Transfusion-dependent β-thalassemia (TDT) – Market outlook, Epidemiology, Competitive Landscape and Market Forecast Report – 2020 To 2030


 β-thalassemia are monogenic disorders usually caused by reduced or absent synthesis of the βglobin chain, one of the main components of adult hemoglobin (HbA, α2β2. This results in a relative increase in the unattached αglobin chains (α/β-chain imbalance) that form insoluble Hemi-chromes in the erythrocyte progenitors. The Hemi-chromes damage the erythrocyte membrane, leading to severe intramedullary erythrocyte apoptosis (ineffective erythropoiesis, IE) and severely shortened red blood cell (RBC) life span due to extra-medullary hemolysis, leading to severe anemia.

·       Transfusion-dependent β-thalassemia (TDT) is the most severe form of the disease, characterized by severe anemia, and requires lifelong, regular blood transfusions to maintain Hb levels. These transfusions carry the risk of progressive multi-organ damage due to unavoidable iron overload

·       In the USA the estimated β-thalassemia cases range between 1.5 to 2.6 cases per 100,000 population, however it varies based on geographic presence

 

The competitive landscape of Transfusion-dependent β-thalassemia (TDT) includes country-specific approved as well as pipeline therapies. Any asset/product-specific designation or review and Accelerated Approval are being tracked and supplemented with analyst commentary.

KOLs insights of Transfusion-dependent β-thalassemia (TDT) 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.

Transfusion-dependent β-thalassemia (TDT) Market Forecast: Patient Based Forecast Model (MS. Excel Based Automated Dashboard) which 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.

Read more: Transfusion-dependent β-thalassemia (TDT) – Market outlook, Epidemiology, Market Forecast and Competitive Landscape Report – 2020 To 2030

S. No    Asset               Company                                 Stage

1          ST-400 Sangamo Therapeutics  Phase 2

2          CTX001            CRISPR Therapeutics   Phase 2

3          ET-01   EdiGene (GuangZhou) Inc.        Phase 1

4          VIT-2763          Vifor Pharma     Phase 2

5          OTL-300           Orchard Therapeutics    Phase 2

6          LentiGlobin BB305        bluebird bio       Phase 3

7          Mitapivat           Agios Pharmaceuticals, Inc.       Phase 3

8          PTG-300           Protagonist Therapeutics, Inc.    Phase 2

9          Deferasirox       Novartis Pharmaceuticals          Phase 2

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