B型血友病市場:KOL的洞察

本報告提供全球B型血友病市場相關調查，提供市場概要，以及已上市治療方法，開發平台趨勢，未來預測等資訊。

目錄

摘要整理

B型血友病的現在及未來的治療流程

調查目的

重組第九凝血因子(FIX)補充療法

• 已上市治療方法

基因治療

• 已上市治療方法
  • Hemgenix（etranacogene dezaparvovec；CSL Behring/uniQure）
• 開發中產品
  • Fidanacogene elaparvovec（輝瑞/Spark Therapeutics）

Non係數再調整代理商

• 開發中產品
• 抗TFPI單株抗體
  • Concizumab（諾和諾德）
  • Marstacimab（輝瑞）
• 抗凝血酵素III抑制劑/RNA干擾
  • Fitusiran（Alnylam 製藥公司/賽諾菲）
• 活性化蛋白質C受體調製器
  • SerpinPC（Centessa 製藥公司）

B型血友病治療的未來趨勢與機會

• 重要的洞察概要

附錄

Extended half-life products have become the standard of care for haemophilia B patients, so what factors influence KOL prescribing decisions with respect to Sanofi/Sobi's Alprolix, CSL Behring's Idelvion and Novo Nordisk's Rebinyn/Refixia? While experts welcome the approval of CSL Behring/uniQure's gene therapy Hemgenix, will long-term safety concerns and cost limit its uptake? KOLs critically assess the prospects of launched and pipeline therapies.

Table of Contents

Executive summary (12)

Current and future treatment algorithm for haemophilia B

Research objectives (10)

Recombinant Factor IX (FIX) replacement therapies (28)

• Marketed therapies (28)
  • Standard half-life (SHL) products: BeneFIX (nonacog alfa; Pfizer), Ixinity (trenonacog alfa; Medexus Pharma) and Rixubis (nonacog gamma; Takeda) (8)
  • Extended half-life (EHL) products: Alprolix (eftrenonacog alfa; Sanofi/Sobi), Idelvion (albutrepenonacog alfa; CSL Behring) and Rebinyn/Refixia (nonacog beta pegol; Novo Nordisk) (13)
  • Bypassing agents: NovoSeven (eptacog alfa; Novo Nordisk), FEIBA (Factor Eight Inhibitor Bypassing Agent; Takeda), Sevenfact/Cevenfacta (coagulation Factor VIIa [recombinant]-jncw; LFB Biotechnologies/LFB USA) (7)

Gene therapy (21)

• Marketed therapies (13)
  • Hemgenix (etranacogene dezaparvovec; CSL Behring/uniQure) (13)
• Pipeline products (8)
  • Fidanacogene elaparvovec (Pfizer/Spark Therapeutics) (8)

Non-factor rebalancing agents (37)

• Pipeline products
• Anti-TFPI monoclonal antibodies (19)
  • Concizumab (Novo Nordisk) (13)
  • Marstacimab (Pfizer) (6)
• Antithrombin III inhibitors/RNA interference (9)
  • Fitusiran (Alnylam Pharmaceuticals/Sanofi) (9)
• Activated protein C receptor modulators (9)
  • SerpinPC (Centessa Pharmaceuticals) (9)

Future trends and opportunities in haemophilia B treatment (5)

• Key insights summary (5)

Appendix (4)

• KOL details (4)
  • KOLs from North America (1)
  • KOLs from Europe (2)