Published Date : 2022-09-07
Published Date : 2022-09-07
Updated On : 2023-09-15
Pages : 151
Thelansis’s “Thrombotic Thrombocytopenic Purpura (TTP) Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2022 To 2032" covers disease overview, epidemiology, drug utilization, prescription share analysis, competitive landscape, clinical practice, regulatory landscape, patient share, market uptake, market forecast, and key market insights under the potential Thrombotic Thrombocytopenic Purpura treatment modalities options for eight major markets (USA, Germany, France, Italy, Spain, UK, Japan, and China).
Thrombotic thrombocytopenic purpura (TTP) is a form of microangiopathic hemolytic anemia characterized by the classic pentad of symptoms: fever, thrombocytopenia, hemolytic anemia, renal dysfunction, and neurologic dysfunction. TTP arises from a deficiency or absence of ADAMTS13 (a disintegrin and metalloproteinase with a thrombospondin type 1 motif member 13), either congenital or acquired. Reduced levels of ADAMTS13 lead to the formation of microthrombi, resulting in ischemic damage to various organs. ADAMTS13 cannot effectively inhibit the large multimer von Willebrand factor (VWF), crucial for preventing spontaneous coagulation. Differential diagnoses encompass other thrombotic microangiopathies (TMAs), such as hemolytic uremic syndrome and TMA, secondary to factors like transplantation, chemotherapy, drugs, HIV/AIDS, and HELLP syndrome. Other conditions include catastrophic antiphospholipid syndrome, severe sepsis, vitamin B12 deficiency, malignant hypertension, and disseminated intravascular coagulopathy. Due to the severity of TTP and the risk of sudden organ failure, it is advisable to manage patients in intensive care units until their platelet count returns to normal. The current standard treatment regimen involves plasma exchange to replenish active ADAMTS13 from donor plasma (and, to a lesser extent, remove anti-ADAMTS13 antibodies and pro-aggregate substances). Immunosuppressive therapies like glucocorticoids and rituximab are administered to prevent the production of ADAMTS13 antibodies, and caplacizumab, a nanobody targeting von Willebrand factor (VWF), is employed to expedite platelet count recovery and prevent adverse outcomes. Without intervention, the mortality rate for thrombotic thrombocytopenic purpura is 90%. However, early treatment involving plasma exchange and corticosteroids reduces mortality by 15%. The longer a patient delays seeking treatment, the greater the likelihood of experiencing unfavorable outcomes.
North America- the United States and Canada
Europe- EU5 (Germany, France, Italy, Spain, and the United Kingdom)
Other countries- Japan & China
This section of the study covers country-specific current clinical practice, the standard of care, and significant limitations around addressing the unmet needs. Retrospective analysis and bench-marking of clinical study outcomes are presented in terms of Pre-treatment & post-treatment clinical and demographic patient characteristics. Essentially, this section will cover the evolution of the current competitive landscape and its impact on the future treatment paradigm.
KOLs across 8 MM markets from the center of Excellence/ Public/ Private hospitals participated in the study. Insights around current treatment landscape, epidemiology, clinical characteristics, future treatment paradigm, and Unmet needs
- Data Inputs with sourcing
- Market Event and Product Event
- Country-specific Forecast Model
- Market uptake and patient share uptake
- Attribute Analysis
- Analog Analysis
- Disease burden and pricing scenario
- Summary and Insights
Optimization of cash flow/ revenue flow concerning all fixed and variable investments throughout the product development process. The rate of return on an investment is a critical indicator to ensure the profitability and break-even of the project.
The competitive landscape includes country-specific approved as well as pipeline therapies. Any asset/product-specific designation or review such as Orphan drug designation, Fast track, Priority Review, Breakthrough Therapy Designation, Rare Pediatric Disease Designation, and Accelerated Approval are tracked and supplemented with analyst commentary.
Detailed clinical trial data analysis and critical product positioning include trial design, primary outcomes, secondary outcomes, dosing and schedules, inclusion and exclusion criteria, recruitment status and essentially covers the reported adverse events. Majorly the trial analysis helps determine the potential of the critical assets and their probable filing and launch date.
This report presents the most important clinical unmet needs in the treatment, according to Thelansis research and analysis. Other essential unmet needs identified through our study include decreased cost burden on patients, improved administration convenience, and improved patient compliance.
S. no | Asset | Company | Stage |
1 | GC1126A | GC Biopharma | Phase 3 |
2 | BAX930 | Shire | Phase 3 |
3 | Sym001 | Symphogen A/S | Phase 3 |
4 | Romiplostim | Amgen | Phase 3 |
5 | R788 | Kissei Pharmaceutical Co., Ltd. | Phase 3 |
6 | Hetrombopag Olamine | Jiangsu HengRui Medicine Co., Ltd. | Phase 3 |
7 | Fostamatinib disodium | Rigel Pharmaceuticals | Phase 3 |
8 | PG2 | PhytoHealth Corporation | Phase 2 |
9 | GNR-069 | AO GENERIUM | Phase 3 |
10 | AMG 531 | Kyowa Kirin Co., Ltd. | Phase 3 |
Continued...
KOLs across 8 MM market from the center of Excellence/ Public/ Private hospitals participated in the study. Insights around current treatment landscape, epidemiology, clinical characteristics, future treatment paradigm, and Unmet needs.
COUNTRY | No. Of KOLs |
USA | 17 |
GERMANY | 4 |
UK | 4 |
SPAIN | 3 |
FRANCE | 2 |
ITALY | 3 |
JAPAN | 3 |
CHINA | 4 |
Data Inputs with sourcing, Market Event, Product Event, Country specific Forecast Model, Market uptake and patient share uptake, Attribute Analysis, Analog Analysis, Disease burden, and pricing scenario, Summary, and Insights.
1. Thrombotic Thrombocytopenic Purpura (TTP) – Key Findings Summary |
1.1. Clinical findings |
1.1.1. Disease overview |
1.1.2. Therapeutic practices |
1.1.3. Future outlook |
1.2. Commercial findings |
1.2.1. Thrombotic Thrombocytopenic Purpura (TTP) market scenario 2022 |
1.2.2. Thrombotic Thrombocytopenic Purpura (TTP) market scenario 2025 |
1.2.3. Thrombotic Thrombocytopenic Purpura (TTP) market scenario 2032 |
2. Thrombotic Thrombocytopenic Purpura (TTP) Overview |
2.1. Disease Introduction |
2.2. Pathophysiology |
2.3. Signs and Symptoms |
2.4. Risk Factors |
2.5. Etiology |
2.6. Classification |
2.7. Pathogenesis |
2.8. Diagnosis |
2.9. Complications |
2.10. Treatment Algorithm |
2.10.1. Treatment in US (guidelines) |
2.10.2. Treatment in EU-5 (guidelines) |
2.10.3. Treatment in Japan (guidelines) |
2.10.4. Treatment in China (guidelines) |
2.11. Treatment Goals for Thrombotic Thrombocytopenic Purpura (TTP) |
2.12. Referral Patterns |
2.12.1. Referral Scenario in US |
2.12.2. Referral Scenario in EU-5 |
2.12.3. Referral Scenario in Japan |
2.12.4. Referral Scenario in China |
2.13. Thrombotic Thrombocytopenic Purpura (TTP) Prognosis |
2.14. Healthcare burden |
2.14.1. Healthcare burden in US |
2.14.2. Healthcare burden in EU-5 |
2.14.3. Healthcare burden in Japan |
2.14.4. Healthcare burden in China |
2.15. Unmet Needs in Thrombotic Thrombocytopenic Purpura (TTP) management |
2.16. Market Opportunity for Thrombotic Thrombocytopenic Purpura (TTP) |
2.17. KOL Comments on current and upcoming/expected treatment practices in Thrombotic Thrombocytopenic Purpura (TTP) |
3. Epidemiology |
3.1. Epidemiology Overview |
3.2. Epidemiology by Geography |
3.2.1. Thrombotic Thrombocytopenic Purpura (TTP) Epidemiology in US (2022-2032) |
3.2.1.1. Incidence of Thrombotic Thrombocytopenic Purpura (TTP) |
3.2.1.2. Diagnosed cases |
3.2.1.3. Treatable Patient Pool |
3.2.1.4. Epidemiology Trends |
3.2.2. Thrombotic Thrombocytopenic Purpura (TTP) Epidemiology in EU-5 (2022-2032) |
3.2.2.1. Incidence of Thrombotic Thrombocytopenic Purpura (TTP) |
3.2.2.2. Diagnosed cases |
3.2.2.3. Treatable Patient Pool |
3.2.2.4. Epidemiology Trends |
3.2.3. Thrombotic Thrombocytopenic Purpura (TTP) Epidemiology in Japan (2022-2032) |
3.2.3.1. Incidence of Thrombotic Thrombocytopenic Purpura (TTP) |
3.2.3.2. Diagnosed cases |
3.2.3.3. Treatable Patient Pool |
3.2.3.4. Epidemiology Trends |
3.2.4. Thrombotic Thrombocytopenic Purpura (TTP) Epidemiology in China (2022-2032) |
3.2.4.1. Incidence of Thrombotic Thrombocytopenic Purpura (TTP) |
3.2.4.2. Diagnosed cases |
3.2.4.3. Treatable Patient Pool |
3.2.4.4. Epidemiology Trends |
3.3. Epidemiology Trends (World-wide) |
4. Market Outlook |
4.1. US Thrombotic Thrombocytopenic Purpura (TTP) Market Forecast 2022-2032 |
4.1.1. Market Progression (Futuristic) |
4.1.2. Market Trends and Expectations |
4.1.2.1. Worst case scenario |
4.1.2.2. Base Case Scenario |
4.1.2.3. Best Case Scenario |
4.1.3. Drivers and Barriers |
4.2. UK Thrombotic Thrombocytopenic Purpura (TTP) Market Forecast 2022-2032 |
4.2.1. Market Progression (Futuristic) |
4.2.2. Market Trends and Expectations |
4.2.2.1. Worst case scenario |
4.2.2.2. Base Case Scenario |
4.2.2.3. Best Case Scenario |
4.2.3. Drivers and Barriers |
4.3. France Thrombotic Thrombocytopenic Purpura (TTP) Market Forecast 2022-2032 |
4.3.1. Market Progression (Futuristic) |
4.3.2. Market Trends and Expectations |
4.3.2.1. Worst case scenario |
4.3.2.2. Base Case Scenario |
4.3.2.3. Best Case Scenario |
4.3.3. Drivers and Barriers |
4.4. Germany Thrombotic Thrombocytopenic Purpura (TTP) Market Forecast 2022-2032 |
4.4.1. Market Progression (Futuristic) |
4.4.2. Market Trends and Expectations |
4.4.2.1. Worst case scenario |
4.4.2.2. Base Case Scenario |
4.4.2.3. Best Case Scenario |
4.4.3. Drivers and Barriers |
4.5. Italy Thrombotic Thrombocytopenic Purpura (TTP) Market Forecast 2022-2032 |
4.5.1. Market Progression (Futuristic) |
4.5.2. Market Trends and Expectations |
4.5.2.1. Worst case scenario |
4.5.2.2. Base Case Scenario |
4.5.2.3. Best Case Scenario |
4.5.3. Drivers and Barriers |
4.6. Spain Thrombotic Thrombocytopenic Purpura (TTP) Market Forecast 2022-2032 |
4.6.1. Market Progression (Futuristic) |
4.6.2. Market Trends and Expectations |
4.6.2.1. Worst case scenario |
4.6.2.2. Base Case Scenario |
4.6.2.3. Best Case Scenario |
4.6.3. Drivers and Barriers |
4.7. Japan Thrombotic Thrombocytopenic Purpura (TTP) Market Forecast 2022-2032 |
4.7.1. Market Progression (Futuristic) |
4.7.2. Market Trends and Expectations |
4.7.2.1. Worst case scenario |
4.7.2.2. Base Case Scenario |
4.7.2.3. Best Case Scenario |
4.7.3. Drivers and Barriers |
4.8. China Thrombotic Thrombocytopenic Purpura (TTP) Market Forecast 2022-2032 |
4.8.1. Market Progression (Futuristic) |
4.8.2. Market Trends and Expectations |
4.8.2.1. Worst case scenario |
4.8.2.2. Base Case Scenario |
4.8.2.3. Best Case Scenario |
4.8.3. Drivers and Barriers |
4.9. Key Expected Milestones (world-wide) Impacting the Market |
5. Competitive Landscape |
5.1. Pipeline Therapies Overview |
5.1.1. Phase III Therapies |
5.1.1.1. Current Status |
5.1.1.2. Trial details, results |
5.1.1.3. Approval Timeline |
5.1.1.4. Likelihood of approval |
5.1.1.5. Expected Product Positioning |
5.1.1.2. All other Phase III Therapies ….. |
5.1.1.3. Attribute Analysis of Phase III molecules |
5.1.2. Phase II and Phase I/II Therapies |
5.1.2.1. Current Status |
5.1.2.2. Trial details, results |
5.1.2.3. Approval Timelines |
5.1.3. List of active Pre-clinical Therapies |
5.1.3.1. Status in Thrombotic Thrombocytopenic Purpura (TTP) |
5.1.3.2. Company positioning |
5.1.3.2. All other pre-clinical therapies |
5.1.4. List of Inactive/discontinued assets |
5.1.4.1. Business impact of discontinuations on current pipeline |
5.1.5. Potential winners from Thrombotic Thrombocytopenic Purpura (TTP) Pipeline |
5.1.5.1. Potential Blockbusters across the pipeline |
6. Regulatory/Approval Scenario |
6.1. Regulatory/Approval Framework in US |
6.1.1. Policy Framework |
6.1.2. Payer Expectations |
6.2. Regulatory/Approval Framework in UK |
6.2.1. Policy Framework |
6.2.2. Payer Expectations |
6.3. Regulatory/Approval Framework in France |
6.3.1. Policy Framework |
6.3.2. Payer Expectations |
6.4. Regulatory/Approval Framework in Germany |
6.4.1. Policy Framework |
6.4.2. Payer Expectations |
6.5. Regulatory/Approval Framework in Italy |
6.5.1. Policy Framework |
6.5.2. Payer Expectations |
6.6. Regulatory/Approval Framework in Spain |
6.6.1. Policy Framework |
6.6.2. Payer Expectations |
6.7. Regulatory/Approval Framework in Japan |
6.7.1. Policy Framework |
6.7.2. Payer Expectations |
6.8. Regulatory/Approval Framework in China |
6.8.1. Policy Framework |
6.8.2. Payer Expectations |
7. Clinical Trial Assessment – Current and Future Paradigm |
7.1. Distribution of Primary Endpoints across trials |
7.2. Distribution of Secondary Endpoints across trials |
7.3. Evolution and acceptance of surrogate endpoints |
7.4. Key Investigator initiated trials |
7.5. Attrition analysis |
7.5.1. Suspended/Discontinued Assets |
7.5.2. Failed Trials, Reasons and Business Impact |
7.5.3. Terminated Trials, Reasons and Business Impact |
7.5.4. Withdrawn Trials, Reasons and Business Impact |
7.6. Trial enrollment scenario and challenges |
7.7. Clinical Trial Guidance (across geographies) |
8. Thelansis Commentary |
8.1. Key Unmet needs in Thrombotic Thrombocytopenic Purpura (TTP) |
8.2. Possible Best-case Clinical Trial Strategies |
8.3. Possible Best Case Targeted Product Profile (TPP) |
8.4. Possible Best-case Market positioning strategies |
8.5. Possible Best-case Market Access Strategies |
8.6. Possible Best-case LCM Strategies |
8.7. Overall View on Thrombotic Thrombocytopenic Purpura (TTP) Market in Dollar Value |
9. Report Methodology |
9.1. Secondary research |
9.2. Primary research |
9.3. Data collation |
9.4. Insight Generation |
10. About Thelansis |
10.1. Our Capabilities |
10.2. Our Services |
10.3. Our Contacts |
10.4. Disclaimer |