Familial Chylomicronemia Syndrome Treatment Market 2019 Trend by Estimated Size, Valuable Share, Business Insights, Demand and Business Opportunities, Global Technological Developments By 2026
Familial Chylomicronemia Syndrome is a genetic condition characterized by inability of the body to digest fats mainly triglyceride. In familial chylomicronemia syndrome, the lipoprotein lipase (LPS) is not functional, which is the enzyme that breaks down chylomicrons in the blood. Healthcare providers can clinically diagnose FCS by looking for acute pancreatitis, abdominal pain, uncontrolled diabetes, excess alcohol intake, and hypertriglyceridemia.
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The most distinctive sign of familial chylomicronemia syndrome is the appearance of fatty blood on the skin, owing to high level of fat in the body. People with extremely high triglycerides levels are more prone to FCS. For instance, according to the Familial Chylomicronemia Syndrome (FCS) Foundation report, in 2017, around 30 million people suffered from rare diseases, and accounted for every 1 out of 10 people in the U.S, and 95% of rare diseases have no Food and Drug Administration (FDA) approved treatment. Familial Chylomicronemia Syndrome is a rare disorder that affect small population worldwide. For instance, according to a report by National Organiztion for Rare Disorders (NORD), in 2017, FCS is more prevalent in French community and certain communities of the Netherland, where the prevalence is as high as every 19 to 20 per one million individual.
Severe complications associated with FCS include fatty liver diseases, enlarged liver, enlarged spleen, and pancreatitis, which can be life-threatening. In pancreatitis, pancreas produces hormones insulin, which helps to digest food and regulates blood sugar level. It is characterized by inflammation of pancreas, which is extremely painful and can be severe. Frequent attacks of pancreatitis may result in long-term organ damage or death. Due to the rarity of the condition, the clinical progression of this syndrome is not well understood, which is expected to create growth opportunities for the market players over the forecast period.
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In people with FCS, the triglyceride level can increase up to 20 to 30 times as compared to normal individuals, even with medication of a low-fat diet. Researchers are working to develop a therapy with a potential to reduce the level of ApoC-III in the blood. ApoC-III has proven to be a protein that regulate triglycerides level in the human body. Thus, reducing the amount of ApoC-III may help to regulate the elevated levels of triglyceride, which is a significant risk factor for pancreatitis.
Non-renewal of Glybera and launch of Volanesorsen to govern the total Familial Chylomicronemia Syndrome treatment market growth
In September 2017, Akcea Therapeutics Inc., a subsidiary of Ionis Pharmaceutical Inc., announced the filing of New Drug Submission (NDS) to Health Canada for Volanesorsen with the focus to develop and commercialize drugs to treat severe cardio metabolic disease caused by lipid disorder. Volanesorsen is designed to decrease the production of ApoC-III protein production in liver, which plays a vital role in the regulation of plasma TG. The Health Canada granted priority review to the drug for the treatment of Familial Chylomicronemia Syndrome in Canada, which could help accelerate the access of this medicine to the people of Canada suffering from FCS and is expected to fuel the growth of the market in this region. Akcea Therapeutics achieved successful results in its phase 3 trails – APPROACH Study and is currently conducting the APPROACH Open Label Study to further evaluate is efficacy with extended administration of the drug in FCS patients.
Glybera, a gene therapy is designed to restore the LPL enzyme required to clear the fat-carrying chylomicron particles from the intestine. It is a one-time gene therapy treatment, which includes a series of 60 intramuscular injections in the legs. In October 2012, the European commission granted marketing authorization of Glybera under exceptional circumstances as a treatment for small subsets of adult patients diagnosed with familial LPL deficiency, which was confirmed by genetic testing. In April 2017, uniQure announced non-renewal of Glybera gene therapy authorization in Europe, which is scheduled to expire in October 2017. The non-renewal of Glybera’s marketing authorization in Europe is expected to affect the overall market of FCS.
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Table of Content
Global Familial Chylomicronemia Syndrome Treatment Market Research Report
Section 1: Global Familial Chylomicronemia Syndrome Treatment Industry Overview
Section 2: Global Economic Impact on Familial Chylomicronemia Syndrome Treatment Industry
Section 3: Global Market Competition by Industry Producers
Section 4: Global Productions, Revenue (Value), according to Regions
Section 5: Global Supplies (Production), Consumption, Export, Import, geographically
Section 6: Global Productions, Revenue (Value), Price Trend, Product Type
Section 7: Global Market Analysis, on the basis of Application
Section 8: Familial Chylomicronemia Syndrome Treatment Market Pricing Analysis
Section 9: Market Chain, Sourcing Strategy and Downstream Buyers
Section 10: Strategies and key policies by Distributors/Suppliers/Traders
Section 11: Key Marketing Strategy Analysis, by Market Vendors
Section 12: Market Effect Factors Analysis
Section 13: Global Familial Chylomicronemia Syndrome Treatment Market Forecast
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