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市場調查報告書
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2092505

Dravet症候群（DS）：新療法、未滿足的需求和TPP洞察報告，2026年

Dravet Syndrome (DS) - Emerging Therapy, with Unmet Needs and TPP Insights Report - 2026
出版日期: | 出版商: Thelansis Knowledge Partners | 英文 53 Pages | 商品交期: 2-3個工作天內

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簡介目錄

市場概覽

  • 法國Dravet症候群市場預計將從2025年的9,975萬美元成長到2035年的2.625億美元。
  • 推動市場成長的因素如下：
  • 引入新的抗癲癇療法
  • 擴大精準醫療方法的應用
  • 預計未來市場擴張將主要由疾病修正治療和基因療法推動。

德拉韋氏症候群 (DS) 的新療法及對 TPP 的深入了解

Thelansis 的《Dravet 症候群 (DS)：新型療法、未滿足的需求和目標產品概況 (TPP) 洞察報告，2026》對該適應症的關鍵新興療法和主要藥物開發機會進行了全面分析，包括新興的競爭格局、未滿足的需求、目標產品概況 (TPP)、臨床試驗設計以及關鍵意見領袖 (KOL) 的見解。

Dravet症候群（DS）概述

Dravet症候群，又稱重症嬰兒肌陣攣性癲癇，是一種罕見且難治性遺傳性癲癇，通常在出生後第一年內發病。此病以頻繁、持續時間長的癲癇發作為特徵，常由體溫過高（如發燒或溫水浴）誘發，最終發展為多種類型的癲癇發作，包括肌陣攣性發作和非典型失神發作。約80-85%的病例是由SCN1A基因的突變或缺失引起的，該基因編碼Nav1.1電壓門控鈉通道，該通道對抑制性神經元功能至關重要。雖然大多數此類突變是自發性的，但少數屬於GEFS+家族頻譜，其他病例可能涉及PCDH19基因突變或體細胞嵌合。除癲癇發作外，此症候群還伴隨明顯的發育遲緩、共濟失調、肌張力低下和睡眠障礙。由於常見的鈉通道阻斷劑可能會異常增加癲癇發作的頻率，因此治療特別複雜，要更好地控制癲癇發作可能需要使用特定的療法，例如司替戊醇、氟苯丙胺或大麻二酚。

主要亮點

  • 在德國，經確診的 Dravet 症候群突變病例數預計將從 2025 年的 1749 例增加到 2035 年的 1781 例。
  • Dravet 症候群是一種嚴重的神經發育障礙和癲癇性腦病變，通常由 SCN1A 基因突變引起。
  • 患者會出現難治性癲癇發作和嚴重的神經發育障礙。
  • 標靶治療的廣泛應用提高了癲癇控制效果和患者預後。

透過對醫生和關鍵意見領袖 (KOL) 的調查所獲得的見解：

  • 透過對關鍵意見領袖 (KOL) 的訪談，我們獲得了更多見解，從而進一步完善了調查結果。
  • 調查問卷將根據客戶的要求進行客製化。

交付成果格式：

  • PowerPoint簡報
  • MS Excel

主要問題

  • 詳細的競爭格局趨勢
  • 管道分析
  • 符合新興療法條件的患者
  • 大公司
  • 主要作用機制
  • 發布日期預測等。
  • 臨床試驗趨勢分析
  • 目標患者群
  • 試驗終點
  • 測試設計
  • 受試者招募標準等。
  • 未滿足的需求和機遇
  • 目前主要療法的有效性
  • 未滿足需求的關鍵領域
  • 主要未滿足需求的市場規模估算
  • 目標產品概況
  • 屬性和級別
  • 醫生開立的處方
  • 預期患者佔有率
  • 關鍵意見領袖對領先新興療法的見解
  • 意識
  • 預期用途/處理線
  • 以滿足重大未滿足需求的程度
  • KOL評論

目標國家

  • G8
    • 美國
    • EU5
      • 法國
      • 德國
      • 義大利
      • 西班牙
      • 英國
    • 日本
    • 中國

大公司

  • Ionis Pharmaceuticals, Inc.
  • Stoke Therapeutics, Inc.
  • Encoded Therapeutics
  • UCB BIOSCIENCES, Inc.
  • Epygenix
  • Longboard Pharmaceuticals
  • Zogenix International Limited, Inc., a subsidiary of Zogenix, Inc.
  • Takeda
  • Zogenix, Inc.
  • Bright Minds Biosciences Pty Ltd.
  • Jazz Pharmaceuticals
  • Biocodex

目錄

第1章：主要調查結果及分析師說明

  • 主要趨勢：市場概況、SWOT分析、商業性利益與風險等。

第2章 競爭情勢

  • 目前的治療方法
  • 重點
  • 診斷和治療過程/演算法
  • 主要療法概述及關鍵意見領袖見解
  • 新興療法
  • 重點
  • 診斷和治療過程/演算法
  • 領先的新興療法—概述和關鍵意見領袖見解

第3章 產品屬性分析

  • 重點
  • 科學屬性
  • 商業性屬性
  • 產品定位

第4章：初步調查

  • 目前治療狀態
  • 主要治療方法與目標患者族群的比較
  • 主要特點和優勢
  • 未來治療環境
  • 當前挑戰
  • 未滿足的需求
  • 新興療法
  • 主要治療方法與目標患者族群的比較
  • 主要特點和優勢
  • 未來治療前景
  • 未滿足的需求和關鍵意見領袖的期望

第5章：未滿足的需求和TPP分析

  • 新興療法的主要未滿足需求和未來成就
  • TPP分析與KOL預期

第6章 監理與報銷環境

第7章附錄

簡介目錄

Dravet Syndrome (DS) Emerging Therapy and TPP Insights

Thelansis's "Dravet Syndrome (DS) Emerging Therapy, with Unmet Needs and TPP Insights Report - 2026" provides a comprehensive analysis of the emerging competitive landscape, unmet needs, target product profiles (TPPs), trial designs, and KOL insights on key emerging therapies and key drug development opportunities in the indication.

Dravet Syndrome (DS) Overview

Dravet syndrome, also known as severe myoclonic epilepsy of infancy, is a rare and refractory genetic epilepsy that typically begins in the first year of life. The disorder is primarily characterized by frequent, prolonged seizures often triggered by hyperthermia, such as fevers or warm baths, and eventually progresses to include multiple seizure types including myoclonic and atypical absence seizures. Approximately 80 to 85 percent of cases result from a mutation or deletion in the SCN1A gene, which encodes the Nav1.1 voltage-gated sodium channel essential for the function of inhibitory neurons. While most of these mutations occur spontaneously, a small percentage are part of the GEFS+ familial spectrum, and other cases may involve mutations in the PCDH19 gene or somatic mosaicism. In addition to seizures, the syndrome is associated with significant developmental delays, ataxia, hypotonia, and sleep disturbances. Management is particularly complex because common sodium-channel-blocking medications can paradoxically increase seizure frequency, requiring the use of specific treatments such as stiripentol, fenfluramine, or cannabidiol to achieve better seizure control.

Key Highlights

  • In Germany, mutation-positive Dravet Syndrome cases are projected to increase from 1,749 in 2025 to 1,781 by 2035.
  • Dravet Syndrome is a severe developmental and epileptic encephalopathy typically caused by SCN1A mutations.
  • Patients experience treatment-resistant seizures and substantial neurodevelopmental impairment.
  • Increasing use of targeted therapies is improving seizure control and patient outcomes.

Market Overview

  • The France Dravet Syndrome market is projected to grow from $99.75M in 2025 to $262.5M by 2035.
  • Market growth is driven by:
  • Adoption of novel anti-seizure therapies
  • Increasing use of precision medicine approaches
  • Future expansion will depend on disease-modifying and gene-based therapies.

Insights driven by surveys with physician / key opinion leaders:

  • Survey findings are corroborated and enriched by insights from interviews with leading KOLs
  • Survey is customized based on client requirements

Deliverables format:

  • PowerPoint presentation
  • MS Excel

Key business questions answered:

  • Detailed emerging competitive landscape
  • Pipeline analysis
  • Target patients for emerging therapies
  • Key companies
  • Key mechanism of actions
  • Launch date estimates, etc.
  • Clinical trial landscape analysis
  • Target patient segments
  • Trial endpoints
  • Trial design
  • Recruitment criteria, etc.
  • Unmet Needs and Opportunities
  • Performance of key current therapies
  • Top areas of unmet needs
  • Opportunity sizing for key unmet needs
  • Target Product Profiles
  • Attributes and levels
  • Physician likelihood of prescribing
  • Expected patient shares
  • KOL insights on key emerging therapies
  • Level of awareness
  • Expected use / line of therapy
  • Extent to fulfil key unmet needs
  • KOL quotes

Countries Covered

  • G8
    • United States
    • EU5
      • France
      • Germany
      • Italy
      • Spain
      • U.K.
    • Japan
    • China

Apart from the G8 Market, adding any additional country data to the dashboard will cost USD 1,750 per country

Companies Mentioned

  • Ionis Pharmaceuticals, Inc.
  • Stoke Therapeutics, Inc.
  • Encoded Therapeutics
  • UCB BIOSCIENCES, Inc.
  • Epygenix
  • Longboard Pharmaceuticals
  • Zogenix International Limited, Inc., a subsidiary of Zogenix, Inc.
  • Takeda
  • Zogenix, Inc.
  • Bright Minds Biosciences Pty Ltd.
  • Jazz Pharmaceuticals
  • Biocodex

Table of Contents

1. Key Findings and Analyst Commentary

  • Key trends: market snapshots, SWOT analysis, commercial benefits and risk, etc.

2. Competitive Landscape

  • Current therapies
  • Key takeaways
  • Dx and Tx journey/algorithm
  • Key current therapies - profiles and KOL insights
  • Emerging therapies
  • Key takeaways
  • Dx and Tx journey/algorithm
  • Key emerging therapies - profiles and KOL insights

3. Product Attribute Analysis

  • Key takeaways
  • Scientific attributes
  • Commercial attributes
  • Product positioning

4. Primary Market Research

  • Current treatment landscape
  • Key therapies vs. focused patient segment
  • Key attributes and benefits
  • Futures treatment landscape
  • Current challenges
  • Unmet needs
  • Emerging therapies
  • Key therapies vs. focused patient segment
  • Key attributes and benefits
  • Futures treatment landscape
  • Unmet needs and KOL expectations

5. Unmet Need and TPP Analysis

  • Top unmet needs and future attainment by emerging therapies
  • TPP analysis and KOL expectations

6. Regulatory and Reimbursement Environments (by country and payer insights)

7. Appendix (e.g., bibliography, methodology)