家族性カイロミクロン血症症候群（FCS）- 市場考察、疫学、市場予測（2034年）

主なハイライト

• 主要7市場の家族性カイロミクロン血症症候群（FCS）の総市場規模は、2022年に約1,500万米ドルであり、予測期間（2023年～2034年）に増加が見込まれます。
• 2022年、家族性カイロミクロン血症症候群（FCS）の市場規模は主要7市場の中でドイツがもっとも大きく、約500万米ドルを占め、2034年までにさらに増加すると予測されます。
• 家族性カイロミクロン血症症候群（FCS）患者の大半は、診断されないままか、誤診されたまま重症化します。
• 2022年にWaylivra（volanesorsen）がより大きな市場をカバーしますが、この薬剤は欧州でのみ使用可能です。米国の規制当局は、超希少な脂質異常症である家族性カイロミクロン血症症候群（FCS）に対するAkcea/IonisのWAYLIVRAの上市申請を却下しました。
• 家族性カイロミクロン血症症候群（FCS）治療市場は、2022年に主要7市場全体で約1,350人の患者がいるにもかかわらず、米国と日本では承認された治療法がありません。
• 米国では2024年にOlezarsen（ApoC-III）が上市される予定であり、家族性カイロミクロン血症症候群（FCS）市場に成長可能性をもたらすと予測されます。
• 新治療法は、家族性カイロミクロン血症症候群（FCS）の市場規模に大きなプラスの変化をもたらす可能性を秘めています。

家族性カイロミクロン血症症候群（FCS）市場の見通し

FCSの承認された治療薬はありません。現在、FCSの治療薬として米国FDAから承認されているものはありません。同様に、日本でも承認された治療法はありません。しかし、EMAは2019年5月にEUでWAYLIVRA（Volanesorsen）に上市許可を与えました。同薬は米国FDAとEMAからもODDを付与されていますが、米国での承認は得られませんでした。

ClinicalTrials.govやその他の二次ドメインの検索によると、FCSのパイプラインはあまり強固ではありません。この病態の高い医療要求を満たすために取り組みを行った企業は多くありません。しかし、これに反して、Akcea Therapeutics、Ionis Pharmaceuticals、Arrowhead Pharmaceuticalsなどの企業がこの分野に参入しました。

米国では、トリグリセリド低下薬の収益は、パイプライン候補の登場により停滞すると予測されます。Olezarsen（Ionis Pharmaceuticals, Inc.）とARO-APOC3（Arrowhead Pharmaceuticals）が予測期間（2023年～2034年）に上市される見込みです。

• 主要7市場の家族性カイロミクロン血症症候群（FCS）の市場規模は、2022年に約1,500万米ドルであり、予測期間（2023年～2034年）に増加が見込まれます。
• 主要7市場の家族性カイロミクロン血症症候群（FCS）の市場規模は、疾患に対する認識の高まり、診断の向上、FCSに対する新たな標的治療の上市により、一定のCAGRで増加すると予測されます。
• 欧州4ヶ国・英国のうち、2022年の家族性カイロミクロン血症症候群（FCS）の市場規模はドイツが36%の寄与度で最大となり、スペインは最下位となっています。
• 日本の治療市場は、家族性カイロミクロン血症症候群（FCS）の標的治療薬が承認されていませんが、2026年までにIonis PharmaceuticalsのOlezarsen（ApoC-III）が上市される見込みであり、市場はプラス成長が予測されます。

当レポートでは、家族性カイロミクロン血症症候群（FCS）の主要7市場（米国、ドイツ、スペイン、イタリア、フランス、英国、日本）について調査分析し、各地域の市場規模、現在の治療法、アンメットニーズ、新薬などの情報を提供しています。

目次

第1章 重要考察

第2章 レポートのイントロダクション

第3章 家族性カイロミクロン血症症候群（FCS）市場の概要

• 家族性カイロミクロン血症症候群（FCS）の市場シェアの分布：治療法別（2020年）
• 家族性カイロミクロン血症症候群（FCS）の市場シェアの分布：治療法別（2034年）

第4章 疫学と市場調査手法

第5章 家族性カイロミクロン血症症候群（FCS）のエグゼクティブサマリー

第6章 重要な出来事

第7章 疾患の背景と概要、家族性カイロミクロン血症症候群（FCS）

• イントロダクション
• FCSの病態生理学と組織病理学
• FCSの症状
• 予後と合併症
• 診断
  • FCSの診断基準
  • 診断アルゴリズム
• 治療と管理

第8章 家族性カイロミクロン血症症候群（FCS）の疫学と患者人口

• 主な調査結果
• 前提条件と根拠：主要7市場
• 主要7市場の家族性カイロミクロン血症症候群（FCS）の診断された患者の総数
• 米国
• 欧州4ヶ国・英国
  • ドイツ
  • フランス
  • イタリア
  • スペイン
  • 英国
• 日本

第9章 ペイシェントジャーニー

第10章 上市済み薬品

• WAYLIVRA（volanesorsen）：Akcea Therapeutics Ireland Limited

第11章 新薬

第11章 主な競合

• Olezarsen：Ionis Pharmaceuticals
• ARO-APOC3：Arrowhead Pharmaceuticals, Inc.

第12章 家族性カイロミクロン血症症候群（FCS）：主要7市場の分析

• 主な調査結果
• 市場見通し
• 主な市場予測の前提条件
• コンジョイント分析
• 主要7市場の家族性カイロミクロン血症症候群（FCS）の総市場規模
• 主要7市場の家族性カイロミクロン血症症候群（FCS）の市場規模：治療法別
• 米国の市場規模
  • 米国のFCSの総市場規模
  • 米国のFCSの市場規模：治療法別
• 欧州4ヶ国・英国の市場規模
  • ドイツ
  • フランス
  • イタリア
  • スペイン
  • 英国
• 日本の市場規模
  • 日本のFCSの総市場規模
  • 日本のFCSの市場規模：治療法別

第13章 SWOT分析

第14章 アンメットニーズ

第15章 KOLの見解

第16章 市場参入と償還

• 米国
• 欧州4ヶ国・英国
  • ドイツ
  • フランス
  • イタリア
  • スペイン
  • 英国
• 日本

第17章 付録

第18章 DelveInsightのサービス内容

第19章 免責事項

第20章 DelveInsightについて

List of Tables

• Table 1 Summary of FCS Market and Epidemiology (2020-2034)
• Table 2 Key Events
• Table 3 Lipid Panel Parameters for Diagnosing FCS
• Table 4 Drugs Indicated to Lower Triglycerides
• Table 5 Total Diagnosed Prevalent cases of FCS in the 7MM (2020-2034)
• Table 6 Total Diagnosed Prevalent Cases of FCS in the US (2020-2034)
• Table 7 Age-specific Diagnosed Prevalent Cases of FCS in the US (2020-2034)
• Table 8 Total Diagnosed Prevalent Cases of FCS in Germany (2020-2034)
• Table 9 Total Diagnosed Prevalent Cases of FCS in France (2020-2034)
• Table 10 Total Diagnosed Prevalent Cases of FCS in Italy (2020-2034)
• Table 11 Total Diagnosed Prevalent Cases of FCS in Spain (2020-2034)
• Table 12 Total Diagnosed Prevalent Cases of FCS in the UK (2020-2034)
• Table 13 Total Diagnosed Prevalent Cases of FCS in EU4 and the UK (2020-2034)
• Table 14 Age-specific Diagnosed Prevalent Cases of FCS in Germany, (2020-2034)
• Table 15 Age-specific Diagnosed Prevalent Cases of FCS in France, (2020-2034)
• Table 16 Age-specific Diagnosed Prevalent Cases of FCS in Italy, (2020-2034)
• Table 17 Age-specific Diagnosed Prevalent Cases of FCS in Spain, (2020-2034)
• Table 18 Age-specific Diagnosed Prevalent Cases of FCS in the UK, (2020-2034)
• Table 19 Age-specific Diagnosed Prevalent Cases of FCS in EU4 and the UK, (2020-2034)
• Table 20 Total Diagnosed Prevalent Cases of FCS in Japan, (2020-2034)
• Table 21 Age-specific Diagnosed Prevalent Cases of FCS in Japan, (2020-2034)
• Table 22 WAYLIVRA (Volanesorsen), Clinical Trial Description, 2024
• Table 23 Additional Results for Changes in Triglycerides in the APPROACH Study (Primary Endpoint at Month 3)
• Table 24 Keycross of Emerging Drugs Under Development
• Table 25 Olezarsen, Clinical trial description, 2024
• Table 26 ARO-APOC3, Clinical trial description, 2024
• Table 27 Key Market Forecast Assumptions for Olezarsen
• Table 28 Key Market Forecast Assumptions for ARO-APOC3
• Table 29 Total Market Size of FCS in the 7MM, in USD million (2020-2034)
• Table 30 Market Size of FCS by Therapies in the 7MM in USD million (2020-2034)
• Table 31 Total Market Size of FCS in the US, in USD million (2020-2034)
• Table 32 Market Size of FCS by Therapies in the US, in USD million (2020-2034)
• Table 33 Total Market Size of FCS in EU4 and the UK, in USD million (2020-2034)
• Table 34 Total Market Size of FCS in Germany, in USD million (2020-2034)
• Table 35 Total Market Size of FCS in France, in USD million (2020-2034)
• Table 36 Total Market Size of FCS in Italy, in USD million (2020-2034)
• Table 37 Total Market Size of FCS in Spain, in USD million (2020-2034)
• Table 38 Total Market Size of FCS in the UK, in USD million (2020-2034)
• Table 39 Market Size of FCS by Therapies in Germany, in USD million (2020-2034)
• Table 40 Market Size of FCS by Therapies in France, in USD million (2020-2034)
• Table 41 Market Size of FCS by Therapies in Italy, in USD million (2020-2034)
• Table 42 Market Size of FCS by Therapies in Spain, in USD million (2020-2034)
• Table 43 Market Size of FCS by Therapies in the UK, in USD million (2020-2034)
• Table 44 Market Size of FCS by Therapies in EU4 and the UK, in USD million (2020-2034)
• Table 45 Market Size of FCS in Japan, in USD million (2020-2034)
• Table 46 Market Size of FCS by Therapies in Japan, in USD million (2020-2034)

List of Figures

• Figure 1 Total Diagnosed Prevalent cases of FCS in the 7MM (2020-2034)
• Figure 2 Total Diagnosed Prevalent Cases of FCS in the US (2020-2034)
• Figure 3 Age-specific Diagnosed Prevalent Cases of FCS in the US (2020-2034)
• Figure 4 Total Diagnosed Prevalent Cases of FCS in Germany (2020-2034)
• Figure 5 Total Diagnosed Prevalent Cases of FCS in France (2020-2034)
• Figure 6 Total Diagnosed Prevalent Cases of FCS in Italy (2020-2034)
• Figure 7 Total Diagnosed Prevalent Cases of FCS in Spain (2020-2034)
• Figure 8 Total Diagnosed Prevalent Cases of FCS in the UK (2020-2034)
• Figure 9 Total Diagnosed Prevalent Cases of FCS in EU4 and the UK (2020-2034)
• Figure 10 Age-specific Diagnosed Prevalent Cases of FCS in Germany, (2020-2034)
• Figure 11 Age-specific Diagnosed Prevalent Cases of FCS in France, (2020-2034)
• Figure 12 Age-specific Diagnosed Prevalent Cases of FCS in Italy, (2020-2034)
• Figure 13 Age-specific Diagnosed Prevalent Cases of FCS in Spain, (2020-2034)
• Figure 14 Age-specific Diagnosed Prevalent Cases of FCS in the UK, (2020-2034)
• Figure 15 Age-specific Diagnosed Prevalent Cases of FCS in EU4 and the UK, (2020-2034)
• Figure 16 Total Diagnosed Prevalent Cases of FCS in Japan, (2020-2034)
• Figure 17 Age-specific Diagnosed Prevalent Cases of FCS in Japan, (2020-2034)
• Figure 18 Patient Journey
• Figure 19 Total Market Size of FCS in the 7MM, in USD million (2020-2034)
• Figure 20 Market Size of FCS by Therapies in the 7MM in USD million (2020-2034)
• Figure 21 Total Market Size of FCS in the US, in USD million (2020-2034)
• Figure 22 Market Size of FCS by Therapies in the US, in USD million (2020-2034)
• Figure 23 Total Market Size of FCS in EU4 and the UK, in USD million (2020-2034)
• Figure 24 Total Market Size of FCS in Germany, in USD million (2020-2034)
• Figure 25 Total Market Size of FCS in France, in USD million (2020-2034)
• Figure 26 Total Market Size of FCS in Italy, in USD million (2020-2034)
• Figure 27 Total Market Size of FCS in Spain, in USD million (2020-2034)
• Figure 28 Total Market Size of FCS in the UK, in USD million (2020-2034)
• Figure 29 Market Size of FCS by Therapies in Germany, in USD million (2020-2034)
• Figure 30 Market Size of FCS by Therapies in France, in USD million (2020-2034)
• Figure 31 Market Size of FCS by Therapies in Italy, in USD million (2020-2034)
• Figure 32 Market Size of FCS by Therapies in Spain, in USD million (2020-2034)
• Figure 33 Market Size of FCS by Therapies in the UK, in USD million (2020-2034)
• Figure 34 Market Size of FCS by Therapies in EU4 and the UK, in USD million (2020-2034)
• Figure 35 Market Size of FCS in Japan, in USD million (2020-2034)
• Figure 36 Market Size of FCS by Therapies in Japan, in USD million (2020-2034)
• Figure 37 SWOT Analysis
• Figure 38 Unmet Needs

Key Highlights:

• The total Familial Chylomicronemia Syndrome Market Size in the 7MM was approximately USD 15 million in 2022 and is projected to increase during the forecast period (2023-2034)
• In 2022, the Familial Chylomicronemia Syndrome Market Size was highest in Germany among the 7MM accounting for approximately USD 5 million that is further expected to increase by 2034.
• The majority of Familial Chylomicronemia Syndrome patients either remain undiagnosed or get misdiagnosed leading to the progression of the disease to severe conditions.
• While the larger market is covered by Waylivra (volanesorsen) in 2022, this drug is available only in Europe. US regulators have rejected Akcea/Ionis' application to market WAYLIVRA for the ultra-rare rare lipid disorder familial chylomicronemia syndrome.
• Despite of prevalence with total diagnosed cases of around 1,350 in 2022 in the 7MM, the Familial Chylomicronemia Syndrome treatment market lacks approved therapy in the US and Japan.
• Olezarsen (ApoC-III) estimated to launch by 2024 in the US, is expected to be to bring a potential growth in the FCS market.
• Emerging therapies have the potential to create a significant positive shift in the Familial Chylomicronemia Syndrome market size.

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.

Familial Chylomicronemia Syndrome Epidemiology

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.

• As per DelveInsight's estimates, in 2022 theUS alone accounted for 65% of diagnosed prevalent FCS cases. The diagnosed prevalence of FCS is estimated to increase during the forecast period (2023-2034). The potential increase could be due to improved awareness, genetic predisposition, lifestyle changes, obesity rates, improved survival, shifting demographics, and environmental factors.
• Among the 7MM, EU4 and the UK accounted for nearly 23% diagnosed prevalent cases of Familial Chylomicronemia Syndrome (FCS) in 2022.
• Among EU4 and the UK, Germany had the highest diagnosed prevalent population of FCS, with nearly 135 cases, followed by the France and the UK in 2022. On the other hand, Spain had the lowest diagnosed prevalent population in EU4 and the UK in 2022.
• In Japan, there were around 4,886k diagnosed prevalent cases of FCS in 2022. These cases are expected to increase at a significant CAGR.
• Age-specific diagnosed prevalent cases of Familial Chylomicronemia Syndrome (FCS) were distributed in two age groups 0-17 years and 18 years and above.
• The highest proportion of FCS cases was estimated in the 0-17 years. FCS has been observed across all ages, making age one of the major factors for this condition. While understanding this indication and analyzing the various research papers, we observed that FCS cases are most commonly diagnosed in the younger population (infants and younger adults).

Familial Chylomicronemia Syndrome Recent Developments

• In January 2025, the FDA accepted a new drug application (NDA) for Arrowhead Pharmaceuticals' plozasiran, following the approval of the first treatment for familial chylomicronemia syndrome (FCS) a month earlier.
• In December 2024, the U.S. Food and Drug Administration approved Tryngolza (olezarsen) as an adjunct to diet for reducing triglycerides in adults with familial chylomicronemia syndrome (FCS), a condition that can cause life-threatening acute pancreatitis, according to Ionis Pharmaceuticals.

Familial Chylomicronemia Syndrome Drug Chapters

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

• WAYLIVRA (volanesorsen) is a self-administered SC injection with a prefilled syringe with a dosage for one-time use. In February 2019, the European Medicines Agency (EMA) gave conditional marketing authorization for the drug for patients with confirmed FCS at high risk of pancreatitis. There is an inadequate response to TG reduction therapy.
• Ionis Pharmaceuticals holds patents in the US and Europe covering the use of antisense compounds targeting human apoC-III, including the site of action for WAYLIVRA, providing protection against generics until 2023-2024.
• In August 2018, the US Food and Drug Administration (FDA) refused to approve volanesorsen for the treatment of patients with FCS based on safety issues of thrombocytopenia and risks of bleeding.

Familial Chylomicronemia Syndrome Emerging Drugs

• Olezarsen: Ionis Pharmaceuticals

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.

Familial Chylomicronemia Syndrome Market Outlook

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).

• The total Familial Chylomicronemia Syndrome Market Size in the 7MM was approximately USD 15 million in 2022 and is projected to increase during the forecast period (2023-2034)
• The Familial Chylomicronemia Syndrome Market Size in the 7MM will increase at a constant CAGR due to increasing awareness of the disease, better diagnosis, and the launch of the emerging target therapy for FCS.
• Among EU4 and the UK , Germany accounted for the maximum Familial Chylomicronemia Syndrome Market Size in 2022 with a contribution of 36%, while Spain occupied the bottom of the ladder in 2022.
• Japan's treatment market lacks approved targeted treatment for Familial Chylomicronemia Syndrome, but with the expected launch of Ionis Pharmaceuticals Olezarsen (ApoC-III) by 2026 the market is expected to experience positive growth.

Familial Chylomicronemia Syndrome Drugs Uptake

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

• The Familial Chylomicronemia Syndrome treatment market report covers a segment of key events, an executive summary, descriptive overview, explaining its causes, signs and symptoms, pathogenesis, and currently available therapies
• Comprehensive insight has been provided into the epidemiology segments and forecasts, the future growth potential of diagnosis rate, disease progression along with treatment guidelines
• Additionally, an all-inclusive account of both the current and emerging therapies along with the elaborative profiles of late-stage and prominent therapies will have an impact on the current Familial Chylomicronemia Syndrome treatment market landscape
• A detailed review of the Familial Chylomicronemia Syndrome treatment market; historical and forecasted Familial Chylomicronemia Syndrome drugs market size, Familial Chylomicronemia Syndrome market share by therapies, detailed assumptions, and rationale behind our approach is included in the report, covering the 7MM drug outreach
• The Familial Chylomicronemia Syndrome treatment market report provides an edge while developing business strategies, by understanding trends, through SWOT analysis and expert insights/KOL views, patient journey, and treatment preference that help in shaping and driving the 7MM Familial Chylomicronemia Syndrome Drugs market

Familial Chylomicronemia Syndrome Treatment Market Report Insights

• Patient-based Familial Chylomicronemia Syndrome Market Forecasting
• Familial Chylomicronemia Syndrome Therapeutic Approaches
• Familial Chylomicronemia Syndrome Pipeline Analysis
• Familial Chylomicronemia Syndrome Market Size and Trends
• Existing and future Familial Chylomicronemia Syndrome Drugs Market Opportunity

Familial Chylomicronemia Syndrome Treatment Market Report Key Strengths

• 12 Years Familial Chylomicronemia Syndrome Market Forecast
• 7MM Coverage
• Familial Chylomicronemia Syndrome Epidemiology Segmentation
• Key Cross Competition
• Attribute analysis
• Familial Chylomicronemia Syndrome Drugs Uptake
• Key Familial Chylomicronemia Syndrome Market Forecast Assumptions

Familial Chylomicronemia Syndrome Treatment Market Report Assessment

• Current Familial Chylomicronemia Syndrome Treatment Market Practices
• Familial Chylomicronemia Syndrome Unmet Needs
• Familial Chylomicronemia Syndrome Pipeline Product Profiles
• Familial Chylomicronemia Syndrome Drugs Market Attractiveness
• Qualitative Analysis (SWOT and Conjoint Analysis)
• Familial Chylomicronemia Syndrome Market Drivers
• Familial Chylomicronemia Syndrome Market Barriers

Key Questions Answered

Familial Chylomicronemia Syndrome Treatment Market Insights:

• What was the Familial Chylomicronemia Syndrome treatment market size, the treatment market size by therapies, and Familial Chylomicronemia Syndrome drugs market share (%) distribution in 2020, and how it would all look in 2034? What are the contributing factors for this growth?
• What are the unmet needs are associated with the current treatment market of FCS?
• What will be the impact of Olezarsen launch in in the market?
• Which drug is going to be the largest contributor in 2034?
• What are the pricing variations among different geographies for approved and off-label therapies?
• How would the market drivers, barriers, and future opportunities affect the market dynamics and subsequent analysis of the associated trends?

Familial Chylomicronemia Syndrome Epidemiology Insights:

• What are the disease risk, burden, and Familial Chylomicronemia Syndrome Unmet Needs? What will be the growth opportunities across the 7MM concerning the patient population of Familial Chylomicronemia Syndrome?
• What is the historical and forecasted Familial Chylomicronemia Syndrome patient pool in the United States, EU4 (Germany, France, Italy, and Spain) the United Kingdom, and Japan?
• Why do only limited patients appear for diagnosis? Why is the current year diagnosis rate not high?
• Which age group is more prevalent and why?
• What factors are affecting the diagnosis of the indication?

Current Familial Chylomicronemia Syndrome Treatment Market Scenario, Marketed Drugs, and Emerging Therapies:

• What are the current options for the treatment of Familial Chylomicronemia Syndrome? What are the current treatment guidelines for the treatment of Familial Chylomicronemia Syndrome in the US and Europe?
• How many companies are developing therapies for the treatment of Familial Chylomicronemia Syndrome?
• How many emerging therapies are in the mid-stage and late stage of development for the treatment of Familial Chylomicronemia Syndrome?
• What are the recent novel therapies, targets, mechanisms of action, and technologies developed to overcome the limitation of existing therapies?
• What are the key designations that have been granted for the emerging therapies for Familial Chylomicronemia Syndrome?
• What is the cost burden of approved therapies on the patient?
• Patient acceptability in terms of preferred treatment options as per real-world scenarios?
• What are the country-specific accessibility issues of expensive, recently approved therapies? Focus on reimbursement policies.
• What are the 7MM historical and forecasted Familial Chylomicronemia Syndrome Market?

Reasons to Buy:

• The Familial Chylomicronemia Syndrome treatment market report will help in developing business strategies by understanding the latest trends and changing treatment dynamics driving the Familial Chylomicronemia Syndrome Drugs Market
• Insights on patient burden/disease Familial Chylomicronemia Syndrome prevalence, evolution in diagnosis, and factors contributing to the change in the epidemiology of the disease during the forecast years
• To understand the existing Familial Chylomicronemia Syndrome drugs market opportunity in varying geographies and the growth potential over the coming years.
• Distribution of historical and current patient share based on real-world prescription data along with reported sales of approved products in the US, EU4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan.
• Identification of strong upcoming players in the Familial Chylomicronemia Syndrome drugs market will help in devising strategies that will help in getting ahead of competitors.
• Detailed analysis and ranking of class-wise potential current and emerging therapies under the Conjoint analysis section to provide visibility around leading classes
• Highlights of Access and Reimbursement policies of approved therapies, barriers to accessibility of off-label expensive therapies, and patient assistance programs
• To understand the perspective of Key Opinion Leaders' around the accessibility, acceptability, and compliance-related challenges of existing treatment to overcome barriers in future
• Detailed insights on the unmet need of the existing Familial Chylomicronemia Syndrome drugs market so that the upcoming players can strengthen their development and launch strategy

Table of Contents

1 Key Insights

2 Report Introduction

3 Familial Chylomicronemia Syndrome Market Overview at a Glance

• 3.1 Market Share (%) Distribution of Familial Chylomicronemia Syndrome by Therapies in 2020
• 3.2 Market Share (%) Distribution of Familial Chylomicronemia Syndrome by Therapies in 2034

4 Epidemiology and Market Methodology

5 Executive Summary of Familial Chylomicronemia Syndrome (FCS)

6 Key Events

7 Disease Background and Overview Familial Chylomicronemia Syndrome (FCS)

• 7.1 Introduction
• 7.2 Pathophysiology and Histopathology of FCS
• 7.3 Symptoms of FCS
• 7.4 Prognosis and Complications
• 7.5 Diagnosis
  • 7.5.1 Diagnostic Criteria for FCS
  • 7.5.2 Diagnostic Algorithm
• 7.6 Treatment and Management

8 Epidemiology and Patient Population of Familial Chylomicronemia Syndrome (FCS)

• 8.1 Key Findings
• 8.2 Assumptions and Rationale: The 7MM
• 8.3 Total Diagnosed Prevalent Cases of Familial Chylomicronemia Syndrome (FCS) in the 7MM
• 8.4 The United States
  • 8.4.1 Total Diagnosed Prevalent Cases of FCS in the US
  • 8.4.2 Age-specific Diagnosed Prevalent Cases of FCS in the US
• 8.5 EU4 and the UK
  • 8.5.1 Germany
    • 8.5.1.1 Total Diagnosed Prevalent Cases of FCS in Germany
    • 8.5.1.2 Age-specific Diagnosed Prevalent Cases of FCS in Germany
  • 8.5.2 France
    • 8.5.2.1 Total Diagnosed Prevalent Cases of FCS in France
    • 8.5.2.2 Age-specific Diagnosed Prevalent Cases of FCS in France
  • 8.5.3 Italy
    • 8.5.3.1 Total Diagnosed Prevalent Cases of FCS in Italy
    • 8.5.3.2 Age-specific Diagnosed Prevalent Cases of FCS in Italy
  • 8.5.4 Spain
    • 8.5.4.1 Total Diagnosed Prevalent Cases of FCS in Spain
    • 8.5.4.2 Age-specific Diagnosed Prevalent Cases of FCS in Spain
  • 8.5.5 The United Kingdom
    • 8.5.5.1 Total Diagnosed Prevalent Cases of FCS in the UK
    • 8.5.5.2 Age-specific Diagnosed Prevalent Cases of FCS in the UK
• 8.6 Japan
  • 8.6.1 Total Diagnosed Prevalent Cases of FCS in Japan
  • 8.6.2 Age-specific Diagnosed Prevalent Cases of FCS in Japan

9 Patient Journey

10 Marketed Drugs

• 10.1 WAYLIVRA (volanesorsen): Akcea Therapeutics Ireland Limited
  • 10.1.1 Product description
  • 10.1.2 Regulatory milestones
  • 10.1.3 Other development activities
  • 10.1.4 Clinical development
  • 10.1.5 Clinical trials information
  • 10.1.6 Safety and efficacy
  • 10.1.7 Product profile

11 Emerging Drugs

11. Key Cross Competition

• 11.2 Olezarsen: Ionis Pharmaceuticals
  • 11.2.1 Product description
  • 11.2.2 Other developmental activity
  • 11.2.3 Clinical development
  • 11.2.4 Clinical trials information
  • 11.2.5 Product profile
  • 11.2.6 Analysts' views
• 11.3 ARO-APOC3: Arrowhead Pharmaceuticals, Inc.
  • 11.3.1 Product description
  • 11.3.2 Other developmental activity
  • 11.3.3 Clinical development
  • 11.3.4 Clinical trials information
  • 11.3.5 Safety and efficacy
  • 11.3.6 Product profile
  • 11.3.7 Analysts' views

12 Familial Chylomicronemia Syndrome (FCS): Seven Major Market Analysis

• 12.1 Key Findings
• 12.2 Market Outlook
• 12.3 Key Market Forecast Assumptions
• 12.4 Conjoint Analysis
• 12.5 Total Market Size of Familial Chylomicronemia Syndrome (FCS) in the 7MM
• 12.6 Market Size of Familial Chylomicronemia Syndrome (FCS) by Therapies in the 7MM
• 12.7 The United States Market Size
  • 12.7.1 Total Market Size of FCS in the US
  • 12.7.2 Market Size of FCS by Therapies in the US
• 12.8 EU4 and the UK Market Size
  • 12.8.1 Germany
    • 12.8.1.1 Total Market Size of FCS
    • 12.8.1.2 Market Size of FCS by Therapies
  • 12.8.2 France
    • 12.8.2.1 Total Market Size of FCS
    • 12.8.2.2 Market Size of FCS by Therapies
  • 12.8.3 Italy
    • 12.8.3.1 Total Market Size of FCS
    • 12.8.3.2 Market Size of FCS by Therapies
  • 12.8.4 Spain
    • 12.8.4.1 Total Market Size of FCS
    • 12.8.4.2 Market Size of FCS by Therapies
  • 12.8.5 The UK
    • 12.8.5.1 Total Market Size of FCS
    • 12.8.5.2 Market Size of FCS by Therapies
• 12.9 Japan Market Size
  • 12.9.1 Total Market Size of FCS in Japan
  • 12.9.2 Market Size of FCS by Therapies in Japan

13 SWOT Analysis

14 Unmet Needs

15 Key Opinion Leaders' Views

16 Market Access and Reimbursement

• 16.1 The United States
  • 16.1.1 Center for Medicare and Medicaid Services (CMS)
• 16.2 In EU4 and the UK
  • 16.2.1 Germany
  • 16.2.2 France
  • 16.2.3 Italy
  • 16.2.4 Spain
  • 16.2.5 United Kingdom
• 16.3 Japan
  • 16.3.1 MHLW

17 Appendix

• 17.1 Bibliography
• 17.2 Acronyms and Abbreviations
• 17.3 Report Methodology

18 DelveInsight Capabilities

19 Disclaimer

20 About DelveInsight