血友病A市場：KOLの洞察

当レポートでは、世界の血友病A市場について調査し、市場の概要とともに、上市済み治療法、パイプライン動向、将来の見通しなどを提供しています。

目次

エグゼクティブサマリー

• 現在および将来の治療アルゴリズム

調査目的

• 血液凝固因子刺激薬
• パイプライン医薬品

新しい長期作用型組換え因子VIII療法

• 承認済み医薬品
  • アルツビオ（エファネソクトコグアルファ、アムニクス/Bioverativ[Sanofi]）

非因子補充療法

• 遺伝子治療
  • 主な洞察の要約
  • 承認済み医薬品
  • ロクタビアン（バロクトコゲンロキサパルボベック、BioMarin Pharmaceutical）
  • パイプライン医薬品
  • ASC618（CRISPR/Cas9編集hFVIII遺伝子治療、ASC Therapeutics）
  • ペボクトコゲンカマパルボベック（DTX201、Bayer HealthCare/Dimension Therapeutics）
  • ジロクトコゲンフィテルパルボベック（PF-07055480/SB-525、Sangamo Therapeutics/Pfizer）
  • ディルロクトコゲンサモパルボベック（SPK-8011、Spark Therapeutics/Roche）
  • TAK-754（SHP654、武田）
• 抗組織因子経路阻害モノクローナル抗体
  • 主な洞察の要約
  • 承認済み医薬品
  • アルヘモ（コンシズマブ、Novo Nordisk）
  • パイプライン医薬品
  • マルスタシマブ（抗TFPI mAb、Pfizer）
• アンチトロンビンIII阻害剤/RNA干渉
  • 主な洞察の要約
  • パイプライン医薬品
  • フィトゥシラン（Alnylam Pharmaceuticals/Sanofi）
• 活性化プロテインC受容体モジュレーター
  • パイプライン医薬品
  • セルピンPC（Centessa Pharmaceuticals）
• その他のプログラム
  • パイプライン医薬品
  • BT200（RNAモジュレーター/vWF阻害剤、Band Therapeutics）

付録

ニュース

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)