Familial Chylomicronemia Syndrome is a genetic condition characterized by an 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. The most distinctive sign of Familial Chylomicronemia Syndrome is the appearance of fatty blood on the skin, mainly owing to high level of fat in the body. People with high level of triglycerides are more subjected to FCS. According to the Familial Chylomicronemia Syndrome (FCS) Foundation report, in 2017, around 30 million people suffer from rare diseases, accounted for every 1 out of 10 people in the U.S, and also 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. According to a report by National Organization 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 individuals.
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 lead to 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 creating an opportunity for the Familial Chylomicronemia Syndrome treatment market player in near future.
With FCS, the triglyceride level can increase up to 20 to 30 time 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 level 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 Familial Chylomicronemia Syndrome treatment 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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