Familial Chylomicronemia Syndrome Treatment Market By Drug (Glybera and Volanesorsen) - Global Industry Insights, Trends, Outlook, and Opportunity Analysis, 2018-2026

  • To Be Published : Jun 2019 |
  • Format : CMI Insight PPT Format CMI Insight PDF Format

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. 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.

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.  

Research Methodology

Coherent Market Insights followsa comprehensive research methodology focused on providing the most precise market analysis. The company leverages a data triangulation model which helps company to gauge the market dynamics and provide accurate estimates. Key components of the research methodologies followed for all our market reports include:

  • Primary Research (Trade Surveys and Experts Interviews)
  • Desk Research
  • Proprietor Data Analytics Model

In addition to this, Coherent Market Insights has access to a wide range of the regional and global reputed paid data bases, which helps the company to figure out the regional and global market trends and dynamics. The company analyses the industry from the 360 Degree Perspective i.e. from the Supply Side and Demand Side which enables us to provide granular details of the entire ecosystem for each study. Finally, a Top-Down approach and Bottom-Up approach is followed to arrive at ultimate research findings.

Smiley face

Coherent Market Insights desk research is based on a principle set of research techniques:

  • National level desk research: It Includes research analysis of regional players, regional regulatory bodies, regional trade associations, and regional organization.
  • Multinational level desk research: The research team keeps a track of multinational players, global regulatory bodies, global trade associations, and global organization.

Coherent Market Insights has a large amount of in-house repository of industry database. This is leveraged as a burner for initiating a new research study. Key secondary sources include:

  • Governmental bodies, National and international social welfare institutions, and organizations creating economic policies among others.
  • Trade association, National and international media and trade press.
  • Company Annual reports, SEC filings, Corporate Presentations, press release, news, and specification sheet of manufacturers, system integrators, brick and mortar - distributors and retailers, and third party online commerce players.
  • Scientific journals, and other technical magazines and whitepapers.
Smiley face

Preliminary Data Mining

The raw data is obtained through the secondary findings, in house repositories, and trade surveys. It is then filtered to ensure that the relevant information including industry dynamics, trends, and outlook is retained for further research process.

Data Standardization:

Holistic approach is used to ensure that the granular and uncommon parameters are taken into consideration to ensure accurate results. The information from the paid databases are further combined to the raw data in order to standardize it.

Coherent Statistical model

We arrive at our final research findings through simulation models. Coherent Data Analytics Model is a statistical tool that helps company to forecast market estimates. Few of the parameters considered as a part of the statistical model include:

  • Micro-economic indicators
  • Macro-economic indicators
  • Environmental indicators
  • Socio-political indicators
  • Technology indicators

Data Processing

Once the findings are derived from the statistical model, large volume of data is process to confirm accurate research results. Data analytics and processing tools are adopted to process large chunk of collected informative data. In case, a client customizes the study during the process, the research finding till then are benchmarked, and the process for new research requirement is initiated again.

Data Validation

This is the most crucial stage of the research process. Primary Interviews are conducted to validate the data and analysis. This helps in achieving the following purposes:

  • It provides first-hand information on the market dynamics, outlook, and growth parameters.
  • Industry experts validates the estimates which helps the company to cement the on-going research study.
  • Primary research includes online surveys, face-to face interviews, and telephonic interviews.

The primary research is conducted with the ecosystem players including, but not limited to:

  • Raw Material Suppliers
  • Manufacturers
  • System Integrators
  • Distributors
  • End-users

Choose License Type

"How Can I Help You" ?

Finding it difficult to find the research that would cater to your business demands? Give us a chance to help you. One of our Research Consultants will connect to provide a customized solution.

[email protected]

Key Benefits

  • Custom Research Solution

    Talk to our research consultant to design an exclusive report as per your research needs.

  • Customer Fulfilment

    We aim to fulfil client's research demands with tailored research solutions.

  • Quick Turnaround Time Reports

    We aim to provide research studies in quickest turnaround time and in a much cost effective manner.

  • 360-degree Approach

    We cover each industry from supply and demand side with an aim to provide a most holistic research study.

  • Assured Quality

    We strive to provide most accurate and reliable research findings in our research reports.