Market Research Report
Familial Chylomicronemia Syndrome (FCS) - Market Insights, Epidemiology and Market Forecast - 2030
|Familial Chylomicronemia Syndrome (FCS) - Market Insights, Epidemiology and Market Forecast - 2030|
DelveInsight Business Research LLP
Content info: 200 Pages
Delivery time: 2-10 business days
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.
Study Period: 2017-2030
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.
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.
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.
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…
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).
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).
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).
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:
Pipeline Development Activities
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
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.
Current Treatment Scenario, Marketed Drugs and Emerging Therapies: