A型血友病市場:KOL的洞察

本報告提供全球A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

• 凝血因子刺激藥
• 開發平台(管線)醫藥品

新的長期作用型重組因素VIII療法

• 已通過核准醫藥品
  • Artubio（Efanesoctocog alpha、amnicus/Bioverativ [賽諾菲]）

非因素補充療法

• 基因治療
  • 主要見解摘要
  • 核准的藥品
  • Roctavian（Baroctocogen loxaparvovec，BioMarin Pharmaceutical）
  • 正在研究藥物
  • ASC618（CRISPR/Cas9 編輯的 hFVIII 基因療法，ASC Therapeutics）
  • Peboctocogenkamaparvovec（DTX201，拜耳醫療保健/Dimension Therapeutics）
  • Diroctocogenphytelparvovec（PF-07055480/SB-525，Sangamo Therapeutics/輝瑞）
  • Dilroctocogen Samopalvovec（SPK-8011，Spark Therapeutics/羅氏）
  • TAK-754（SHP654，武田）
• 抗組織因子途徑阻斷單株抗體
  • 主要見解摘要
  • 核准的藥品
  • Arhemo（concizumab，諾和諾德）
  • 正在研究藥物
  • Marstacimab（抗 TFPI 單株抗體，輝瑞）
• 抗凝血? III 抑制劑/RNA 幹擾
  • 主要見解摘要
  • 正在研究藥物
  • Fitusiran（Alnylam 製藥公司/賽諾菲）
• 活化蛋白 C 受體調節劑
  • 正在研究藥物
  • Serpin PC（Centessa Pharmaceuticals）
• 其他計劃
  • 正在研究藥物
  • BT200（RNA 調節劑/vWF 抑制劑，Band Therapeutics）

附錄

新聞

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)