B型血友病市场:KOL的洞察

本报告提供全球B型血友病市场相关调查，提供市场概要，以及已上市治疗方法，开发平台趋势，未来预测等资讯。

目录

摘要整理

B型血友病的现在及未来的治疗流程

调查目的

重组第九凝血因子(FIX)补充疗法

• 已上市治疗方法

基因治疗

• 已上市治疗方法
  • Hemgenix（etranacogene dezaparvovec；CSL Behring/uniQure）
• 开发中产品
  • Fidanacogene elaparvovec（辉瑞/Spark Therapeutics）

Non係数再调整代理商

• 开发中产品
• 抗TFPI单株抗体
  • Concizumab（诺和诺德）
  • Marstacimab（辉瑞）
• 抗凝血酵素III抑制剂/RNA干扰
  • Fitusiran（Alnylam 製药公司/赛诺菲）
• 活性化蛋白质C受体调製器
  • SerpinPC（Centessa 製药公司）

B型血友病治疗的未来趋势与机会

• 重要的洞察概要

附录

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)