(Albany, USA) DelveInsight’s “Thrombocytopenia Market Insights, Epidemiology and Market Forecast – 2034” report delivers an in-depth understanding of thrombocytopenia, historical and forecasted epidemiology as well as the thrombocytopenia market trends in the United States, EU4 (Germany, Spain, Italy, and France) and the United Kingdom, and Japan.
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Key Takeaways from the Thrombocytopenia Market Report
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Thrombocytopenia Epidemiology Segmentation in the 7MM
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Thrombocytopenia Marketed Drugs
• ADZYNMA: Takeda
ADZYNMA (ADAMTS13, recombinant-krhn) is a human recombinant (a disintegrin and metalloproteinase with thrombospondin motifs 13) ADAMTS13 (rADAMTS13) indicated for prophylactic or on-demand enzyme replacement therapy (ERT) in adult and pediatric patients with congenital thrombotic thrombocytopenic purpura (cTTP). ADZYNMA was previously granted Orphan Drug Designation (ODD) by the US FDA for the treatment and prevention of TTP, including its acquired idiopathic and secondary forms, as well as Fast Track and Rare Pediatric Disease Designation. The US FDA also granted Takeda a Rare Pediatric Disease Voucher for the approval of ADZYNMA. ADZYNMA has also been granted ODD by the European Medicines Agency (EMA) and Japan’s Ministry of Health, Labour and Welfare (MHLW) for the treatment of TTP.
• TAVALISSE (fostamatinib): Rigel Pharmaceuticals/Kissei Pharmaceutical
Fostamatinib disodium (also known as TAVALISSE; R-985788) is an orally-bioavailable investigational agent being developed by Rigel pharmaceuticals and approved for the treatment of patients suffering from persistent/chronic adult idiopathic thrombocytopenic purpura. The therapeutic candidate inhibits FcR-triggered, Syk-dependent cytoskeletal rearrangement during phagocytosis.
Thrombocytopenia Emerging Drugs
• Rilzabrutinib (PRN-1008): Sanofi/Principia Biopharma
Rilzabrutinib (PRN-1008) is an orally administered reversible covalent inhibitor of Bruton tyrosine kinase (BTK). BTK is an essential signaling element downstream of the B-cell receptor (BCR), Fc-gamma receptor, and Fc-epsilon receptor pathways. Rilzabrutinib is currently being evaluated in the Phase III stage of development to treat ITP in adults and adolescents with persistent or chronic ITP. Recently, the LUNA 3 Phase III study showed that rilzabrutinib at a dose of 400 mg twice daily orally successfully met the primary endpoint of achieving a durable platelet response in adult patients with persistent or chronic immune thrombocytopenia (ITP). The safety profile observed for rilzabrutinib in this study was consistent with previous findings from other studies.
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Thrombocytopenia Market Insights
Thrombocytopenia is a condition in which the blood has a lower than the normal platelet count. When a patient’s blood has too few platelets, mild to serious bleeding can occur. Bleeding can be internal or underneath the skin, or external. The approved therapies for treating thrombocytopenia in the US include MULPLETA, DOPTELET,TAVALISSE, PROMACTA, NPLATE, CABLIVI, GAMMAPLEX, OCTAGAM, RHOPHYLAC, and PRIVIGEN. These therapies are approved for various patient groups; for example, MULPLETA and DOPTELET are approved for thrombocytopenia in chronic liver disease, PROMACTA is approved for chronic immune (idiopathic) thrombocytopenic purpura (ITP).
Thrombocytopenia Treatment Landscape
Treatment for thrombocytopenia depends on its cause and severity. The main goal of treatment is to prevent death and disability caused by bleeding. The approved therapies for treating thrombocytopenia in the US include MULPLETA, DOPTELET, TAVALISSE, PROMACTA, NPLATE, CABLIVI, GAMMAPLEX, OCTAGAM, RHOPHYLAC, and PRIVIGEN. Danaparoid, argatroban, and lepirudin were approved in the US to treat thrombosis in patients with heparin-induced thrombocytopenia. Current treatment options for severe thrombocytopenia in chronic liver disease include platelet transfusion, splenic artery embolization, splenectomy, and placement of a transjugular intrahepatic portosystemic stent shunt (TIPSS).
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Scope of the Thrombocytopenia Market Report
Table of Content
1. Key Insights
2. Report Introduction
3. Thrombocytopenia Market Overview at a Glance
4. Executive Summary of Thrombocytopenia
5. Key events
6. Thrombocytopenia Market Disease Background and Overview
7. Thrombocytopenia Methodology
8. Thrombocytopenia Epidemiology and Patient Population
9. Patient Journey
10. Thrombocytopenia Marketed Products
11. Thrombocytopenia Emerging Therapies
12. Thrombocytopenia Seven Major Market Analysis
13. Market Access and Reimbursement
14. KOL Views
15. Thrombocytopenia Unmet Needs
16. SWOT Analysis
17. Appendix
18. DelveInsight Capabilities
19. Disclaimer
20. About DelveInsight
About DelveInsight
DelveInsight is a leading Healthcare Business Consultant, and Market Research firm focused exclusively on life sciences. It supports Pharma companies by providing comprehensive end-to-end solutions to improve their performance. It also offers Healthcare Consulting Services, which benefits in market analysis to accelerate the business growth and overcome challenges with a practical approach.
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