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DelveInsight's "Familial Chylomicronemia Syndrome Market Insights, Epidemiology and Market Forecast- 2034" report delivers an in-depth understanding of the Familial Chylomicronemia Syndrome, historical and forecasted epidemiology as well as the Familial Chylomicronemia Syndrome market trends in the United States, EU4 (Germany, Spain, Italy, and France) and the United Kingdom, and Japan.
The Familial Chylomicronemia Syndrome Treatment Market report provides current treatment practices, emerging drugs, market share of individual therapies, and current and forecasted 7MM Familial Chylomicronemia Syndrome market size from 2020 to 2034. The report also covers current Familial Chylomicronemia Syndrome treatment market practices/algorithms and unmet medical needs to curate the best of the opportunities and assesses the underlying potential of the market.
Familial Chylomicronemia Syndrome Disease Treatment Market
Familial Chylomicronemia Syndrome (FCS) is a rare genetic lipid disorder characterized by an increase in the levels of triglycerides (TGs) due to a mutation in the LPL gene. Due to the rarity of the disease, there is a lack of awareness among patients and healthcare professionals, leading to delayed diagnosis and treatment.
Familial Chylomicronemia Syndrome Diagnosis
FCS is an invisible genetic disorder present at birth; the symptoms of the disease typically depend on the type of mutation a person carries. Some patients get diagnosed during infancy due to severe symptoms, but in many people, the symptoms are mild and remain undiagnosed until they are in their teens or 20s and hence pancreatitis attacks become more frequent. The diagnosis is further delayed by the lack of knowledge among physicians and limited diagnosis and management guidelines data. FCS is often associated with misdiagnosis, as stated in many studies that misdiagnosis is common with a disease like hypertriglyceridemia and AP of unknown cause.
These patients suffer substantial effects from the delayed diagnosis, which raises their chance of acute pancreatitis episodes and hospitalization. Arriving at a diagnosis liberates people from the psychological burdens of uncertainty about their condition and allows them to seek the best care possible. Definitive diagnosis also enables patients to seek out groups to share their experiences, which may help reduce isolation, guilt, and stigmatization.
Familial Chylomicronemia Syndrome Treatment
The main cause of FCS is the abnormal functioning of the LPL gene, which fails to produce the LPL enzyme that breaks down the fat content in the body. When a person takes even a small amount of extra fat, they experience severe abdominal pain along with other symptoms, as a result of which they have to be hospitalized in the emergency department. Currently, there is no FDA-approved treatment for FCS; only one drug is approved in Europe. Diet management is the best way to control the disease, which is usually difficult to maintain.
Other triglyceride-reducing off-label used drugs are available but have shown to be less effective. Also, the associated comorbidities due to the elevation of triglyceride level is another important issue that needs to be treated. This lack of an effective treatment to reduce the symptoms and cure the disease poses a basic unmet need that must be tackled. Treatments that cure genetic defects, such as gene replacement therapies, may effectively treat FCS.
As the market is derived using the patient-based model, the Familial Chylomicronemia Syndrome epidemiology chapter in the report provides historical as well as forecasted epidemiology segmented by, Total Diagnosed Prevalent Cases of Familial Chylomicronemia Syndrome, Age-specific Diagnosed Prevalent Cases of Familial Chylomicronemia Syndrome in the 7MM covering the United States, EU4 countries (Germany, France, Italy, and Spain) and the United Kingdom, and Japan from 2020 to 2034.
Familial Chylomicronemia Syndrome Recent Developments
The drug chapter segment of the Familial Chylomicronemia Syndrome drugs market report encloses a detailed analysis of Familial Chylomicronemia Syndrome marketed drugs and late-stage (Phase-III and Phase-II) Familial Chylomicronemia Syndrome pipeline drugs analysis It also helps to understand the Familial Chylomicronemia Syndrome clinical trials details, expressive pharmacological action, agreements and collaborations, approval and patent details, advantages and disadvantages of each included drug, and the latest Familial Chylomicronemia Syndrome news and press releases.
Familial Chylomicronemia Syndrome Marketed Drugs
WAYLIVRA (volanesorsen): Akcea Therapeutics Ireland Limited/ Ionis Pharmaceuticals
Familial Chylomicronemia Syndrome Emerging Drugs
Olezarsen, formerly known as IONIS-APOCIII-LRx and AKCEA-APOCIII-LRx, is a ligand-conjugated (LICA) investigational antisense medicine designed to inhibit the production of Apoc-III in the liver. In January 2023, Ionis Pharmaceuticals, Inc. received FTD from the US FDA for olezarsen to treat FCS. Olezarsen is patent protected in the US and Europe until at least 2034. Olezarsen is far ahead in its clinical development and may face competition with ARO-APOC3. WAYLIVRA is another drug approved in Europe for FCS, which will compete with Olezarsen in capturing the market. The clinical trials ongoing for Olezarsen have not yet produced concrete results to predict its future.
Familial Chylomicronemia Syndrome Drugs Market Insights
The available treatment choices for FCS can be separated into managing acute crises related to pancreatitis and chronic management of hypertriglyceridemia to reduce the risk of future episodes. Current pharmacological treatment options for patients suffering from FCS are fibrates, niacin, omega-3, and statins. However, in severely Interfered cases, these are often ineffective in lowering TG levels.
The replacement of LPL with genetic replacement therapy is a hopeful treatment choice. One example of gene replacement therapy is the GLYBERA (alipogene tiparvovec). GLYBERA was the first gene therapy approved for the management of FCS. GLYBERA was administered by a series of intramuscular injections. However, this therapy was pulled from the market in 2017 due to poor commercial prospects and uncertainties about reimbursement.
Familial Chylomicronemia Syndrome Companies such as Ionis Pharmaceuticals and Arrowhead Pharmaceuticals are working toward developing a better treatment approach for FCS patients to overcome this major unmet need. Expected entry of their investigational products or treatment options would offer considerable hope to sufferers of FCS and their families.
There is a lack of approved therapies for FCS in the market. Currently, no drug is approved by the United States FDA for managing FCS. Likewise, no therapy is approved in Japan as well. However, the EMA granted marketing authorization to WAYLIVRA (Volanesorsen) in the EU in May 2019. The drug has also been granted ODD by the US FDA and EMA but failed to get approval in the US.
As per the ClinicalTrials.gov and additional secondary domain searches, the pipeline of FCS is not very robust. Not many companies have taken the initiative to meet the high medical requirement of this condition. But contrary to this, companies like Akcea Therapeutics, Ionis Pharmaceuticals, and Arrowhead Pharmaceuticals stepped into this field.
In the US, the revenue of triglyceride-lowering medication is anticipated to be stagnant due to the emergence of pipeline candidates. Olezarsen (Ionis Pharmaceuticals, Inc.) and ARO-APOC3 (Arrowhead Pharmaceuticals) are anticipated to be launched during the forecast period (2023-2034).
This section focuses on the rate of uptake of the potential Familial Chylomicronemia Syndrome drugs expected to get launched in the market during the study period 2020-2034. For example, for Olezarsen (ApoC-III), we expect the drug uptake to be medium with a probability-adjusted peak share of around 13%, and years to the peak is expected to be 6 years from the year of launch in the US.
Familial Chylomicronemia Syndrome Pipeline Development Activities
The Familial Chylomicronemia Syndrome therapeutics market report provides insights into Familial Chylomicronemia Syndrome Clinical Trials within Phase III, Phase II, and Phase I stage. It also analyzes key Familial Chylomicronemia Syndrome Companies involved in developing targeted therapeutics.
Pipeline Development Activities
The Familial Chylomicronemia Syndrome therapeutics market report covers detailed information on collaborations, acquisitions and mergers, licensing, and patent details for Familial Chylomicronemia Syndrome emerging therapies.
KOL- Views
To keep up with current market trends, we take KOLs and SMEs' opinions working in the domain through primary research to fill the data gaps and validate our secondary research. Industry Experts contacted for insights on FCS evolving treatment landscape, patient reliance on conventional therapies, patient's therapy switching acceptability, and drug uptake along with challenges related to accessibility, include University of Southern California, California, the US; Klinik und Poliklinik fur Kardiologie, Universitatsklinikum Leipzig, Germany; Centro regionale delle malattie rare del metabolismo dell'adulto (CERMMET) Policlinico, Palermo, Italy; St. Thomas' Hospital, Westminster Bridge Road, London, the UK; Kanazawa University Graduate School of Medical Science, Kanazawa, Japan and others.
Delveinsight's analysts connected with 50+ KOLs to gather insights, however, interviews were conducted with 15+ KOLs in the 7MM. Their opinion helps to understand and validate current and emerging therapies and treatment patterns or Familial Chylomicronemia Syndrome market trends. This will support the clients in potential upcoming novel treatments by identifying the overall scenario of the market and the unmet needs.
Familial Chylomicronemia Syndrome Drugs Market: Qualitative Analysis
We perform Qualitative and market Intelligence analysis using various approaches, such as SWOT analysis, and Conjoint Analysis. In the SWOT analysis, strengths, weaknesses, opportunities, and threats in terms of disease diagnosis, patient awareness, patient burden, competitive landscape, cost-effectiveness, and geographical accessibility of therapies are provided. These pointers are based on the Analyst's discretion and assessment of the patient burden, cost analysis, and existing and evolving treatment landscape.
Conjoint Analysis is done to analyze multiple approved and emerging therapies based on relevant attributes such as safety, efficacy, frequency of administration, route of administration, and order of entry. Scoring is given based on these parameters to analyze the effectiveness of therapy. Further, the therapies' safety is evaluated wherein the acceptability, tolerability, and adverse events are majorly observed, and it sets a clear understanding of the side effects posed by the drug in the trials. In addition, the scoring is also based on the route of administration, order of entry and designation, probability of success, and the addressable patient pool for each therapy. According to these parameters, the final weightage score and the ranking of the emerging therapies are decided.
Familial Chylomicronemia Syndrome Therapeutics Market Access and Reimbursement
Reimbursement is a crucial point for any drug after its approval. Many drugs or therapies are not properly recognized by the reimbursement body and may fail to get reimbursed or their reimbursement process gets delayed. Akcea's WAYLIVRA for treating FCS was rejected by the FDA in 2018 on concerns about its safety, including the risk of serious bleeding and low blood platelet count, but was approved in the EU in 2019. That leaves Europe as the main market for WAYLIVRA, and Italy, Germany, Spain, Portugal, and Austria have also agreed to reimbursement deals for the FCS drug (Pharmaphorum, n.d.).
In 2020, Akcea announced new pricing and reimbursement for WAYLIVRA in Germany as an adjunct to diet in adult patients with genetically confirmed FCS. The product was launched there a year ago, but the initial price was renegotiated per Germany's reimbursement and market access protocol (Akcea Pharma, 2020). The Familial Chylomicronemia Syndrome therapeutics market report further provides detailed insights on the country-wise accessibility and reimbursement scenarios, cost-effectiveness scenario of approved therapies, programs making accessibility easier and out-of-pocket costs more affordable, insights on patients insured under federal or state government prescription drug programs, etc.
Familial Chylomicronemia Syndrome Treatment Market Report Scope
Key Questions Answered
Familial Chylomicronemia Syndrome Treatment Market Insights:
Familial Chylomicronemia Syndrome Epidemiology Insights:
Current Familial Chylomicronemia Syndrome Treatment Market Scenario, Marketed Drugs, and Emerging Therapies: