Familial Chylomicronemia Syndrome Treatment Market Moving Toward 2026 With New Procedures

Familial Chylomicronemia Syndrome Treatment
Market Moving Toward 2026 With New Procedures
Familial Chylomicronemia Syndrome Treatment Market By Drug (Glybera and
Volanesorsen) - Global Industry Insights, Trends, Outlook, and Opportunity
Analysis, 2018-2026
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. Healthcare providers
can clinically diagnosed FCS by looking for acute pancreatitis, abdominal pain, uncontrolled diabetes,
excess alcohol intake and hypertriglyceridemia. Click To Read More On Familial Chylomicronemia
Syndrome Treatment Market
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 extremely high triglycerides levels
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 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 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.
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.
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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.
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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. Healthcare providers can clinically diagnosed FCS by looking for acute pancreatitis, abdominal pain, uncontrolled diabetes, excess alcohol intake and hypertriglyceridemia. Click To Read More On Familial Chylomicronemia Syndrome Treatment Market

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