Familial Chylomicronemia Syndrome Market to Exhibit Substantial Growth Rate During the Forecast Period (2023-2032) - DelveInsight | Ionis Pharmaceuticals (Olezarsen), Ionis Pharmaceuticals, Pfizer, Arrowhead Pharmaceuticals, Lipigon Pharmaceuticals

As per DelveInsight, the Familial Chylomicronemia Syndrome Market is anticipated to evolve immensely in the coming years owing to the rise in the number of cases of Familial Chylomicronemia Syndrome and the launch of new therapies in the market.

DelveInsight's "Familial Chylomicronemia Syndrome Market Insights, Epidemiology, and Market Forecast 2032" report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the Familial Chylomicronemia Syndrome market size, share, trends, and growth opportunities in the seven major markets (7MM) (i.e., the United States, EU4 (Germany, Spain, Italy, France), the United Kingdom and Japan).

The report covers emerging Familial Chylomicronemia Syndrome drugs, current treatment practices, market share of individual therapies, and current & forecasted market size from 2019 to 2032. It also evaluates the current Familial Chylomicronemia Syndrome treatment practice/algorithm, key drivers & barriers impacting the market growth, and unmet medical needs to curate the best of the opportunities and assess the underlying potential of the market.

Familial Chylomicronemia Syndrome: An Overview
Familial chylomicronemia syndrome (FCS) is a serious rare genetic disorder that prevents the body from breaking down fats consumed through the diet, or triglycerides. These triglycerides are carried in the blood by large structures called chylomicrons, which help move triglycerides to different parts of the body where they are needed for energy and fat storage.

FCS is a rare autosomal recessive metabolic disorder caused by a mutation in lipoprotein lipase. Lipoprotein lipase is a digestive enzyme in the blood that helps the body break down chylomicrons. People who have FCS are either unable to make lipoprotein lipase or have a broken form of it. This leads to elevated triglycerides and chylomicrons in the plasma which causes an array of symptoms including serious episodes of acute pancreatitis. It correlates with significant morbidity and distress among the patients.

Also known as lipoprotein lipase deficiency (LPLD), Fredrickson Type 1 hyperlipoproteinemia, or familial hypertriglyceridemia, it results from homozygosity or compound heterozygosity for null mutations in one or more genes that compromise chylomicron-lipolysis and clearance, most commonly the lipoprotein lipase (LPL) gene or, less frequently, other genes directly affecting LPL activity, namely APOC2, APOA5, LMF1, and GPIHBP1.

Being an inherited disorder though the signs and symptoms can develop as early as infancy or childhood, it is more common in adolescence. Few may not present symptoms until adulthood. The common signs include Lipemia Retinalis, Eruptive xanthomas, Severe pain in the abdomen, often including back pain, besides bloating, asthenia, and indigestion. Acute or recurrent episodes of pancreatitis are one of the severe manifestations that can lead to can lead to long-term pancreatic dysfunction. Foggy-headedness, depression, and difficulty in concentration are also some of the commonly reported symptoms.

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Familial Chylomicronemia Syndrome Market Key Facts
• The total Familial Chylomicronemia Syndrome market size in the 7MM was around USD 15 million in 2022.
• Among the 7MM, Germany captured the highest Familial Chylomicronemia Syndrome market in 2022, with an estimated USD 5 million in 2022, which is anticipated to grow during the forecast period.
• Among the forecasted emerging therapies, Olezarsen (ApoC-III) is expected to capture the highest market in the 7MM by 2032.
• The total Familial Chylomicronemia Syndrome diagnosed with prevalent cases in the 7MM is expected to rise by 2032. However, the rise in many cases is not significant as FCS is an ultra-rare indication and is underdiagnosed.
• The United States showed the highest Familial Chylomicronemia Syndrome diagnosed prevalent population compared to other 7MM countries, with 1.3K cases in 2022. As per DelveInsight's estimates, the country alone accounts for nearly 65% of Familial Chylomicronemia Syndrome diagnosed prevalent cases in the 7MM countries.
• In 2022, in the US, the Familial Chylomicronemia Syndrome age-specific distribution showed 877 and 472 cases in the age groups 0-17 and =18, respectively.
• EU4 and the UK countries accounted for nearly 485 Familial Chylomicronemia Syndrome diagnosed prevalent cases in 2022. Of these cases, 135 cases were estimated in Germany, accounting for the highest number of Familial Chylomicronemia Syndrome diagnosed prevalent cases in Europe.
• Japan accounted for 12% of the total Familial Chylomicronemia Syndrome diagnosed prevalent cases in the 7MM in 2022.

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Familial Chylomicronemia Syndrome Market
The market outlook section of the report helps to build a detailed comprehension of the historical, current, and forecasted market size by analyzing the impact of current and emerging Familial Chylomicronemia Syndrome pipeline therapies. It also thoroughly assesses the Familial Chylomicronemia Syndrome market drivers & barriers, unmet needs, and emerging technologies set to impact the market dynamics.

The report gives complete details of the market trend for each marketed Familial Chylomicronemia Syndrome drug and mid & late-stage pipeline therapies by evaluating their impact based on the annual cost of therapy, their Mechanism of Action (MOA), Route of Administration (ROA), molecule types, competition with other therapies, brand value, and their impact on the market.

Familial Chylomicronemia Syndrome Epidemiology Assessment
The epidemiology section provides insights into the historical, current, and forecasted Familial Chylomicronemia Syndrome epidemiology trends in the seven major countries (7MM) from 2019 to 2032. It helps to recognize the causes of current and forecasted Familial Chylomicronemia Syndrome epidemiology trends by exploring numerous studies and research. The epidemiology section also provides a detailed analysis of diagnosed and prevalent patient pools, future trends, and views of key opinion leaders.

The Report Covers the Familial Chylomicronemia Syndrome Epidemiology, Segmented as -
• Diagnosed prevalent cases of Familial Chylomicronemia Syndrome
• Age-specific diagnosed prevalent cases of Familial Chylomicronemia Syndrome
And Many Others

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Familial Chylomicronemia Syndrome Drugs Uptake and Pipeline Development Activities
The drug uptake section focuses on the uptake rate of potential drugs recently launched in the Familial Chylomicronemia Syndrome market or expected to be launched during the study period. The analysis covers the Familial Chylomicronemia Syndrome market uptake by drugs, patient uptake by therapies, and sales of each drug. Moreover, the therapeutics assessment section helps understand the market dynamics by drug sales, the most rapid drug uptake, and the reasons behind the maximal use of particular drugs. Additionally, it compares the Familial Chylomicronemia Syndrome drugs based on their sale and market share.

The report also covers the Familial Chylomicronemia Syndrome pipeline development activities. It provides valuable insights about different therapeutic candidates in various stages and the key Familial Chylomicronemia Syndrome companies involved in developing targeted therapeutics. It also analyzes recent developments such as collaborations, acquisitions, mergers, licensing patent details, and other information for emerging therapies.

Familial Chylomicronemia Syndrome Therapeutics Analysis
FCS cannot be cured, but it can be managed. The available treatment choices for FCS can be separated into managing acute crises related to pancreatitis and chronic management of hypertriglyceridemia to reduce the risk of future episodes. The current treatment for Familial Chylomicronemia Syndrome involves the restriction of the dietary fat intake allied with traditional TG-lowering agents like fibrates, statins, and niacin's. However, these medications are not very effective in treating FCS.

To improve the treatment scenario, several prominent companies are actively engaged in developing therapies for Familial Chylomicronemia Syndrome. Among the key players, Ionis Pharmaceuticals stands out for advancing its Familial Chylomicronemia Syndrome drug candidates to the most advanced stage of clinical trials, specifically Phase III. This significant progress underscores the commitment of Ionis Pharmaceuticals and other stakeholders to address the challenges posed by Familial Chylomicronemia Syndrome, with the aim of providing effective treatments and improving the quality of life for individuals affected by this condition.

On February 15, 2024, Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug designation to the investigational medicine Olezarsen for the treatment of familial chylomicronemia syndrome (FCS).

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Familial Chylomicronemia Syndrome Companies Actively Working in the Therapeutics Market Include
• Ionis Pharmaceuticals
• Pfizer
• Arrowhead Pharmaceuticals
• Lipigon Pharmaceuticals
And Many Others

Emerging and Marketed Familial Chylomicronemia Syndrome Therapies Covered in the Report Include:
• Olezarsen: Ionis Pharmaceuticals
• WAYLIVRA (volanesorsen): Akcea Therapeutics Ireland Limited
• ARO-APOC3: Arrowhead Pharmaceuticals, Inc
And Many More

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Table of Content (TOC)
1. Key Insights
2. Executive Summary
3. Familial Chylomicronemia Syndrome Competitive Intelligence Analysis
4. Familial Chylomicronemia Syndrome Market Overview at a Glance
5. Familial Chylomicronemia Syndrome Disease Background and Overview
6. Familial Chylomicronemia Syndrome Patient Journey
7. Familial Chylomicronemia Syndrome Patient Population and Epidemiology Trends (In the US, EU5, and Japan)
8. Familial Chylomicronemia Syndrome Treatment Algorithm, Current Treatment, and Medical Practices
9. Familial Chylomicronemia Syndrome Unmet Needs
10. Key Endpoints of Familial Chylomicronemia Syndrome Treatment
11. Familial Chylomicronemia Syndrome Marketed Therapies
12. Familial Chylomicronemia Syndrome Emerging Drugs and Latest Therapeutic Advances
13. Familial Chylomicronemia Syndrome Seven Major Market Analysis
14. Attribute Analysis
15. Familial Chylomicronemia Syndrome Market Outlook (In US, EU5, and Japan)
16. Familial Chylomicronemia Syndrome Companies Active in the Market
17. Familial Chylomicronemia Syndrome Access and Reimbursement Overview
18. KOL Views on the Familial Chylomicronemia Syndrome Market
19. Familial Chylomicronemia Syndrome Market Drivers
20. Familial Chylomicronemia Syndrome Market Barriers
21. Appendix
22. DelveInsight Capabilities
23. Disclaimer
*The Table of Contents (TOC) is not exhaustive; the final content may vary. Refer to the sample report for the complete table of contents.

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