Familial Chylomicronemia Syndrome Market
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.
• The United States
• EU5 (Germany, France, Italy, Spain and the United Kingdom)
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.
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.
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
- 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.
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.
- 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
- 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?
- 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