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市場調查報告書

美國的Cryopyrin相關週期性症候群(CAPS)市場:洞察,流行病學,2030年為止的預測

Cryopyrin-associated Periodic Syndromes (CAPS) - Market Insights, Epidemiology, and Market Forecast - 2030 (US)

出版商 DelveInsight Business Research LLP 商品編碼 973318
出版日期 按訂單生產 內容資訊 英文 81 Pages
商品交期: 10個工作天內
價格
美國的Cryopyrin相關週期性症候群(CAPS)市場:洞察,流行病學,2030年為止的預測 Cryopyrin-associated Periodic Syndromes (CAPS) - Market Insights, Epidemiology, and Market Forecast - 2030 (US)
出版日期: 按訂單生產內容資訊: 英文 81 Pages
簡介

本報告提供美國的Cryopyrin相關週期性症候群(CAPS)的相關調查,提供目前治療方法及新藥,各治療方法的市場佔有率,從2017年到2030年的市場規模及預測,成長要素和課題,未滿足需求等資訊。

目錄

第1章 主要洞察

第2章 摘要整理

第3章 SWOT分析

第4章 市場佔有率:各治療法流通概要

第5章 流行病學與市場預測流通量

第6章 疾病的背景和概要

  • 伴隨徵兆與症狀的重病度
  • 原因
  • 病因
  • 診斷
    • 成像
  • 診斷標準
  • 診斷演算法

第7章 流行病學和患者人口

  • 流行病學的主要調查結果
  • 前提條件與理論的根據
  • 美國流行病學
    • 美國CAPS的整體盛行率
    • 美國確診的CAPS的盛行率總合
    • 美國CAPS的各年齡的盛行率
    • 美國CAPS的各類型的盛行率
    • 美國CAPS的重症度別的盛行率
    • 美國的CAPS的治療案例

第8章 目前治療方法

  • 標準的治療方法
    • NSAID
    • 皮質類固醇

第9章 未滿足需求

第10章 患者療程

第11章 CAPS臨床試驗的主要端點

第12章 市售的治療藥

  • Kineret (Anakinra): Amgen/Swedish Orphan Biovitrum
    • 產品概要
    • 法規的里程碑
    • 其他開發活動
    • 主要的臨床實驗
  • Ilaris (Canakinumab): Novartis
  • Arcalyst (Rilonacept): Regeneron Pharmaceuticals
    • 主要的臨床實驗

第13章 新治療方法

  • ATI-450:Aclaris Therapeutics
    • 產品概要
    • 其他開發活動
    • 臨床開發

第14章 CAPS:美國市場分析

  • 主要調查結果
  • 市場預測
  • 美國的市場規模
    • CAPS的整體市場規模
    • 美國的各治療法整體市場規模

第15章 CAPS治療的市場進入與償付

  • 罕見疾病治療的償付
  • CAPS的患者支援計劃

第16章 市場成長

第17章 市場障礙

第18章 附錄

  • 參考文獻
  • 報告的調查手法

第19章 DelveInsight的服務內容

第20章 免責聲明

第21章 關於DelveInsight

目錄

DelveInsight's 'Cryopyrin-associated Periodic Syndromes (CAPS) - Market Insights, Epidemiology and Market Forecast-2030' report delivers an in-depth understanding of the CAPS, historical and forecasted epidemiology as well as the CAPS market trends in the United States.

The CAPS market report provides current treatment practices, emerging drugs, CAPS market share of the individual therapies, current and forecasted CAPS market size from 2017 to 2030 in the United States. The Report also covers current CAPS treatment practice/algorithm, market drivers, market barriers and unmet medical needs to curate best of the opportunities and assesses underlying potential of the market.

Geography Covered:

  • The United States

Study Period: 2017-2030

Cryopyrin-associated Periodic Syndromes (CAPS): Disease Understanding and Treatment Algorithm

Overview:

Cryopyrin-associated Periodic Syndromes (CAPS) is a group of auto-inflammatory as well as known autosomal dominant disorder that is believed to be caused by owing to mutations in the NLRP3 gene encoding a protein called cryopyrin. NLRP3 is a part of the inflammasome protein. For initiation of the innate inflammatory response, inflammasome activation is needed. Other CAPS also cause mutations in NLRP3, which in turn leads to the overproduction of Interleukin beta-1 (IL-1B) CAPS and basically comprises of three diseases that vary with the severity and the system they are involved in. The mode of inheritance is autosomal dominant with variable penetrance (means only one copy of the gene from one parent is sufficient enough to cause this medical condition).

CAPS comprises a group of three disorders, varying with their severity and the organs they tend to affect upon the onset, namely Neonatal Onset Multisystem Inflammatory Disease (NOMID), also called as Chronic Inflammatory Neurological Cutaneous Articular Syndrome (CINCA), Familial cold auto inflammatory syndrome (FCAS) and Muckle-Wells syndrome (MWS). NOMID is the most severe form of CAPS, along with episodic fever and chills, is accompanied by other associated symptoms like Central Nervous System Inflammation fever bouts (beginning in infancy), followed by rash flare-ups, and elevated joint pain and red eyes. On the other hand, MWS is an intermediate form of CAPS. In this case, symptoms like frequent rashes starts in infancy or early adolescence. Occasional fever, joint pain, frequent conjunctivitis, gradual hearing loss, and amyloidosis are symptoms of MWS. FCAS, also known as familial cold urticariais is the mildest form of CAPS with fever and other symptoms, the onset of rash flares begins in early childhood or occasionally in early childhood in all FCAS patients. Hearing loss and Amyloidosis renal disease is rare in FCAS patients. The symptoms and severity of CAPS vary by disease, but some of the sign include, inflammation in multiple organs, hive-like rash, periodic fever and flare-ups, headache, bulging eyes, blindness, hearing loss, vomiting, joint pain, swelling etc. Other symptoms that are specific to one type of disease include, hive-like rash on exposure to cold or other environmental triggers in FCAS and in MWS, symptoms may come and go.

Cryopyrin-associated Periodic Syndromes (CAPS) Diagnosis

Diagnosis of CAPS is based on clinical manifestations and on laboratory findings revealing generalized leukocytosis and neurophilia along with others elevated acute phase reactants (C reactive protein and serum amyloid A). Some of the laboratory tests that are used include, genetic testing that recognizes differences in chromosomes, genes, or proteins. Skin Biopsy of the rash, eye examination, hearing tests, imaging tests, etc.

Cryopyrin-associated Periodic Syndromes (CAPS) Treatment

In order to treat a rare disease such as CAPS, several anti-inflammatory medications, immunosuppressants, and antihistamines have been tested to regulate the signs and symptoms seen in CAPS. These include, methotrexate, cyclosporine, azathioprine, cyclophosphamide, and tumor necrosis factor blockers but they have all been unsuccessful. In recent years, research has shown that biologic drugs targeting interleukin-1 are effective in treating CAPS. Anakinra, rilonacept, and canakinumab are included in these IL-1 inhibitors. These three drugs are FDA approved and majorly contributes in treating CAPS's patients. It is essential to take these therapies for life, but it will encourage children to lead near-normal lives. Some therapies may also include physical therapy, joint alignment medication splints, and NSAIDs, symptom-lowering corticosteroids, or methotrexate. Surgery can correct deformities in the joint.

CAPS Epidemiology

The CAPS epidemiology division provides the insights about historical and current CAPS patient pool and forecasted trend for United States. It helps to recognize the causes of current and forecasted trends by exploring numerous studies and views of key opinion leaders. This part of the DelveInsight report also provides the diagnosed patient pool and their trends along with assumptions undertaken.

Key Findings

In the year 2017, the total prevalent case of CAPS was 650 cases in the United States which are expected to grow during the study period, i.e., 2017-2030.

The disease epidemiology covered in the report provides historical as well as forecasted CAPS epidemiology [segmented as Total Prevalent Cases of CAPS, Total Diagnosed Cases of CAPS, Age-specific Prevalent cases of CAPS, Type-specific Prevalent Cases of CAPS, Severity-specific Prevalent Cases of CAPS and Treated cases of CAPS] in the United States from 2017 to 2030.

CAPS Drug Chapters

The drug chapter segment of the CAPS report encloses the detailed analysis of CAPS marketed drugs and pipeline drugs. It also helps to understand the CAPS clinical trial details, expressive pharmacological action, agreements and collaborations, approval and patent details, advantages and disadvantages of each included drug and the latest news and press releases.

CAPS Approved Drugs

Kineret (Anakinra): Amgen/ Swedish Orphan Biovitrum

Kineret (anakinra) is a recombinant, non-glycosylated form of the human interleukin-1 receptor antagonist (IL-1Ra). In January 2013, the US Food and Drug Administration (FDA) approved Kineret for the treatment of NOMID. Kineret was the first and only FDA-approved therapy treatment for NOMID, the most severe form of CAPS.

Ilaris (Canakinumab): Novartis

Ilaris (Canakinumab), previously known as ACZ885, is a recombinant, human anti-human-IL-1B monoclonal antibody that belongs to the IgG1/K isotype subclass. In June 2009, the US FDA approved Ilaris (canakinumab) for the treatment of children and adults with CAPS, which includes a number of rare but life-long auto-inflammatory disorders with debilitating symptoms and limited treatment options. The FDA granted priority review to Ilaris based on its potential to meet an important clinical need for patients with CAPS.

Arcalyst (Rilonacept): Regeneron Pharmaceuticals

Rilonacept, formerly known as IL-1 TRAP, is a targeted inhibitor of interleukin-1 (IL-1), the key driver of inflammation in CAPS. Rilonacept is a dimeric fusion protein consisting of the ligand-binding domains of the extracellular portions of the human interleukin-1 receptor component (IL-1RI) and IL-1 receptor accessory protein (IL-1RAcP) linked in-line to the Fc portion of human IgG1. Arcalyst is used to treat adults and children 12 years of age and older with CAPS, including FCAS and MWS. In February 2008, the US FDA approved Arcalyst (rilonacept) an interleukin-1 blocker, for the treatment of CAPS, including FCAS and MWS in adults and children 12 and older.

Note: Detailed Current therapies assessment will be provided in the full report of CAPS

CAPS Emerging Drugs

ATI-450 (Aclaris Therapeutics)

ATI-450 formerly known as CDD-450 is an investigational drug which is a unique p38α MAPK-MK2 pathway selective inhibitor used to expose the function of this protein complex in inflammasome priming signals. Notably, ATI-450 is as efficacious as global p38α MAPK inhibitors in decreasing inflammation in disease models. CAPS and the most severe form of the disease, NOMID result from dysregulated inflammasome generating elevated systemic levels of the cytokine IL-1B.

In August 2017, Aclaris Therapeutics, announced that it has acquired Confluence Life Sciences-a privately held biotechnology company focused on the discovery and development of kinase inhibitors to treat inflammatory and immunological disorders and cancer. The issued U.S. patents covering ATI-450, are expected to expire in 2034 and other issued patents covering different MK2 signaling pathway inhibitors are expected to expire in 2031 and 2032.

Note: Detailed emerging therapies assessment will be provided in the final report.

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CAPS Market Outlook

Key Findings

The CAPS market size in the US is expected to change during the study period 2017-2030. The therapeutic market of CAPS in the US was USD 36 million in 2017 which is expected to increase during study period (2017-2030).

CAPS Pipeline Development Activities

The drugs which are in pipeline include:

  • ATI-450: Aclaris Therapeutics

Note: Detailed emerging therapies assessment will be provided in the final report.

CAPS Drugs Uptake

Among the approved therapies, Canakinumab is dominating the current market owing to well-established efficacy and safety profile, rapid and sustained response, and a significantly less-frequent dosing schedule (i.e., once every eight weeks).

Access and Reimbursement Scenario in CAPS Therapies

  • The high-cost of rare disease drugs usually have a limited impact on budget due to the small number of eligible patients being prescribed the drug. Several rare disease therapies have been approved by the US FDA in the past few years. From a patient perspective, health insurance and payer coverage guidelines surrounding rare disease treatments restrict broad access to these treatments, leaving only a small number of patients who can bypass insurance and pay for products on their own. Insured patients in the United States are typically granted access to rare disease drugs. This is due to several factors, including about half of rare diseases affecting children, which makes management difficult, without coverage after approval.

KOL-Views

To keep up with current market trends, we take KOLs and SME's opinion working in CAPS domain through primary research to fill the data gaps and validate our secondary research. Their opinion helps to understand and validate current and emerging therapies treatment patterns or CAPS market trend. This will support the clients in potential upcoming novel treatment by identifying the overall scenario of the market and the unmet needs.

Competitive Intelligence Analysis

We perform Competitive and Market Intelligence analysis of the CAPS Market by using various Competitive Intelligence tools that includes - SWOT analysis, PESTLE analysis, Porter's five forces, BCG Matrix, Market entry strategies etc. The inclusion of the analysis entirely depends upon the data availability.

Scope of the Report:

  • The report covers the descriptive overview of CAPS, explaining its causes, signs and symptoms, pathophysiology and currently available therapies.
  • Comprehensive insight has been provided into the CAPS epidemiology and treatment in the US.
  • Additionally, an all-inclusive account of both the current and emerging therapies for CAPS is provided, along with the assessment of new therapies, which will have an impact on the current treatment landscape.
  • A detailed review of CAPS market; historical and forecasted is included in the report, covering drug outreach in the US.
  • The report provides an edge while developing business strategies, by understanding trends shaping and driving the global CAPS market.

Report Highlights:

  • In the coming years, CAPS market is set to change due to the rising awareness of the disease and incremental healthcare spending across the world; which would expand the size of the market to enable the drug manufacturers to penetrate more into the market.
  • The companies and academics are working to assess challenges and seek opportunities that could influence CAPS R&D. The therapies under development are focused on novel approaches to treat/improve the disease condition.
  • Major players are involved in developing therapies for CAPS. Launch of emerging therapies, will significantly impact the CAPS market.
  • A better understanding of disease pathogenesis will also contribute to the development of novel therapeutics for CAPS.
  • Our in-depth analysis of the pipeline assets across different stages of development, different emerging trends and comparative analysis of pipeline products with detailed clinical profiles, key cross-competition, and launch date along with product development activities will support the clients in the decision-making process regarding their therapeutic portfolio by identifying the overall scenario of the research and development activities.

CAPS Report Insights

  • Patient Population
  • Therapeutic Approaches
  • CAPS Pipeline Analysis
  • CAPS Market Size and Trends
  • Market Opportunities
  • Impact of upcoming Therapies

CAPS Report Key Strengths

  • 11-Years Forecast
  • US Coverage
  • CAPS Epidemiology Segmentation
  • Key Cross Competition
  • Highly Analyzed Market
  • Drugs Uptake

CAPS Report Assessment

  • SWOT Analysis
  • Current Treatment Practices
  • Unmet Needs
  • Pipeline Product Profiles
  • Conjoint Analysis
  • Market Attractiveness
  • Market Drivers and Barriers

Key Questions:

Market Insights:

  • What was the CAPS Market share (%) distribution in 2017 and how it would look like in 2030?
  • What would be the CAPS total market size as well as market size by therapies across the US during the study period (2017-2030)?
  • What are the key findings pertaining to the market across the United States (2017-2030)?
  • At what CAGR, the CAPS market is expected to grow in the US during the study period (2017-2030)?
  • What would be the CAPS market outlook across the US during the study period (2017-2030)?
  • What would be the CAPS market growth till 2030 and what will be the resultant market size in the year 2030?
  • How would the market drivers, barriers and future opportunities affect the market dynamics and a subsequent analysis of the associated trends?
  • CAPS patient types/pool where unmet need is more and whether emerging therapies will be able to address the residual unmet need?
  • How emerging therapies are performing on the parameters like efficacy, safety, route of administration (RoA), treatment duration and frequencies on the basis of their clinical trial results?
  • Among the emerging therapies, what are the potential therapies which are expected to disrupt the CAPS market?

Epidemiology Insights:

  • What is the disease risk, burden and unmet needs of the CAPS?
  • What is the historical CAPS patient pool in the United States?
  • What would be the forecasted patient pool of CAPS in the United States?
  • What will be the growth opportunities in the United States with respect to the patient population pertaining to CAPS?
  • At what CAGR the population is expected to grow in the United States during the study period (2017-2030)?
  • What are the various recent and upcoming events which are expected to improve the diagnosis of CAPS?

Current Treatment Scenario and Emerging Therapies:

  • What are the current options for the treatment of CAPS?
  • What are the current treatment guidelines for the treatment of CAPS in the US?
  • How many companies are developing therapies for the treatment of CAPS?
  • How many therapies are developed by each company for the treatment of CAPS?
  • How many emerging therapies are in mid stage, and late stage of development for the treatment of CAPS?
  • What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the CAPS therapies?
  • What are the recent novel therapies, targets, mechanisms of action and technologies developed to overcome the limitation of existing therapies?
  • What are the clinical studies going on for CAPS and their status?
  • What are the key designations that have been granted for the emerging therapies for CAPS?
  • What is the global historical and forecasted market of CAPS?

Reasons to buy:

  • The report will help in developing business strategies by understanding trends shaping and driving the CAPS market.
  • To understand the future market competition in the CAPS market and Insightful review of the key market drivers and barriers.
  • Organize sales and marketing efforts by identifying the best opportunities for CAPS in the US.
  • Identification of strong upcoming players in market will help in devising strategies that will help in getting ahead of competitors.
  • Organize sales and marketing efforts by identifying the best opportunities for CAPS market.
  • To understand the future market competition in the CAPS market.

Table of Contents

1. Key Insights

2. Executive Summary

3. SWOT Analysis

4. Market Share (%) Distribution Overview at a Glance: By Therapies

5. Epidemiology and Market Forecast Flow

6. Cryopyrin-associated Periodic Syndromes (CAPS): Disease Background and Overview

  • 6.1. Severity along with Signs and Symptoms
  • 6.2. Causes
  • 6.3. Pathogenesis
  • 6.4. Diagnosis
    • 6.4.1. Imaging
  • 6.5. Diagnostic Criteria
  • 6.6. Diagnostic Algorithm

7. Epidemiology and Patient Population

  • 7.1. Epidemiology Key Findings
  • 7.2. Assumptions and Rationale
  • 7.3. The United States Epidemiology
    • 7.3.1. Total Prevalent Cases of CAPS in the United States
    • 7.3.2. Total Diagnosed Prevalent Cases of CAPS in the United States
    • 7.3.3. Age-Specific Prevalent Cases of CAPS in the United States
    • 7.3.4. Type-specific Prevalent Cases of CAPS in the United States
    • 7.3.5. Severity-specific Prevalent Cases of CAPS in the United States
    • 7.3.6. Treated cases of CAPS in the United States

8. Current Treatment Practices

  • 8.1. Standard Therapies
    • 8.1.1. NSAIDs
    • 8.1.2. Corticosteroids

9. Unmet Needs

10. Patient Journey

11. Key Endpoints in CAPS Clinical Trials

12. Marketed Therapies

  • 12.1. Kineret (Anakinra): Amgen/ Swedish Orphan Biovitrum
    • 12.1.1. Product Description
    • 12.1.2. Regulatory Milestones
    • 12.1.3. Other Developmental Activities
    • 12.1.4. Pivotal Clinical Trials
  • 12.2. Ilaris (Canakinumab): Novartis
    • 12.2.1. Product Description
    • 12.2.2. Regulatory Milestones
    • 12.2.3. Other Developmental Activities
    • 12.2.4. Pivotal Clinical Trials
  • 12.3. Arcalyst (Rilonacept): Regeneron Pharmaceuticals
    • 12.3.1. Product Description
    • 12.3.2. Regulatory Milestones
    • 12.3.3. Other Developmental Activities
    • 12.3.4. Pivotal Clinical Trial

13. Emerging Therapies

  • 13.1. ATI-450: Aclaris Therapeutics
    • 13.1.1. Product Description
    • 13.1.2. Other Developmental Activities
    • 13.1.3. Clinical Development

14. CAPS: United States Market Analysis

  • 14.1. Key Findings
  • 14.2. Market Outlook
  • 14.3. The United States Market Size
    • 14.3.1. Total Market size of CAPS
    • 14.3.2. Total Market Size by Therapies in the United States

15. Market Access and Reimbursement of CAPS Therapies

  • 15.1. Reimbursement of Rare Diseases Therapies
  • 15.2. Patient Assistance Program for CAPS

16. Market Drivers

17. Market Barriers

18. Appendix

  • 18.1. Bibliography
  • 18.2. Report Methodology

19. DelveInsight Capabilities

20. Disclaimer

21. About DelveInsight

List of Tables

  • Table 1: Summary of CAPS-Market, Epidemiology, and Key Events (2017-2030)
  • Table 2: CAPS Types
  • Table 3: Causes of CAPS
  • Table 4: Raised Inflammatory markers (CRP/SAA) (mandatory criteria) plus
  • Table 5: Items and definitions for diagnostic criteria for CAPS
  • Table 6: Total Prevalent Cases of CAPS in the United States (2017-2030)
  • Table 7: Total Diagnosed Prevalent Cases of CAPS in the United States (2017-2030)
  • Table 8: Age-Specific Prevalent Cases of CAPS in the United States (2017-2030)
  • Table 9: Type-specific Prevalent Cases of CAPS in the United States (2017-2030)
  • Table 10: Severity-specific Prevalent Cases of CAPS in the United States (2017-2030)
  • Table 11: Total Treated cases of CAPS in the United States (2017-2030)
  • Table 12:Advantages and Disadvantages of Treatment Options
  • Table 13: ATI-450, Clinical Trial Description, 2020
  • Table 14: United States Market Size of CAPS, in USD Million (2017-2030)
  • Table 15: Total Market Size of CAPS by Therapies in the United States, in USD Million (2017-2030)

List of Figures

  • Figure 1: CAPS SWOT Analysis
  • Figure 2: Manifestation of Severity
  • Figure 3: Symptoms of different forms of CAPS (I) Hive-like rashes (II) Conjunctivitis (III) Fever (IV) Joint Pain
  • Figure 4:The initiation of diseases
  • Figure 5 The structure of the inflammasome, intracellular signaling pathways, and therapeutic targets in autoinflammatory diseases
  • Figure 6: Items and definitions for diagnostic criteria for CAPS
  • Figure 7: Total Prevalent Cases of CAPS in the United States (2017-2030)
  • Figure 8: Total Diagnosed Prevalent Cases of CAPS in the United States (2017-2030)
  • Figure 9: Age-Specific Prevalent Cases of CAPS in the United States (2017-2030)
  • Figure 10: Type-specific Prevalent Cases of CAPS in the United States (2017-2030)
  • Figure 11: Severity-specific Prevalent Cases of CAPS in the United States (2017-2030)
  • Figure 12: Treated cases of CAPS in the United States (2017-2030)
  • Figure 13: Unmet Needs of CAPS
  • Figure 14: Patient Journey
  • Figure 15: Group Mean Daily Symptom Scores by Treatment Group in Part A and Single-blind Arcalyst Treatment Phase from Week -3 to Week 15
  • Figure 16: United States Market Size of CAPS, in USD Million (2017-2030)
  • Figure 17: Total Market Size of CAPS by Therapies in the United States, in USD Million (2017-2030)
  • Figure 18: Market Drivers
  • Figure 19: Market Barriers