Congenital Thrombotic Thrombocytopenic Purpura (cTTP) – Market Outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2022 To 2032

Congenital Thrombotic Thrombocytopenic Purpura (cTTP) Market Outlook

Thelansis’s “Congenital Thrombotic Thrombocytopenic Purpura (cTTP) 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 Congenital thrombotic thrombocytopenic purpura treatment modalities options for eight major markets (USA, Germany, France, Italy, Spain, UK, Japan, and China).

Congenital Thrombotic Thrombocytopenic Purpura (cTTP) Overview

Congenital thrombotic thrombocytopenic purpura (cTTP) is an inherited condition characterized by severe reduction of platelets in the bloodstream, destruction of red blood cells, and varying degrees of organ failure. While most individuals with this condition experience symptoms in infancy or childhood, the severity and specific manifestations can differ significantly. Some individuals may only have mild symptoms of low platelet count throughout childhood. In contrast, others may develop significant jaundice and experience episodes of low platelet count and red blood cell destruction soon after birth. In some cases, the initial onset of TTP occurs during adulthood, particularly in women, and is triggered by pregnancy. Infections and vaccinations can also act as precipitating factors. Congenital TTP is caused by a mutation in the ADAMTS13 gene located on chromosome 9q34. This gene provides instructions for producing ADAMTS13, an enzyme breaking down excessively large von Willebrand factor molecules in the blood. Approximately 50% of individuals with congenital TTP develop a chronic and frequently recurring condition. Recurrent episodes typically characterize congenital TTP over a longer period compared to the immune-mediated form of the disease (iTTP). Both forms of TTP can lead to various complications, including neurological issues, kidney problems, heart dysfunction, and gastrointestinal symptoms, all resulting from the widespread formation of small blood clots in the body's blood vessels. TTP is a rapidly fatal disease without treatment, with a mortality rate exceeding 90%. However, introducing therapeutic plasma exchange and infusion has significantly decreased the mortality rate to around 15%. Plasma therapy is also effective in preventing long-term organ complications resulting from recurrent episodes of TTP.

• The annual incidence in the USA is 2.99 cases per million annually.

Geography Covered:

North America- the United States and Canada

Europe- EU5 (Germany, France, Italy, Spain, and the United Kingdom)

Other countries- Japan & China

Study Period: 2022-2032

Current Clinical Practice and Treatment Algorithm

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.

KOL Insights:

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

Market Forecast: Patient Based Forecast Model (MS. Excel Based Automated Dashboard)

- 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

NPV/ IRR Calculator-

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.

Competitive Landscape:

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.

Clinical Trial Assessment-

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.

Unmet Medical Needs Overview-

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.

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