating disease. Unlike most other drug technologies that work by affecting existing proteins in the body, antisense medicines target RNA, the intermediary that conveys genetic information from a gene to the protein synthesis machinery in the cell. By targeting RNA instead of proteins, antisense technology can be used to increase, decrease or alter the production of specific proteins.

Products detail in the report…

Familial Chylomicronemia Syndrome Emerging Drugs

AKCEA-ANGPTL3-LRx: Akcea Therapeutics/Ionis Pharmaceuticals

AKCEA-ANGPTL3-LRx (ISIS 703802) is under development by Akcea and Ionis. It is a subcutaneously administered investigational antisense therapy being developed to treat patients with certain cardiovascular and metabolic diseases.

AKCEA-ANGPTL3-LRx is designed to reduce the production of angiopoietin-like 3 (ANGPTL3) protein in the liver. It is a key regulator of triglycerides, cholesterol, and glucose and energy metabolism. This drug was developed using Ionis’s advanced Ligand Conjugated Antisense (LICA) technology platform.

ARO-APOC3: Arrowhead Pharmaceuticals

ARO-APOC3 which is under development by Arrowhead Pharmaceuticals, is a subcutaneously administered RNAi therapeutic targeting Apolipoprotein C-III (APOC3). The company utilizes Targeted RNAi Molecule (TRiMTM) platform for ligand-mediated delivery of this drug. This is mainly designed to enable tissue-specific targeting while being structurally simple. This mechanism offers many potential advantages in the development of disease therapies, such as the ability to target a broad range of genes and proteins with high specificity, and also the disease pathways that have proven difficult to address with traditional small molecule and biologic therapeutics.

AKCEA -APOCIII – LRx: Ionis Pharmaceuticals/Akcea Therapeutics

AKCEA-APOCIII-LRx which is under development by Ionis and Akcea, is a subcutaneously administered generation 2+ ligand-conjugated antisense (LICA) drug designed to inhibit the production of apoC-III, for patients who are at risk of disease due to elevated triglyceride levels.

In January 2020, based on the positive results of phase II trial for hypertriglyceridemia and cardiovascular diseases company announced to pursue the rapid development of AKCEA- APOCIII-LRx for FCS. Results of phase II clinical trial has shown favorable safety and tolerability in hypertriglyceridemia patients. Both primary and key secondary endpoints were met with significant reduction in apoC-III and triglyceride levels. Furthermore, more than 90% of patients achieved serum triglycerides ≤ 150 mg/dL at the highest monthly dose.

Products detail in the report…

Familial Chylomicronemia Syndrome Market Outlook
Current treatment choices for FCS can be fragmented down into managing acute crisis related to pancreatitis and chronic management of hypertriglyceridemia to reduce the risk of future episodes. During an episode of acute pancreatitis, complete fasting with parenteral fluid support and analgesia if required is usually very effective.

There is a lack of approved therapies for FCS in the market. As of now, no drug is approved by the United States (Food and Drug Administration) FDA for the management of FCS. Likewise, no therapy is approved in Japan as well.

Only one approved drug for the treatment FCS is currently present which was approved by the EC in Europe is Waylivra. Glybera was the another drug which was approved in October 2012 by European Comission under exceptional circumstances as a treatment for small subsets of adult patients diagnosed with familial LPL deficiency, which was confirmed by genetic testing. But later it was withdrawn from the market by uniQure in October 2017.

Key Findings
According to DelveInsight, Familial Chylomicronemia Syndrome market in the 7MM is expected to change in the study period 2017–2030. The therapeutic market of Familial Chylomicronemia Syndrome in seven major markets was found to be USD 1.0 million in 2017 which is expected to increase during study period (2017–2030).

The United States Market Outlook

In 2017, the total market size of FCS therapies was found to be USD 0.3 million in the United States which is expected to increase in the study period (2017–2030).

EU-5 Countries: Market Outlook

In 2017, the total market size of FCS therapies was found to be USD 0.5 million in the EU-5 countries which is expected to increase in the study period (2017–2030).

Japan Market Outlook

The total market size of FCS therapies in Japan was found to be USD 0.2 million in 2017.

Familial Chylomicronemia Syndrome Pipeline Development Activities
The drugs which are in pipeline includes:

1. AKCEA-ANGPTL3-LRx (Akcea Therapeutics/Ionis Pharmaceuticals): Phase II

2. Lomitapide (Amryt Pharma/Novelion Therapeutics): Phase II

3. AKCEA -APOCIII – LRx (Ionis Pharmaceuticals/Akcea Therapeutics): Phase II

4. ARO-APOC3 (Arrowhead Pharmaceuticals): Phase I/IIa

Pipeline Development Activities

Key Points:

The AKCEA-ANGPTL3-LRx Phase II program was designed to include three clinical studies in patients with one of three rare hyperlipidemias, including FCS, Familial Partial Lipodystrophy (FPL), and Homozygous Familial Hypercholesterolemia (HoFH).]
Arrowhead Pharmaceuticals is planning to initiate pivotal studies of ARO-APOC3 in 2020 for FCS.
Amryt Pharma and Novelion Therapeutics is expecting the launch of Lomitapide in 2023 for the treatment of FCS as mentioned in its May 2019 annual report of the company.
In January 2020, based on the positive results of phase II trial for hypertriglyceridemia and cardiovascular diseases Ionis Pharmaceuticals and Akcea Therapeutics announced to pursue the rapid development of AKCEA- APOCIII-LRx for FCS.
Familial Chylomicronemia Syndrome Drugs Uptake
In February 2020, Arrowhead Pharmaceuticals reported the interim results for ARO-APOC3 which shows about 95% of reductions in the elevated triglyceride results. Based on its efficacy and safety results this therapy expected to impact the upcoming therapeutic market of FCS significantly.

Access and Reimbursement Scenario in Familial Chylomicronemia Syndrome Therapies:

For Waylivra early access program is ongoing in Europe, the US and Canada and in March 2018, Medicines and Healthcare Products Regulatory Agency (MHRA) granted a positive scientific opinion through the Early Access to Medicines Scheme (EAMS) for the treatment of FCS. According to the decision, eligible patients with FCS are able to access volanesorsen before the EC makes a formal decision for its use in Europe.
According to the recommendation by National Institute for Health and Care Excellence (NICE) given in January 2020. Waylivra is not recommended, within its marketing authorization, for treating FCS in adults with genetically confirmed FCS who are at high risk of pancreatitis, and when the response to diet and triglyceride-lowering therapy has been inadequate.
It was also stated by NICE that this recommendation is not intended to affect treatment with Waylivra that was started in the NHS before this guidance was published. People having treatment outside this recommendation may continue without change to the funding arrangements in place for them before this guidance was published, until they and their NHS clinician consider it appropriate to stop.
KOL- Views
To keep up with current market trends, we take KOLs and SME’s opinion working in Familial Chylomicronemia Syndrome domain through primary research to fill the data gaps and validate our secondary research. Their opinion helps to understand and validate current and emerging therapies treatment patterns or Familial Chylomicronemia Syndrome market trend. This will support the clients in potential upcoming novel treatment by identifying the overall scenario of the market and the unmet needs.

Competitive Intelligence Analysis
We perform Competitive and Market Intelligence analysis of the Familial Chylomicronemia Syndrome Market by using various Competitive Intelligence tools that includes – 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 Chylomicronemia Syndrome, explaining its causes, signs and symptoms, pathophysiology and currently available therapies.
Comprehensive insight has been provided into the Familial Chylomicronemia Syndrome epidemiology and treatment in the 7MM.
Additionally, an all-inclusive account of both the current and emerging therapies for Familial Chylomicronemia Syndrome are provided, along with the assessment of new therapies, which will have an impact on the current treatment landscape.
A detailed review of Familial Chylomicronemia Syndrome market; historical and forecasted is included in the report, covering drug outreach in the 7MM.
The report provides an edge while developing business strategies, by understanding trends shaping and driving the global Familial Chylomicronemia Syndrome market.
Report Highlights
In the coming years, Familial Chylomicronemia Syndrome 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 Chylomicronemia Syndrome R&D. The therapies under development are focused on novel approaches to treat/improve the disease condition.
Major players are involved in developing therapies for Familial Chylomicronemia Syndrome. Launch of emerging therapies, will significantly impact the Familial Chylomicronemia Syndrome market.
A better understanding of disease pathogenesis will also contribute to the development of novel therapeutics for Familial Chylomicronemia Syndrome.
Our in-depth analysis of the pipeline assets across different stages of development (Phase III and Phase II), different emerging trends and comparative analysis of pipeline products with detailed clinical profiles, key cross-competition, launch date along with product development activities will support the clients in the decision-making process regarding their therapeutic portfolio by identifying the overall scenario of the research and development activities.
Familial Chylomicronemia Syndrome Report Insights
Patient Population
Therapeutic Approaches
Familial Chylomicronemia Syndrome Pipeline Analysis
Familial Chylomicronemia Syndrome Market Size and Trends
Market Opportunities
Impact of upcoming Therapies
Familial Chylomicronemia Syndrome Report Key Strengths
11 Years Forecast
7MM Coverage
Familial Chylomicronemia Syndrome Epidemiology Segmentation
Key Cross Competition
Highly Analyzed Market
Drugs Uptake
Familial Chylomicronemia Syndrome Report Assessment
Current Treatment Practices
Unmet Needs
Pipeline Product Profiles
Market Attractiveness
Market Drivers and Barriers
Key Questions
Market Insights:

What was the Familial Chylomicronemia Syndrome Market share (%) distribution in 2017 and how it would look like in 2030?
What would be the Familial Chylomicronemia Syndrome total market Size as well as market size by therapies across the 7MM during the study period (2017–2030)?
What are the key findings pertaining to the market across the 7MM and which country will have the largest Familial Chylomicronemia Syndrome market size during the study period (2017–2030)?
At what CAGR, the Familial Chylomicronemia Syndrome market is expected to grow in 7MM during the study period (2017–2030)?
What would be the Familial Chylomicronemia Syndrome market outlook across the 7MM during the study period (2017–2030)?
What would be the Familial Chylomicronemia Syndrome 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 a subsequent analysis of the associated trends?
Familial Chylomicronemia Syndrome patient types/ pool where unmet need is more and whether emerging therapies will be able to address the residual unmet need?
How emerging therapies are performing on the parameters like efficacy, safety, route of administration (RoA), treatment duration and frequencies on the basis of their clinical trial results?
Among the emerging therapies, what are the potential therapies which are expected to disrupt the Familial Chylomicronemia Syndrome market?
Epidemiology Insights:

What is the disease risk, burden and unmet needs of the Familial Chylomicronemia Syndrome?
What is the historical Familial Chylomicronemia Syndrome patient pool in seven major markets covering the United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom) and Japan?
What would be the forecasted patient pool of Familial Chylomicronemia Syndrome in 7 major markets covering the United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom) and Japan?
What will be the growth opportunities in the 7MM with respect to the patient population pertaining to Familial Chylomicronemia Syndrome?
Out of all the 7MM countries, which country would have the highest prevalent population of Familial Chylomicronemia Syndrome during the study period (2017–2030)?
At what CAGR the population is expected to grow in the 7MM during the study period (2017–2030)?
What are the various recent and upcoming events which are expected to improve the diagnosis of Familial Chylomicronemia Syndrome?
Percentage of Familial Chylomicronemia Syndrome patients who are significantly affected in terms of their self-worth, emotional well-being, sleep, and mental functioning?
Current Treatment Scenario, Marketed Drugs and Emerging Therapies:
What are the current options for the treatment of Familial Chylomicronemia Syndrome along with the approved therapy?
What are the current treatment guidelines for the treatment of Familial Chylomicronemia Syndrome in the US, Europe and Japan?
What are the Familial Chylomicronemia Syndrome 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 Chylomicronemia Syndrome?
How many therapies are developed by each company for the treatment of Familial Chylomicronemia Syndrome?
How many emerging therapies are in mid stage, and late stage of development for the treatment of Familial Chylomicronemia Syndrome?
What are the key collaborations (Industry–Industry, Industry–Academia), Mergers and acquisitions, licensing activities related to the Familial Chylomicronemia Syndrome 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 Chylomicronemia Syndrome and their status?
What are the key designations that have been granted for the emerging therapies for Familial Chylomicronemia Syndrome?
What are the global historical and forecasted market of Familial Chylomicronemia Syndrome?

Reasons to buy
The report will help in developing business strategies by understanding trends shaping and driving the Familial Chylomicronemia Syndrome market.
To understand the future market competition in the Familial Chylomicronemia Syndrome market and Insightful review of the key market drivers and barriers.
Organize sales and marketing efforts by identifying the best opportunities for Familial Chylomicronemia Syndrome in the US, Europe (Germany, France, Italy, Spain, and the United Kingdom) and Japan.
Identification of strong upcoming players in 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 Chylomicronemia Syndrome market.
To understand the future market competition in the Familial Chylomicronemia Syndrome market.

What is Familial Chylomicronemia Syndrome?

Familial Chylomicronemia Syndrome is a rare genetic lipid disorder that is associated with the buildup of chylomicrons (a large lipoprotein particle that transports dietary fat and cholesterol in the body). It is also known as lipoprotein lipase deficiency (LPLD), Type 1 hyperlipoproteinemia, endogenous hypertriglyceridemia, familial fat-induced hypertriglyceridemia, familial hyperchylomicronemia, and familial LPL deficiency, hyperlipidemia Type I (fredrickson), hyperlipoproteinemia Type IA, lipase D deficiency, and Burger-Grutz syndrome

How is epidemiology segmented for Familial Chylomicronemia Syndrome?

Total Prevalence of Familial Chylomicronemia Syndrome, Total Diagnosed Prevalence of Familial Chylomicronemia Syndrome, Age-specific Diagnosed Prevalence of Familial Chylomicronemia Syndrome, Severity- Specific Diagnosed Prevalence of Familial Chylomicronemia Syndrome, and Treated Patient Pool of Familial Chylomicronemia Syndrome

What are the total prevalent cases of Familial Chylomicronemia Syndrome in 2017?

The total prevalent cases of Familial Chylomicronemia Syndrome in the 7MM were found to be 5,801 in 2017

What was the Familial Chylomicronemia Syndrome market in 2017?

The market of Familial Chylomicronemia Syndrome in seven major markets was found to be USD 1.0 million in 2017

Which companies are developing drugs for Familial Chylomicronemia Syndrome?

Akcea Therapeutics, Ionis Pharmaceuticals, Arrowhead Pharmaceuticals are developing drugs for Familial Chylomicronemia Syndrome

【レポートの目次】

1. Key Insights

2. Executive Summary of FCS

3. SWOT Analysis for FCS

4. FCS: Patient Share Overview at a Glance

4.1. Total Patient Share (%) Distribution of FCS in 2017

4.2. Total Patient Share (%) Distribution of FCS in 2030

5. FCS: Market Overview at a Glance

5.1. Total Market Share (%) Distribution of FCS in 2017

5.2. Total Market Share (%) Distribution of FCS in 2030

6. Familial Chylomicronemia Syndrome (FCS): Disease Background and Overview

6.1. Introduction

6.1.1. Signs and Symptoms of FCS

6.1.2. Genetics and Pathogenesis of FCS

6.2. Diagnosis of FCS

6.2.1. Diagnostic Algorithm

7. Epidemiology and Patient Population

7.1. Key Findings: 7MM

7.2. Assumptions and Rationale: 7MM

7.3. Total Prevalence of FCS in the 7MM

7.4. Total Diagnosed Prevalence of FCS in the 7MM

7.5. Age-specific Diagnosed Prevalence of FCS in the 7MM

8. United States

8.1. Total Prevalent cases of FCS in the United States

8.2. Total Diagnosed Prevalent cases of FCS in the United States

8.3. Severity- Specific Diagnosed Prevalence of FCS in the United States

8.4. Treated Patient Pool of FCS in the United States

9. EU-5 Country-wise Epidemiology

9.1. Germany

9.1.1. Total Prevalent cases of FCS in Germany

9.1.2. Total Diagnosed Prevalent cases of FCS in Germany

9.1.3. Severity- Specific Diagnosed Prevalence of FCS in Germany

9.1.4. Treated Patient Pool of FCS in Germany

9.2. France

9.2.1. Total Prevalent cases of FCS in France

9.2.2. Total Diagnosed Prevalent cases of FCS in France

9.2.3. Severity- Specific Diagnosed Prevalence of FCS in France

9.2.4. Treated Patient Pool of FCS in France

9.3. Italy

9.3.1. Total Prevalent cases of FCS in Italy

9.3.2. Total Diagnosed Prevalent cases of FCS in Italy

9.3.3. Severity-Specific Prevalence of FCS in Italy

9.3.4. Treated Patient Pool of FCS in Italy

9.4. Spain

9.4.1. Total Prevalent cases of FCS in Spain

9.4.2. Total Diagnosed Prevalent cases of FCS in Spain

9.4.3. Severity- Specific Diagnosed Prevalence of FCS in Spain

9.4.4. Treated Patient Pool of FCS in Spain

9.5. United Kingdom

9.5.1. Total Prevalent cases of FCS in the United Kingdom

9.5.2. Total Diagnosed Prevalent cases of FCS in the United Kingdom

9.5.3. Severity- Specific Diagnosed Prevalence of FCS in the United Kingdom

9.5.4. Treated Patient Pool of FCS in the United Kingdom

10. Japan

10.1. Total Prevalent cases of FCS in Japan

10.2. Total Diagnosed Prevalent cases of FCS in Japan

10.3. Severity- Specific Diagnosed Prevalence of FCS in Japan

10.4. Treated Patient Pool of FCS in Japan

11. Current Treatment Practices for FCS

11.1. Novel Therapies

11.2. Guidelines for FCS

11.3. Dietary Guidelines for all Patients with FCS

11.4. Dietary Guidelines by Population

12. Patient Journey

13. Unmet Needs

14. Key Endpoints in FCS Clinical Trials

15. Marketed Product

15.1. WAYLIVRA (volanesorsen): Ionis Pharmaceuticals/Akcea Therapeutic

15.1.1. Product Description

15.1.2. Regulatory Milestones

15.1.3. Other Developmental Activities

15.1.4. Pivotal Clinical Trials

16. Emerging Therapies

16.1. Key Cross

16.2. AKCEA-ANGPTL3-LRx: Akcea Therapeutics/Ionis Pharmaceuticals

16.2.1. Product Description

16.2.2. Other Developmental Activities

16.2.3. Clinical Development

16.3. ARO-APOC3: Arrowhead Pharmaceuticals

16.3.1. Product Description

16.3.2. Other Developmental Activities

16.3.3. Clinical Development

16.3.4. Safety and Efficacy

16.4. Lomitapide: Amryt Pharma/Novelion Therapeutics

16.4.1. Product Description

16.4.2. Other Developmental Activities

16.5. AKCEA -APOCIII – LRx: Ionis Pharmaceuticals/Akcea Therapeutics

16.5.1. Product Description

16.5.2. Other Developmental Activities

16.5.3. Clinical Development

16.5.4. Safety and Efficacy

17. Conjoint Analysis of Familial Chylomicronemia Syndrome (FCS) Therapies

18. Familial Chylomicronemia Syndrome (FCS): Seven Major Market Analysis

18.1. Key Findings

18.2. Market Outlook: 7MM

18.3. Total Market Size of FCS in the 7MM

18.4. Market Size of FCS by Therapies in the 7MM

19. United States Market Size

19.1. Total Market size of FCS in the United States

20. EU-5 Market Size

20.1. Germany Market Size

20.1.1. Total Market size of FCS in Germany

20.2. France Market Size

20.2.1. Total Market size of FCS in France

20.3. Italy Market Size

20.3.1. Total Market size of FCS in Italy

20.4. Spain Market Size

20.4.1. Total Market size of FCS in Spain

20.5. United Kingdom Market Size

20.5.1. Total Market size of FCS in the United Kingdom

21. Japan Market Size

21.1. Total Market size of FCS in Japan

22. Market Access and Reimbursement Scenario of FCS Therapies

23. Market Drivers

24. Market Barriers

25. Appendix

25.1. Bibliography

25.2. Report Methodology

26. Disclaimer

27. DelveInsight Capabilities

28. About DelveInsight

Table 1 Summary of FCS Market, Epidemiology, and Key Events (2017–2030)

Table 2 Known Mutations Responsible for FCS

Table 3 Lipid Panel Parameters for Diagnosing FCS

Table 4 Major Sources Used

Table 5 Total prevalence of FCS in the 7MM (2017–2030)

Table 6 Total Diagnosed Prevalence of FCS in the 7MM (2017–2030)

Table 7 Age-specific Diagnosed Prevalence of FCS in the 7MM (2017–2030)

Table 8 Total Prevalent cases of FCS in the United States (2017–2030)

Table 9 Total Prevalent Cases of FCS in the United States (2017–2030)

Table 10 Severity- Specific Diagnosed Prevalence of FCS in the United States (2017–2030)

Table 11 Treated Patient Pool of FCS in the United States (2017–2030)

Table 12 Total Prevalent cases of FCS in Germany (2017–2030)

Table 13 Total Diagnosed Prevalent Cases of FCS in Germany (2017–2030)

Table 14 Severity- Specific Diagnosed Prevalence of FCS in Germany (2017–2030)

Table 15 Treated Patient Pool of FCS in Germany (2017–2030)

Table 16 Total Prevalent cases of FCS in France (2017–2030)

Table 17 Total Diagnosed Prevalent Cases of FCS in France (2017–2030)

Table 18 Severity- Specific Diagnosed Prevalence of FCS in France (2017–2030)

Table 19 Treated Patient Pool of FCS in France (2017–2030)

Table 20 Total Prevalent cases of FCS in Italy (2017–2030)

Table 21 Total Diagnosed Prevalent Cases of FCS in Italy (2017–2030)

Table 22 Severity-Specific Diagnosed Prevalence of FCS in Italy (2017–2030)

Table 23 Treated Patient Pool of FCS in Italy (2017–2030)

Table 24 Total Prevalent cases of FCS in Spain (2017–2030)

Table 25 Total Diagnosed Prevalent Cases of FCS in Spain (2017–2030)

Table 26 Severity-Specific Diagnosed Prevalence of FCS in Spain (2017–2030)

Table 27 Treated Patient Pool of FCS in Spain (2017–2030)

Table 28 Total Prevalent cases of FCS in the United Kingdom (2017–2030)

Table 29 Total Diagnosed Prevalent Cases of FCS in the United Kingdom (2017–2030)

Table 30 Severity-Specific Prevalence of FCS in the United Kingdom (2017–2030)

Table 31 Treated Patient Pool of FCS in the United Kingdom (2017–2030)

Table 32 Total Prevalent cases of FCS in Japan (2017–2030)

Table 33 Total Diagnosed Prevalent Cases of FCS in Japan (2017–2030)

Table 34 Severity- Specific Diagnosed Prevalence of FCS in Japan (2017–2030)

Table 35 Treated Patient Pool of FCS in Japan (2017–2030)

Table 36 Dietary and Treatment Guidelines for People with FCS throughout the Life Span

Table 37 Patent Information Of WAYLIVRA

Table 38 Summary of Fasting Triglycerides (Mean (SD, SEM), mg/dL) Over Time in Study CS7

Table 39 Key Cross Competition of Emerging Therapies

Table 40 AKCEA-ANGPTL3-LRX, Clinical Trial Description, 2020

Table 41 ARO-APOC3, Clinical Trial Description, 2020

Table 42 AKCEA -APOCIII - LRx, Clinical Trial Description, 2020

Table 43 Update on WAYLIVRA—only approved Drug in European Union (EU)

Table 44 7MM Market Size of FCS, in USD Million (2017–2030)

Table 45 Therapy-wise Market Size of FCS in the 7MM, in USD Million (2017–2030)

Table 46 Market Size of FCS in the United States, in USD Millions (2017–2030)

Table 47 Market Size of FCS in Germany, in USD Millions (2017–2030)

Table 48 Market Size of FCS in France, in USD Millions (2017–2030)

Table 49 Market Size of FCS in Italy, in USD Millions (2017–2030)

Table 50 Market Size of FCS in Spain, in USD Millions (2017–2030)

Table 51 Market Size of FCS in the United Kingdom, in USD Millions (2017–2030)

Table 52 Market Size of FCS in Japan, in USD Millions (2017–2030)



List of Figures:

Figure 1 FCS SWOT Analysis

Figure 2 Signs and Symptoms of FCS

Figure 3 FCS Pathophysiology

Figure 4 Stepwise Approach to Identifying the Etiology of Severe Hypertriglyceridemia

Figure 5 A Practical Diagnostic Scoring System for FCS

Figure 6 Diagnostic Algorithm of FCS

Figure 7 Global Heat Map

Figure 8 Total prevalence of FCS in the 7MM (2017–2030)

Figure 9 Total Diagnosed Prevalence of FCS in the 7MM (2017–2030)

Figure 10 Age-specific Diagnosed Prevalence of FCS in the 7MM (2017–2030)

Figure 11 Total Prevalent cases of FCS in the United States (2017–2030)

Figure 12 Total Diagnosed Prevalent Cases of FCS in the United States (2017–2030)

Figure 13 Severity- Specific Diagnosed Prevalence of FCS in the United States (2017–2030)

Figure 14 Treated Patient Pool of FCS in the United States (2017–2030)

Figure 15 Total Prevalent cases of FCS in Germany (2017–2030)

Figure 16 Total Diagnosed Prevalent Cases of FCS in Germany (2017–2030)

Figure 17 Severity- Specific Diagnosed Prevalence of FCS in Germany (2017–2030)

Figure 18 Treated Patient Pool of FCS in Germany (2017–2030)

Figure 19 Total Prevalent cases of FCS in France (2017–2030)

Figure 20 Total Diagnosed Prevalent Cases of FCS in France (2017–2030)

Figure 21 Severity-Specific Prevalence of FCS in France (2017–2030)

Figure 22 Treated Patient Pool of FCS in France (2017–2030)

Figure 23 Total Prevalent cases of FCS in Italy (2017–2030)

Figure 24 Total Diagnosed Prevalent Cases of FCS in Italy (2017–2030)

Figure 25 Severity- Specific Diagnosed Prevalence of FCS in Italy (2017–2030)

Figure 26 Treated Patient Pool of FCS in Italy (2017–2030)

Figure 27 Total Prevalent cases of FCS in Spain (2017–2030)

Figure 28 Total Diagnosed Prevalent Cases of FCS in Spain (2017–2030)

Figure 29 Severity- Specific Diagnosed Prevalence of FCS in Spain (2017–2030)

Figure 30 Treated Patient Pool of FCS in Spain (2017–2030)

Figure 31 Total Prevalent cases of FCS in the United Kingdom (2017–2030)

Figure 32 Total Diagnosed Prevalent Cases of FCS in the United Kingdom (2017–2030)

Figure 33 Severity- Specific Diagnosed Prevalence of FCS in the United Kingdom (2017–2030)

Figure 34 Treated Patient Pool of FCS in in the United Kingdom (2017–2030)

Figure 35 Total Prevalent cases of FCS in Japan (2017–2030)

Figure 36 Total Diagnosed Prevalent Cases of FCS in Japan (2017–2030)

Figure 37 Severity- Specific Diagnosed Prevalence of FCS in Japan (2017–2030)

Figure 38 Treated Patient Pool of FCS in Japan (2017–2030)

Figure 39 Management Goals for FCS

Figure 40 Schematic Diagram Showing Approach to Multidisciplinary Care for FCS Patients

Figure 41 Unmet Needs of FCS

Figure 42 Market Size of FCS in the 7MM Countries, in USD Million (2017–2030)

Figure 43 Therapy-wise Market Size of FCS in the 7MM, in USD Million (2017–2030)

Figure 44 Market Size of FCS in the United States, in USD Millions (2017–2030)

Figure 45 Market Size of FCS in Germany, in USD Millions (2017–2030)

Figure 46 Market Size of FCS in France, in USD Millions (2017–2030)

Figure 47 Market Size of FCS in Italy, in USD Millions (2017–2030)

Figure 48 Market Size of FCS in Spain, in USD Millions (2017–2030)

Figure 49 Market Size of FCS in the United Kingdom, in USD Millions (2017–2030)

Figure 50 Market Size of FCS in Japan, in USD Millions (2017–2030)

Figure 51 Market Drivers of FCS

Figure 52 Market Barriers of FCS

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