Familial Lipoprotein Lipase Deficiency Market Size, Share, Industry, Forecast and Outlook (2024-2031)

Report Overview

The Global Familial Lipoprotein Lipase Deficiency Market reached US$ XX million in 2023 and is expected to reach US$ XX million by 2031 growing with a CAGR of XX% during the forecast period 2024-2031.

Familial chylomicronemia syndrome (FCS), which is also referred to as lipoprotein lipase deficiency (LPLD), is a rare genetic metabolic disorder caused by a lack of the enzyme lipoprotein lipase.

Lipoprotein lipase is an essential enzyme responsible for the proper breakdown of certain types of fats. When this enzyme is deficient, it leads to the buildup of chylomicrons, fatty particles, in the bloodstream. This accumulation directly contributes to the significantly elevated triglyceride levels observed in individuals with familial lipoprotein lipase deficiency (LPLD). As a result, there is also an increase in the plasma levels of triglycerides, which are fatty substances in the blood.

The high triglyceride concentrations associated with LPLD often result in severe clinical manifestations. Patients may experience recurrent episodes of abdominal pain, inflammation of the pancreas (pancreatitis), enlargement of the liver and/or spleen (hepatosplenomegaly), and the development of eruptive xanthomas, which are skin lesions. These symptoms can significantly impact the quality of life and overall health of those affected by this rare genetic disorder.

Market Scope

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Market Dynamics: Drivers

Growing research & development

The demand for the global familial lipoprotein lipase deficiency market is driven by multiple factors. One of the key factors is the growing research & development. Ongoing investigations into the underlying mechanisms of lipoprotein lipase deficiency (LPLD) and the exploration of potential treatments are driving innovation in therapeutic options. Researchers are particularly focusing on dietary management techniques, such as the incorporation of medium-chain triglyceride (MCT) supplementation, which aims to alleviate symptoms and enhance patient outcomes.

Moreover, with the rising prevalence of LPLD, major players in the industry more focus on the treatment for LLPD and the rising number of clinical trials that would drive this market growth. According to a BMC research publication in February 2023, the prevalence of FCS is estimated to be between 1 in 100,000 and 1 in 1,000,000 individuals. From the same source, recent studies indicate that a concerning 48% of patients with Familial Chylomicronemia Syndrome (FCS) have reported receiving an incorrect diagnosis.

For instance, in May 2023, Arrowhead Pharmaceuticals Inc. announced that it has completed enrollment for its international Phase 3 PALISADE clinical trial assessing the efficacy and safety of ARO-APOC3 in treating familial chylomicronemia syndrome (FCS) in adult patients. The primary endpoint of the study is the percent change from baseline in fasting triglycerides at month ten.

In addition, major player's key developments in the treatment of the LPLD would propel this market growth. Recent progress in gene therapy, particularly through the use of alipogene tiparvovec has revealed considerable promise for treating lipoprotein lipase deficiency (LPLD). This therapeutic approach specifically targets the genetic basis of the disorder, which has the potential to significantly alter the treatment landscape for individuals suffering from this condition.

Restraints

Factors such as high treatment costs,  limited availability of drugs & treatment options, limited awareness & diagnosis among people, and regulatory challenges are expected to hamper the market.

Market Segment Analysis

The global familial lipoprotein lipase deficiency market is segmented based on treatment type, age group, gender, end-user, and region.

The olezarsen segment accounted for approximately 43.4% of the global familial lipoprotein lipase deficiency market share.

The olezarsen segment is expected to hold the largest market share over the forecast period. The FDA has granted fast-track designation to olezarsen for the treatment of familial chylomicronemia syndrome.

Olezarsen is an investigational RNA-targeted Ligand Conjugated Antisense (LICA) therapy being studied for individuals at risk of disease due to elevated triglyceride levels, including those with familial chylomicronemia syndrome (FCS) and severe hypertriglyceridemia (sHTG). This medication is designed to reduce the production of apoC-III, a liver-produced protein that plays a crucial role in regulating triglyceride metabolism in the bloodstream.

Moreover, key players in the industry key developments in the treatments for LPLD and research studies & rising number of clinical trials are getting positive results towards the LPLD treatments help to drive this segment growth in this market. For instance, in April 2024, Ionis Pharmaceuticals, Inc. announced comprehensive results from the Phase 3 Balance study evaluating their lead investigational medicine, olezarsen, for the treatment of adults with familial chylomicronemia syndrome (FCS). The study demonstrated that the 80 mg monthly dose of olezarsen successfully met the primary endpoint by significantly lowering triglyceride (TG) levels in patients with genetically confirmed FCS at six months.

Also, in September 2023, Ionis Pharmaceuticals, Inc. reported positive topline results from the Phase 3 Balance study of olezarsen in individuals with familial chylomicronemia syndrome (FCS). The trial successfully achieved its primary efficacy endpoint, demonstrating a statistically significant reduction in triglyceride (TG) levels with the 80 mg monthly dose of olezarsen after six months, with further improvements observed at the 12-month mark compared to the placebo group.

Market Geographical Share

North America accounted for approximately 41.8% of the global familial lipoprotein lipase deficiency market share. 

North America region is expected to hold the largest market share over the forecast period owing to the rising incidence of genetic disorders, advancements in treatment options, and well-advanced healthcare infrastructure driving this market growth in this region.

According to Ionis Pharmaceuticals, Inc. press release in March 2024, familial chylomicronemia syndrome (FCS) is estimated to affect between 1 to 13 individuals per million in the United States. Those diagnosed with FCS face a heightened risk of acute pancreatitis (AP), along with other chronic health problems, including fatigue and severe, recurrent abdominal pain.

Furthermore, individuals living with familial chylomicronemia syndrome (FCS) may experience significant disruptions to their ability to work, further contributing to the overall burden of the disease. The U.S. Food and Drug Administration (FDA) provides Orphan Drug designation to therapies intended for the treatment, prevention, or diagnosis of rare diseases or conditions that impact fewer than 200,000 people in the United States at the time of designation. This designation aims to incentivize the development of therapies for rare diseases like FCS.

In addition, in this region major number of key players' presence, government initiatives, and product launches & approvals that would drive this market growth. For instance, in June 2024, Ionis Pharmaceuticals, Inc. announced that the U.S. Food and Drug Administration (FDA) has accepted the New Drug Application (NDA) for olezarsen, an investigational RNA-targeted therapy aimed at treating adults with familial chylomicronemia syndrome (FCS), for Priority Review.

Also, in February 2024, Ionis Pharmaceuticals, Inc. announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug designation to olezarsen, their investigational medicine, for the treatment of familial chylomicronemia syndrome (FCS). FCS is a rare, genetic disorder characterized by severely elevated triglyceride levels and recurrent episodes of acute pancreatitis.

Market Segmentation

By Treatment Type

• Olezarsen
• Evinacumab
• Orlistat
• Alipogene Tiparvovec

By Age Group

• 1-10 Years
• 13- 60 Years

By Gender

• Male
• Female

By End-User

• Hospitals & Specialty Clinics
• Ambulatory Surgical Centers
• Others

By Region

• North America
  • U.S.
  • Canada
  • Mexico
• Europe
  • Germany
  • U.K.
  • France
  • Spain
  • Italy
  • Rest of Europe
• South America
  • Brazil
  • Argentina
  • The rest of South America
• Asia-Pacific
  • China
  • India
  • Japan
  • South Korea
  • Rest of Asia-Pacific
• Middle East and Africa

Market Competitive Landscape

The major global players in the familial lipoprotein lipase deficiency market include Ionis Pharmaceuticals, Inc., Regeneron Pharmaceuticals, Inc., Aetna Inc., Ultragenyx Pharmaceutical Inc., Arrowhead Pharmaceuticals Inc., MANUS AKTTEVA BIOPHARMA LLP, Silverline Chemicals, Sainor, GSK plc, and PTC Therapeutics among others.

Key Developments

• In December 2023, Ultragenyx Pharmaceutical Inc. announced that the European Commission (EC) has expanded the approval of Evkeeza as an adjunct to diet and other lipid-lowering therapies for treating children aged 5 to 11 years with homozygous familial hypercholesterolemia (HoFH). Evkeeza is the first treatment that inhibits angiopoietin-like 3 (ANGPTL3) specifically indicated for children as young as 5 years old to manage dangerously high levels of low-density lipoprotein cholesterol (LDL-C) associated with HoFH.
• In March 2023, Regeneron Pharmaceuticals, Inc. announced that the U.S. Food and Drug Administration (FDA) has expanded the approval of Evkeeza as an adjunct to other lipid-lowering therapies for treating children aged 5 to 11 with homozygous familial hypercholesterolemia (HoFH). Evkeeza is the first angiopoietin-like 3 (ANGPTL3) inhibitor approved for children as young as 5 years to manage dangerously high levels of low-density lipoprotein cholesterol (LDL-C) associated with HoFH.

Why Purchase the Report?

• To visualize the global familial lipoprotein lipase deficiency market segmentation based on treatment type, age group, gender, end user, and region and understand key commercial assets and players.
• Identify commercial opportunities by analyzing trends and co-development. 
• Excel data sheet with numerous data points of the familial lipoprotein lipase deficiency market with all segments.
• PDF report consists of a comprehensive analysis after exhaustive qualitative interviews and an in-depth study.
• Product mapping is available in Excel consisting of key products of all the major players.

The global familial lipoprotein lipase deficiency market report would provide approximately 70 tables, 63 figures, and 183 pages.

Target Audience 2024

• Manufacturers/ Buyers
• Industry Investors/Investment Bankers
• Research Professionals
• Emerging Companies