DelveInsight added a report on “Familial Chylomicronemia Syndrome Market“. It covers an overview, its causes, signs and symptoms, and therapies. Major players are involved in developing therapies for Familial Chylomicronemia Syndrome. The launch of emerging therapies will significantly impact the Familial Chylomicronemia Syndrome market.
Familial Chylomicronemia Syndrome Overview
Familial Chylomicronemia Syndrome (FCS) is a rare genetic lipid disorder, which is characterized by the increase in the triglycerides (TGs) level due to reduction in the LPL, which is an enzyme that helps to break down chylomicrons (CMs) in the body. The reduction in the LPL leads to the buildup of CMs which are rich in triglycerides.
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Some of the key highlights of the Familial Chylomicronemia Syndrome Market Report
- According to DelveInsight’s analysis, majority of FCS patient population is significantly interfered.
- Familial Chylomicronemia Syndrome is underdiagnosed or not diagnosed at all, as there are so many other causes for high triglycerides level. The total 7MM diagnosed prevalent cases of Familial Chylomicronemia Syndrome was estimated to be highest in the United States, with around 22% of the total diagnosed FCS cases for the year 2017.
- Familial Chylomicronemia Syndrome is mainly diagnosed in the age group of 0-10 years, followed by age group of 11-20 years of age. It is estimated that approximately 73% of FCS cases belong to age-group 0–10 years in 2017 for the 7MM.
- Familial Chylomicronemia Syndrome companies included such as Ionis Pharmaceuticals/Akcea Therapeutic, Akcea Therapeutics/Ionis Pharmaceuticals, Arrowhead Pharmaceuticals, Amryt Pharma/Novelion Therapeutics, and several others.
- Familial Chylomicronemia Syndrome therapies included such as WAYLIVRA (volanesorsen), AKCEA-ANGPTL3-LRx, ARO-APOC3, Lomitapide, AKCEA -APOCIII – LRx, and several others.
Familial Chylomicronemia Syndrome Symptoms
- High levels of triglycerides in the blood
- Veins in the eyes appear
- Memory loss
- Severe pain in the abdomen
- Pancreatitis
Familial Chylomicronemia Syndrome Diagnosis
The diagnosis is mainly supported by the presence of markedly elevated TG concentrations and CM, the latter of which is normally rapidly cleared from the plasma following a meal. Nearly 70% of patients reported that FCS interfered significantly with their self-worth, emotional well-being, sleep, and mental functioning. The dietary restriction of fat intake and traditional TG-lowering agents is not so efficacious in these patients.
Familial Chylomicronemia Syndrome Treatment
The current treatment involves the restriction in the dietary fat intake allied with traditional TG-lowering agents like fibrates, statins, and niacin’s. However, there is no US Food and Drug Administration (FDA) approved drug for the treatment of FCS, but in May 2019, Waylivra (volanesorsen) was conditionally approved by European Commission (EC) for the treatment of FCS.
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Familial Chylomicronemia Syndrome Epidemiology Segmentation
- Total Prevalence
- Age-specific Diagnosed Prevalence
- Treated Patient Pool
- Total Diagnosed Prevalence
Familial Chylomicronemia Syndrome Market Insights
The present mainstay therapy for FCS is a very-low-fat diet (less than 20 g per day) allied with traditional trigelyceride-lowering medication. However, it is noteworthy to mention that such types of diets are very difficult for patients to follow. So, for this type of standard of care to be effective, long‐term patient education, and support aligned mainly to maintain a low‐fat diet is required. In everyday life, adherence to such a regimen is very challenging and is almost never consistently achieved by most patients.
Familial Chylomicronemia Syndrome Market Size
The market size of FCS in the 7MM countries is expected to increase by 2030 from USD 1.0 million in 2017. As per DelveInsight’s estimation, among the 7MM countries, the United States had the highest market size of FCS in 2017, which accounts for approximately 30% of the total market, contributing nearly to USD 0.3 million in 2017.
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Familial Chylomicronemia Syndrome Market Drivers
- Rise of the Oligonucleotides in the treatment
- Non-adherence to Extremely Restrictive Low-fat diet
- Increase in the Prevalence Rate
- Improvement in the Diagnosis
Familial Chylomicronemia Syndrome Market Barriers
- Challenges in the Clinical Trials for Therapies
- Delayed Diagnosis
- Barriers in the Access or Reimbursement of Therapies
- Burden of Disease
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Table of content
1. Key Insights
2. Executive Summary of Familial Chylomicronemia Syndrome (FCS)
3. Competitive Intelligence Analysis for Familial Chylomicronemia Syndrome (FCS)
4. Familial Chylomicronemia Syndrome (FCS): Market Overview at a Glance
5. Familial Chylomicronemia Syndrome (FCS): Disease Background and Overview
6. Patient Journey
7. Familial Chylomicronemia Syndrome (FCS) Epidemiology and Patient Population
8. Treatment Algorithm, Current Treatment, and Medical Practices
9. Familial Chylomicronemia Syndrome (FCS) Unmet Needs
10. Key Endpoints of Familial Chylomicronemia Syndrome (FCS) Treatment
11. Familial Chylomicronemia Syndrome (FCS) Marketed Products
12. Familial Chylomicronemia Syndrome (FCS) Emerging Therapies
13. Familial Chylomicronemia Syndrome (FCS): Seven Major Market Analysis
14. Attribute analysis
15. 7MM: Market Outlook
16. Access and Reimbursement Overview of Familial Chylomicronemia Syndrome (FCS)
17. KOL Views
18. Market Drivers
19. Market Barriers
20. Appendix
21. DelveInsight Capabilities
22. Disclaimer
23. About DelveInsight
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