B型血友病市场:KOL的洞察

本报告对B 型血友病治疗领域进行了深入分析，重点关注了标准产品和延长半衰期产品的不断发展的使用，重点意见领袖的见解强调了基因疗法之间的竞争动态，其中辉瑞的Beqvez/Durveqtix 面临课题。虽然 Hemgenix 显示出了希望，但其采用却受到财务和后勤障碍的阻碍，机构对美国的金融风险持谨慎态度，而定价谈判则减缓了其在欧洲的推出。

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

现在及未来的治疗流程

调查目的

重组的因素IX(FIX)补充疗法

• 核准的药物
  • 标准半衰期 (SHL) 产物：BeneFIX（nonacog alfa；辉瑞）；Ixinity（trenacog alfa；Medexus Pharma）和 Rixubis（nonacog gamma；武田）
  • 延长半衰期 (EHL) 产品：Alprolix（eftrenacog alfa；Sanofi/Sobi）；Idelvion（albutrepenonacog alfa；CSL Behring）和 Rebinyn/Refixia（nonacog beta pegol；诺和诺德）
  • 旁路剂：NovoSeven（eptacog alfa；Novo Nordisk）；FEIBA（因子八抑制旁路剂；Takeda）和 Sevenfact/Cevenfacta（凝血因子 VIIa [重组]-jncw；LFB Biotechnologies/HEMA Biologics）

基因治疗

• 已通过核准药物
  • Hemgenix（etranacogene dezaparvovec；uniQure/CSL Behring）
  • Beqvez/Durveqtix（fidanacogene elaparvovec；辉瑞）

非係数再调整代理商

• 已通过核准药物
  • Hympavzi(marusutashimabu，Pfizer)
• 开发平台(管线)医药品
  • Alhemo（康珠单抗；诺和诺德）
  • Fitusiran（Alnylam 製药公司/赛诺菲）
  • SerpinPC（Centessa 製药公司）

今后的治疗趋势

附录

KOL快报

This report provides an in-depth analysis of the haemophilia B treatment landscape, focusing on the evolving use of standard and extended half-life products. Insights from key opinion leaders highlight the competitive dynamics between gene therapies, with Pfizer's Beqvez/Durveqtix facing challenges against Hemgenix due to corticosteroid use and antibody issues. Sanofi/Sobi's Alprolix emerges as the preferred extended half-life product, favoured for its early market entry and superior extravascular distribution. Additionally, while Hemgenix shows promise, its adoption is hindered by financial and logistical barriers, with institutions cautious about the financial risks in the U.S. and pricing negotiations slowing its rollout in Europe.

Key questions answered:

• How do experts use and prefer standard half-life (SHL) and extended half-life (EHL) products?
• What changes are expected in the use of SHL and EHL products over the next 3-5 years?
• What are the expert views on bypassing agents and their future role?
• How is CSL Behring/uniQure's Hemgenix perceived post-approval?
• What are the anticipated barriers to the uptake of gene therapies?
• How are non-factor rebalancing therapies, like Alhemo and Hympavzi, viewed?
• Are there safety concerns associated with non-factor therapies?
• How will the haemophilia B treatment landscape evolve in the coming years?

Key brands covered in this report:

Companies:

Table of Contents

Executive summary

Current and future treatment algorithm

Research objectives

Recombinant Factor IX (FIX) replacement therapies

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

Gene therapy

• Approved drugs
  • Hemgenix (etranacogene dezaparvovec; uniQure/CSL Behring)
  • Beqvez/Durveqtix (fidanacogene elaparvovec; Pfizer)

Non-factor rebalancing agents

• Approved drugs
  • Hympavzi (marstacimab; Pfizer)
• Pipeline drugs
  • Alhemo (concizumab; Novo Nordisk)
  • Fitusiran (Alnylam Pharmaceuticals/Sanofi)
  • SerpinPC (Centessa Pharmaceuticals)

Future treatment trends

• Key insights summary

Appendix

• KOL details
  • KOLs from North America
  • KOLs from Europe

KOL Bulletins