A型血友病市场:KOL的洞察

本报告提供全球A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

• 凝血因子刺激药
• 开发平台(管线)医药品

新的长期作用型重组因素VIII疗法

• 已通过核准医药品
  • Artubio（Efanesoctocog alpha、amnicus/Bioverativ [赛诺菲]）

非因素补充疗法

• 基因治疗
  • 主要见解摘要
  • 核准的药品
  • Roctavian（Baroctocogen loxaparvovec，BioMarin Pharmaceutical）
  • 正在研究药物
  • ASC618（CRISPR/Cas9 编辑的 hFVIII 基因疗法，ASC Therapeutics）
  • Peboctocogenkamaparvovec（DTX201，拜耳医疗保健/Dimension Therapeutics）
  • Diroctocogenphytelparvovec（PF-07055480/SB-525，Sangamo Therapeutics/辉瑞）
  • Dilroctocogen Samopalvovec（SPK-8011，Spark Therapeutics/罗氏）
  • TAK-754（SHP654，武田）
• 抗组织因子途径阻断单株抗体
  • 主要见解摘要
  • 核准的药品
  • Arhemo（concizumab，诺和诺德）
  • 正在研究药物
  • Marstacimab（抗 TFPI 单株抗体，辉瑞）
• 抗凝血? III 抑制剂/RNA 干扰
  • 主要见解摘要
  • 正在研究药物
  • Fitusiran（Alnylam 製药公司/赛诺菲）
• 活化蛋白 C 受体调节剂
  • 正在研究药物
  • Serpin PC（Centessa Pharmaceuticals）
• 其他计划
  • 正在研究药物
  • BT200（RNA 调节剂/vWF 抑制剂，Band Therapeutics）

附录

新闻

From the dominance of Roche's Hemlibra, the promising rise of Sanofi's Altuviiio, and the potential of gene therapies like BioMarin's Roctavian, uncover the strategic trends driving the future of haemophilia A management. This report offers an in-depth analysis of the evolving therapeutic landscape, highlighting key opportunities and challenges in advancing haemophilia A treatment. Examine the strategic implications of these developments on drug development, market dynamics, and patient care strategies, offering a forward-looking perspective on the haemophilia A landscape.

Key brands covered in this report:

Key questions answered:

• Which drugs constitute the first- and subsequent-line treatments of choice for haemophilia A, and which product attributes contribute to this preference?
• How is the product perceived by the medical community in terms of efficacy, tolerability, ease of administration and other attributes, and how does it compare with other current and pipeline treatment options?
• What do pipeline therapies for haemophilia A need to show in order to become the treatment of choice in a specific line of therapy, and is it likely these products will meet these requirements?
• How will the use of each current and pipeline haemophilia A product change in the future in terms of patient segment, line of therapy and preference?
• What will pipeline products for haemophilia A need to show in terms of efficacy and tolerability endpoints to compete effectively, and what is the likelihood that they will achieve those endpoints?
• Which pipeline products are the most promising and how will they impact current players in the market?
• How will the treatment landscape for haemophilia A evolve in the future for each line of therapy?

Table of Contents

Executive summary (9)

• Current and future treatment algorithm

Research objectives (37)

• Blood coagulation factor stimulants (19)
  • Approved drugs
  • Hemlibra (emicizumab; Roche) (19)
• Pipeline drugs (15)
  • Mim8 (NNC0365-3769 A; Novo Nordisk) (11)
  • NXT-007 (RG-6512/RO-7589655; Chugai/Roche) (4)

Newer long-acting recombinant Factor VIII therapies (16)

• Approved drugs (16)
  • Altuviiio (efanesoctocog alfa; Amunix/Bioverativ [Sanofi]) (16)

Non-factor replacement therapies (67)

• Gene therapy (27)
  • Key insights summary (2)
  • Approved drugs
  • Roctavian (valoctocogene roxaparvovec; BioMarin Pharmaceutical) (1)
  • Pipeline drugs
  • ASC618 (CRISPR/Cas9-edited hFVIII gene therapy; ASC Therapeutics) (1)
  • Peboctocogene camaparvovec (DTX201; Bayer HealthCare/Dimension Therapeutics) (1)
  • Giroctocogene fitelparvovec (PF-07055480/SB-525; Sangamo Therapeutics/Pfizer) (1)
  • Dirloctocogene samoparvovec (SPK-8011; Spark Therapeutics/Roche) (1)
  • TAK-754 (SHP654; Takeda) (20)
• Anti-tissue factor pathway inhibitor mAbs (13)
  • Key insights summary (2)
  • Approved drugs
  • Alhemo (concizumab; Novo Nordisk) (1)
  • Pipeline drugs
  • Marstacimab (anti-TFPI mAb; Pfizer) (10)
• Antithrombin III inhibitors/RNA interference (9)
  • Key insights summary (2)
  • Pipeline drugs
  • Fitusiran (Alnylam Pharmaceuticals/Sanofi) (7)
• Activated protein C receptor modulators (10)
  • Pipeline drugs
  • SerpinPC (Centessa Pharmaceuticals) (10)
• Other programmes (8)
  • Pipeline drugs
  • BT200 (RNA modulator/vWF inhibitor; Band Therapeutics) (8)

Appendix (5)

• KOL details (5)
  • KOLs from the USA (1)
  • KOLs from Europe (3)

News (1)