血栓性血小板減少性紫斑病 (TTP) - 市場考察、疫学、市場予測：2030年
Thrombotic Thrombocytopenic Purpura (TTP) Market Insight, Epidemiology and Market Forecast -2030
|血栓性血小板減少性紫斑病 (TTP) - 市場考察、疫学、市場予測：2030年|
発行: DelveInsight Business Research LLP
ページ情報: 英文 110 Pages
2020年に米国で血栓性血小板減少性紫斑病 (TTP) と診断された有病者数は6,002件でした。米国における血栓性血小板減少性紫斑病 (TTP) の市場規模は、2020年に3億1,600万米ドルとなりました。
当レポートでは、血栓性血小板減少性紫斑病 (TTP) について調査分析し、過去・将来の疫学、米国の市場動向に焦点を当てて、現在の治療法、新薬、市場規模などについて、体系的な情報を提供しています。
DelveInsight's '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.
Study Period: 2018-2030
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.
Continued in the report…..
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.
The epidemiology segment also provides the Thrombotic Thrombocytopenic Purpura (TTP) epidemiology data and findings across the United States.
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.
Products detail in the report…
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).
Products detail in the report…
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]).
Products detail in the report…
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.
Products detail in the report…
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).
This section provides the total Thrombotic Thrombocytopenic Purpura (TTP) market size and; market size by therapies in the United States.
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
We perform 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.
Current Treatment Scenario, Marketed Drugs, and Emerging Therapies: