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US Thrombotic Thrombocytopenic Purpura (TTP) Market Insight, Epidemiology and Market Forecast -2030

  • ID: 5457231
  • Report
  • October 2021
  • Region: United States
  • 110 Pages
  • DelveInsight

FEATURED COMPANIES

  • Baxalta/Takeda
  • Octapharma
  • Omeros Corporation
  • Sanofi
This ‘Thrombotic Thrombocytopenic Purpura (TTP) -Market Insights, Epidemiology, and Market Forecast-2030' report delivers an in-depth understanding of the Thrombotic Thrombocytopenic Purpura (TTP) and its historical and forecasted epidemiology as well as market trends in the United States.

The Thrombotic Thrombocytopenic Purpura (TTP) market report provides current treatment practices, emerging drugs, and market share of the individual therapies, current and forecasted US Thrombotic Thrombocytopenic Purpura (TTP) market size from 2018 to 2030. The Report also covers current Thrombotic Thrombocytopenic Purpura (TTP) treatment practice/algorithm, market drivers, market barriers, unmet medical needs, SWOT analysis to curate the best of the opportunities, and assesses the underlying potential of the market.

Geography Covered

  • The United States

Study Period: 2018-2030

Thrombotic Thrombocytopenic Purpura (TTP) Disease Understanding and Treatment Algorithm


Thrombotic Thrombocytopenic Purpura (TTP) Overview


Thrombotic thrombocytopenic purpura (TTP) is a rare life-threatening thrombotic microangiopathy arteriolar platelet-rich thrombi that cause organ ischemia and produce neurologic abnormalities, kidney dysfunction, thrombocytopenia, and microangiopathic hemolytic anemia (MAHA). TTP is caused by a severe deficiency of the von Willebrand factor-cleaving protease ADAMTS13. TTP can be acquired or hereditary; thus, TTP is classified according to its etiology.

TTP is classified into two types, first is Congenital TTP (cTTP), caused due to a mutation of the gene, ADAMTS13 responsible for desynthesizing, while the other is the most common form of TTP, Acquired TTP (aTTP), in which antibodies are created against ADAMTS13.

Thrombotic Thrombocytopenic Purpura (TTP) Diagnosis


Diagnosis is performed through clinical history and the examination of a peripheral blood smear. The measurement of ADAMTS13 levels and anti ADAMTS13 antibodies is most recommended. However, the treatment with plasmapheresis and plasma exchange is not to be delayed while waiting for the results of these measurements.

Thrombotic Thrombocytopenic Purpura (TTP) Treatment


Plasma therapy (also called plasmapheresis) is the foundation of front-line and relapsed TTP management. It involves two methods: fresh frozen plasma for people who have inherited TTP and plasma exchange for people who have acquired TTP. The proposed mechanism of therapeutic plasma exchange (TPE) supplies adequate levels of ADAMTS13 while removing circulating anti-ADAMTS13 autoantibodies. Any delay in therapy can lead to early mortality, which is preventable with prompt initiation of TPE.

cTTP treatment is primarily focused on maintaining ADAMTS13 levels. This can be achieved by the TPE method, but due to ongoing treatment, this is hardly used. Instead, infusion of ADAMTS13-rich blood products is mainly used. The most preferred treatment is fresh-frozen plasma (FFP) infusion despite the availability of many products, including cryosupernatant, whole blood, and cryoprecipitate, which are historically used methods. In 2013, the US FDA approved Octaplas, a human plasma for infusion, indicated for transfusion or plasma exchange in patients with congenital or acquired TTP. TPE is done once daily, while in relapsed cases, more intense TPE is considered, such as twice-daily TPE.

The standard of care for aTTP patients is caplacizumab used in conjunction with plasma exchange, corticosteroids, and rituximab.

Thrombotic Thrombocytopenic Purpura (TTP) Epidemiology
The disease epidemiology covered in the report provides historical as well as forecasted epidemiology segmented by Total Diagnosed Prevalence of Thrombotic Thrombocytopenic Purpura, Type-specific Diagnosed Prevalence of Thrombotic Thrombocytopenic Purpura, Gender-specific Diagnosed Prevalent Cases of Acquired Thrombotic Thrombocytopenic Purpura (aTTP), Gender-specific Diagnosed Prevalent Cases of Congenital Thrombotic Thrombocytopenic Purpura (cTTP), Age-specific Diagnosed Prevalent Cases of Acquired Thrombotic Thrombocytopenic Purpura (aTTP), and Age-specific Diagnosed Prevalent Cases of Congenital Thrombotic Thrombocytopenic Purpura (cTTP), in the United States from 2018 to 2030.

Key Findings

  • As per the publisher's analysis, the diagnosed prevalent cases of thrombotic thrombocytopenic purpura (TTP) in the United States were 6,002 in 2020.
  • As per the publisher's analysis, the type-specific diagnosed prevalent cases of thrombotic thrombocytopenic purpura (TTP) in the United States showed that there were 5,401 cases of aTTP and 600 cases of cTTP in 2020.
  • According to the publisher, out of 5,401 aTTP cases, there were 1,620 males and 3,781 females in the United States in 2020.
  • According to the publisher, there were 270 males and 330 females diagnosed with congenital TTP (cTTP) in the US in 2020.
  • Acquired TTP (aTTP) was found to be most prevalent in the patients of age group 18-40 years. Diagnosed prevalent cases of aTTP were found to be 74; 2,516; 1,924; and 888 in the age group < 18 years, 18-40 years, 41-60 years, and 60+ years, respectively in the United States, in 2020.
  • Congenital TTP (cTTP) was more prominent in the 18-40 years age group. In 2020, 180; 258; 114; and 48 diagnosed prevalent cases of aTTP were found in the age groups of < 18 years, 18-40 years, 41-60 years, and 60+ years, respectively.

Thrombotic Thrombocytopenic Purpura (TTP) Epidemiology


The epidemiology segment also provides the Thrombotic Thrombocytopenic Purpura (TTP) epidemiology data and findings across the United States.

Thrombotic Thrombocytopenic Purpura (TTP) Drug Chapters


The drug chapter segment of the Thrombotic Thrombocytopenic Purpura (TTP) report encloses the detailed analysis of Thrombotic Thrombocytopenic Purpura (TTP) current therapies, mid-phase, and late-stage pipeline drugs. It also helps to understand the Thrombotic Thrombocytopenic Purpura (TTP) clinical trial details, expressive pharmacological action, agreements and collaborations, approval and patent details of each included drug, and the latest news and press releases.

Thrombotic Thrombocytopenic Purpura (TTP) Marketed Drugs


Cablivi (Caplacizumab/ALX-0081): Sanofi (Ablynx)


Cablivi is a von Willebrand factor (vWF)-directed antibody fragment indicated for treating adult patients with acquired thrombotic thrombocytopenic purpura (aTTP), in combination with plasma exchange and immunosuppressive therapy. It is approved in the US and EU for the treatment of aTTP. A Phase II/III trial is still ongoing in Japan for TTP.

Octaplas/OctaplasLG: Octapharma

OctaplasLG and Octaplas are human plasma for infusion of pharmaceutical grade with standardized quality and unique safety profiles. Their integrated pathogen safety concept represents state-of-the-art security for patients. The standardized content in octaplasLG and Octaplas is achieved by pooling between 630 and 1520 single plasma units from multiple donors. The therapies are indicated in complex deficiencies of coagulation factors such as Therapeutic plasma exchange procedures, including those in thrombotic thrombocytopenic purpura (TTP).

Thrombotic Thrombocytopenic Purpura (TTP) Emerging Drugs


TAK-755 (BAX930/SHP655): Baxalta/Takeda

TAK-755 (BAX930/SHP655) is a human recombinant ADAMTS13, the plasma metalloprotease that regulates the von Willebrand Factor (VWF) multimers. Takeda is currently evaluating TAK-755 in Phase III clinical trial to treat congenital thrombotic. The company is also evaluating the drug for in the Prophylactic and On-demand treatment of subjects with severe Congenital Thrombotic Thrombocytopenic Purpura (cTTP, Upshaw-Schulman Syndrome [USS], Hereditary Thrombotic Thrombocytopenic Purpura [hTTP]).

Narsoplimab (OMS721): Omeros Corporation

Narsoplimab (OMS721) is an intravenous, human monoclonal antibody targeting mannan-binding lectin-associated serine protease-2 (MASP-2), the effector enzyme of the lectin pathway of the complement system. The lectin pathway is one of the principal complement pathways and is activated primarily by tissue damage and microbial infection. Importantly, inhibition of MASP-2 does not appear to interfere with the classical complement pathway, a critical component of the acquired immune response to infection. This novel, proprietary drug is designed to prevent complement-mediated inflammation and endothelial damage while leaving intact the respective functions of the other pathways of innate immunity. The EC also granted narsoplimab designation as an orphan medicinal product for treatment in hematopoietic stem cell transplantation. According to the clinical trial website, the drug is in Phase III studies, but the status is unknown.

Thrombotic Thrombocytopenic Purpura (TTP) Market Outlook


Thrombotic thrombocytopenic purpura (TTP) is a rare blood disorder. TTP is a fatal disease that can cause lasting damage, such as brain damage or a stroke, if not treated. TTP arises abruptly and can stay for days or weeks but can also last for months. Relapses can occur in up to 20-30% of people who have aTTP. Relapses also occur in most people who have inherited TTP. Almost all TTP patients are initiated with plasma treatments. Other strategies include pharmacological approaches and surgery.

In 2019, caplacizumab came into the US market and became the only FDA-approved medication for TTP. The only drawback here is that it prevents vWF platelet interaction and transformation and does not target the underlying pathophysiology of the disease, i.e., it does not address the ADAMTS13 inhibitor or its production/elimination. The disease demands immune system modulation so that patients do not continue to produce the antibody against ADAMTS13. Thus, immunosuppression comes into role. Caplacizumab simply blocks the downstream effects of platelet consumption and microthrombus production. Due to the high cost: benefit ratio of Cablivi, it is not so widely used in general practice.

Hence, along with TPE and Cablivi, immunosuppressive therapy is a cornerstone of acute iTTP management. The therapy generally aims to target the production of antibodies to restore circulating levels of ADAMTS13. Thus, TPE and immunosuppressive therapies are used simultaneously. Glucocorticosteroids are widely used. No clinical trials have proved the superiority of corticosteroids plus TPE vs. TPE alone, yet there is high biological plausibility for concurrent immunosuppression given the autoimmune nature of the condition.

The inclusion of Cablivi to standard treatment of aTTP did not make any positive impact due to its high cost and failure to improve relapse rates. The treatment of the underlying pathophysiology of TTP is an essential part of TTP treatment that is still absent. Although the TTP pipeline seems inadequate, one emerging therapy by Takeda, TAK-755 (BAX930/SHP655), a human recombinant ADAMTS13, the plasma metalloprotease that regulates the von Willebrand Factor (VWF) multimers, which is being evaluated for both iTTP and cTTP shows great potential. We are hopeful that if such novel therapies get approved, the treatment paradigm of TTP might witness a positive shift during the forecast period (2021-2030).

Key Findings

  • The market size for thrombotic thrombocytopenic purpura was USD 316 million in 2020 in the US.
  • In 2020, the total market of thrombotic thrombocytopenic purpura was USD 316 million which included a market of USD 286 million occupied by acquired thrombotic thrombocytopenic purpura and USD 31 million occupied by congenital thrombotic thrombocytopenic purpura.
  • According to the publisher's analysis, among the current market size of cTTP (USD 31 million), prophylactic therapies captured the highest market (USD 25 million) and the remaining market was covered by on-demand therapies (USD 6 million) in 2020.
  • TAK-755 is the only emerging therapy that is expected to get launched in the US in 2023 for cTTP and 2025 for aTTP. This molecule is expected to generate nearly USD 40 million for cTTP and USD 59 million for aTTP in its launch year.

The United States Market Outlook


This section provides the total Thrombotic Thrombocytopenic Purpura (TTP) market size and; market size by therapies in the United States.

Thrombotic Thrombocytopenic Purpura (TTP) Drugs Uptake


This section focuses on the rate of uptake of the drugs expected to get launched in the market during the study period 2018-2030. The analysis covers Thrombotic Thrombocytopenic Purpura (TTP) market uptake by drugs; patient uptake by therapies; and sales of each drug.

This helps in understanding the drugs with the most rapid uptake, reasons behind the maximal use of new drugs, and allows the comparison of the drugs based on market share and size which again will be useful in investigating factors important in market uptake and in making financial and regulatory decisions.

Thrombotic Thrombocytopenic Purpura (TTP) Development Activities


The report provides insights into different therapeutic candidates in phase II, and phase III stage. It also analyzes key players involved in developing targeted therapeutics.

Pipeline Development Activities


The report covers the detailed information of collaborations, acquisition, and merger, licensing, and patent details for Thrombotic Thrombocytopenic Purpura (TTP) emerging therapies.

Reimbursement Scenario in Thrombotic Thrombocytopenic Purpura (TTP)


Approaching reimbursement proactively can have a positive impact both during the late stages of product development and well after product launch. In the report, we consider reimbursement to identify economically attractive indications and market opportunities. When working with finite resources, the ability to select the markets with the fewest reimbursement barriers can be a critical business and price strategy.

Competitive Intelligence Analysis


The publisher performs competitive and market Intelligence analysis of the Thrombotic Thrombocytopenic Purpura (TTP) market by using various competitive intelligence tools that include-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 thrombotic thrombocytopenic purpura (TTP), explaining its signs and symptoms, pathophysiology, and currently available therapies.
  • Comprehensive insight has been provided into the thrombotic thrombocytopenic purpura (TTP) epidemiology and treatment.
  • Additionally, an all-inclusive account of both the current and emerging therapies for thrombotic thrombocytopenic purpura (TTP) is provided, along with the assessment of new therapies, which will have an impact on the current treatment landscape.
  • A detailed review of the thrombotic thrombocytopenic purpura (TTP) market; historical and forecasted is included in the report, covering the US drug outreach.
  • The report provides an edge while developing business strategies, by understanding trends shaping and driving the US thrombotic thrombocytopenic purpura (TTP) market.

Report Highlights

  • The increasing prevalence of the disease along with promising emerging pipeline therapies will positively drive the thrombotic thrombocytopenic purpura (TTP) market.
  • The companies and academics are working to assess challenges and seek opportunities that could influence thrombotic thrombocytopenic purpura (TTP) R&D.
  • Major players are involved in developing therapies for thrombotic thrombocytopenic purpura (TTP). The launch of emerging therapies will significantly impact the thrombotic thrombocytopenic purpura (TTP) market.
  • The in-depth analysis of the pipeline assets across different stages of development (phase III and phase II), different emerging trends, and comparative analysis of pipeline products with detailed clinical profiles, key cross-competition, 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.

Thrombotic Thrombocytopenic Purpura (TTP) Report Insights

  • Patient Population
  • Therapeutic Approaches
  • Thrombotic Thrombocytopenic Purpura (TTP) Pipeline Analysis
  • Thrombotic Thrombocytopenic Purpura (TTP) Market Size and Trends
  • Market Opportunities
  • Impact of Upcoming Therapies

Thrombotic Thrombocytopenic Purpura (TTP) Report Key Strengths

  • 10 Years Forecast
  • US Coverage

 Thrombotic Thrombocytopenic Purpura (TTP) Epidemiology Segmentation

  • Key Cross Competition
  • Highly Analyzed Market
  • Drugs Uptake

Thrombotic Thrombocytopenic Purpura (TTP) Report Assessment

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

Key Questions Answered


Market Insights:


  • What was the thrombotic thrombocytopenic purpura (TTP) market share (%) distribution in 2018 and how it would look like in 2030?
  • What would be the thrombotic thrombocytopenic purpura (TTP) total market size as well as market size by therapies across the US during the forecast period (2021-2030)?
  • What are the key findings pertaining to the market across the US?
  • At what CAGR, the thrombotic thrombocytopenic purpura (TTP) market is expected to grow at the US level during the forecast period (2021-2030)?
  • What would be the thrombotic thrombocytopenic purpura (TTP) market outlook across the US during the forecast period (2021-2030)?
  • What would be the thrombotic thrombocytopenic purpura (TTP) 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 subsequent analysis of the associated trends?

Epidemiology Insights:


  • What are the disease risk, burden, and unmet needs of thrombotic thrombocytopenic purpura (TTP)?
  • What is the historical thrombotic thrombocytopenic purpura (TTP) patient population in the United States?
  • What would be the forecasted patient population of thrombotic thrombocytopenic purpura (TTP) at the US level?
  • What will be the growth opportunities across the US with respect to the patient population pertaining to thrombotic thrombocytopenic purpura (TTP)?
  • At what CAGR the population is expected to grow across the US during the forecast period (2021-2030)?

Current Treatment Scenario, Marketed Drugs, and Emerging Therapies:


  • What are the current options for the treatment of thrombotic thrombocytopenic purpura (TTP) along with the approved therapy?
  • What are the thrombotic thrombocytopenic purpura (TTP) emerging drugs and their MOA, product development activities, advantages, disadvantages, safety, and efficacy, etc.?
  • How many companies are developing therapies for the treatment of thrombotic thrombocytopenic purpura (TTP)?
  • How many emerging therapies are in the mid-stage and late stages of development for the treatment of thrombotic thrombocytopenic purpura (TTP)?
  • What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the thrombotic thrombocytopenic purpura (TTP) 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 thrombotic thrombocytopenic purpura (TTP) and their status?
  • What are the key designations that have been granted for the emerging therapies for thrombotic thrombocytopenic purpura (TTP)?
  • What are the US historical and forecasted market of thrombotic thrombocytopenic purpura (TTP)?

Reasons to Buy

  • The report will help in developing business strategies by understanding trends shaping and driving the thrombotic thrombocytopenic purpura (TTP).
  • To understand the future market competition in the thrombotic thrombocytopenic purpura (TTP) market and an insightful review of the key market drivers and barriers.
  • Organize sales and marketing efforts by identifying the best opportunities for thrombotic thrombocytopenic purpura (TTP) in the US.
  • Identification of strong upcoming players in the 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 the thrombotic thrombocytopenic purpura (TTP) market.
  • To understand the future market competition in the thrombotic thrombocytopenic purpura (TTP) market.
Note: Product cover images may vary from those shown

FEATURED COMPANIES

  • Baxalta/Takeda
  • Octapharma
  • Omeros Corporation
  • Sanofi
1 Key Insights

2 Report Introduction

3 Thrombotic Thrombocytopenic Purpura (TTP) Market Overview at a Glance
3.1. Market Share (%) Distribution of Acquired TTP by Therapies in 2018
3.2. Market Share (%) Distribution of Congenital TTP by Therapies in 2030

4 Thrombotic Thrombocytopenic Purpura Market: Future Perspective

5 Executive Summary of Thrombotic Thrombocytopenic Purpura (TTP)

6 Key Events

7 Disease Background and Overview
7.1. Introduction
7.2. Etiology of Thrombotic Thrombocytopenic Purpura
7.3. Signs and Symptoms
7.4. Types of Thrombotic Thrombocytopenic Purpura
7.5. Pathophysiology of Thrombotic Thrombocytopenic Purpura
7.6. Genetic Background and Environmental Factors Related to TTP
7.7. Biomarkers Associated With Thrombotic Thrombocytopenic Purpura
7.8. Diagnosis
7.8.1. International Society of Thrombosis and Hemostasis (ISTH) Guidelines for the Diagnosis of Thrombotic Thrombocytopenic Purpura

8 Treatment and Management
8.1. Line of Therapies
8.2. Treatment Algorithm
8.3. ISTH Guidelines for the Treatment of Thrombotic Thrombocytopenic Purpura (TTP)

9 Epidemiology and Patient Population
9.1. Key Findings
9.2. Methodology of Epidemiology
9.3. Epidemiology Assumptions
9.4. The United States
9.4.1. Total Diagnosed Prevalent Cases of Thrombotic Thrombocytopenic Purpura (TTP) in the United States
9.4.2. Type-specific Diagnosed Prevalent Cases of TTP in the United States
9.4.3. Gender-specific Diagnosed Prevalent Cases of Acquired TTP in the United States
9.4.4. Gender-specific Diagnosed Prevalent Cases of Congenital TTP in the United States
9.4.5. Age-specific Diagnosed Prevalent Cases of Acquired TTP in the United States
9.4.6. Age-specific Diagnosed Prevalent Cases of Congenital TTP in the United States

10 Patient Journey

11 Marketed Drugs
11.1. Cablivi (Caplacizumab/ALX-0081): Sanofi (Ablynx)
11.1.1. Drug Description
11.1.2. Regulatory Milestones
11.1.3. Clinical Development
11.1.4. Clinical Trial Information
11.1.5. Safety and Efficacy
11.1.6. Product Profile
11.2. Octaplas/octaplasLG: Octapharma
11.2.1. Drug Description
11.2.2. Regulatory Milestones
11.2.3. Regulatory Milestones
11.2.4. Safety and Efficacy
11.2.5. Product Profile

12 Emerging Drugs
12.1. Key Cross Competition
12.2. TAK-755 (BAX930/SHP655): Baxalta/Takeda
12.2.1. Product Description
12.2.2. Other Developmental Activities
12.2.3. Clinical Development
12.2.4. Clinical Trials Information
12.2.5. Safety and Efficacy
12.2.6. Product Profile
12.3. Narsoplimab (OMS721): Omeros Corporation
12.3.1. Product Description
12.3.2. Other Developmental Activities
12.3.3. Clinical Development
12.3.4. Clinical Trials Information
12.3.5. Safety and Efficacy
12.3.6. Product Profile

13 Thrombotic Thrombocytopenic Purpura (TTP): United States Market Analysis
13.1. Key Findings
13.2. Methodology of TTP Market
13.3. Emerging Drug Analysis
13.4. Key Market Forecast Assumptions
13.5. Market Outlook
13.6. Market Size of TTP in the United States
13.6.1. Total Market Size of TTP
13.6.2. Market Size of TTP by Therapies
13.6.2.1. Market Size of Acquired TTP by Therapies in the United States
13.6.2.2. Market Size of Congenital TTP by Therapies in the United States

14 KOL Views

15 Market Drivers

16 Market Barriers

17 SWOT Analysis

18 Unmet Needs

19 Reimbursement and Market Access

20 Appendix
20.1. Bibliography
20.2. Report Methodology

21 Publisher Capabilities

22 Disclaimer

23 About the Publisher
Note: Product cover images may vary from those shown

A selection of companies mentioned in this report includes:

  • Sanofi
  • Octapharma
  • Baxalta/Takeda
  • Omeros Corporation
Note: Product cover images may vary from those shown