B型血友病市場:KOL的洞察

本報告對B 型血友病治療領域進行了深入分析，重點關注了標準產品和延長半衰期產品的不斷發展的使用，重點意見領袖的見解強調了基因療法之間的競爭動態，其中輝瑞的Beqvez/Durveqtix 面臨課題。雖然 Hemgenix 顯示出了希望，但其採用卻受到財務和後勤障礙的阻礙，機構對美國的金融風險持謹慎態度，而定價談判則減緩了其在歐洲的推出。

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

現在及未來的治療流程

調查目的

重組的因素IX(FIX)補充療法

• 核准的藥物
  • 標準半衰期 (SHL) 產物：BeneFIX（nonacog alfa；輝瑞）；Ixinity（trenacog alfa；Medexus Pharma）和 Rixubis（nonacog gamma；武田）
  • 延長半衰期 (EHL) 產品：Alprolix（eftrenacog alfa；Sanofi/Sobi）；Idelvion（albutrepenonacog alfa；CSL Behring）和 Rebinyn/Refixia（nonacog beta pegol；諾和諾德）
  • 旁路劑：NovoSeven（eptacog alfa；Novo Nordisk）；FEIBA（因子八抑制旁路劑；Takeda）和 Sevenfact/Cevenfacta（凝血因子 VIIa [重組]-jncw；LFB Biotechnologies/HEMA Biologics）

基因治療

• 已通過核准藥物
  • Hemgenix（etranacogene dezaparvovec；uniQure/CSL Behring）
  • Beqvez/Durveqtix（fidanacogene elaparvovec；輝瑞）

非係數再調整代理商

• 已通過核准藥物
  • Hympavzi(marusutashimabu，Pfizer)
• 開發平台(管線)醫藥品
  • Alhemo（康珠單抗；諾和諾德）
  • Fitusiran（Alnylam 製藥公司/賽諾菲）
  • SerpinPC（Centessa 製藥公司）

今後的治療趨勢

附錄

KOL快報

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