發育性和癲癇性腦病變（DEE）：新型療法、未滿足的需求和TPP洞察報告，2026年

市場概覽

• 預計到 2035 年，西班牙 DEE 市場規模將從約 1.8 億美元成長到 4.8 億美元。
• 市場成長受以下因素支撐：
• 擴大新型抗癲癇藥物的使用
• 精準醫療和基因治療的擴展
• 市場擴張的驅動力是巨大的未滿足需求和對更完善的治療方案的需求。
• 未來的成長將取決於基因療法和創新的緩解疾病治療方法。

對發育性和癲癇性腦病變(DEE) 的新興療法和 TPP 的見解

Thelansis 的「發育和癲癇性腦病變(DEE)：新型療法、未滿足的需求和 TPP 洞察報告，2026」對該適應症的關鍵新興療法和主要藥物發現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)、臨床試驗設計和關鍵意見領袖 (KOL) 的見解。

發育性和癲癇性腦病變（DEEs）概述

發育性癲癇性腦病變（DEEs）是一組高度異質性的嚴重神經發育症候群，起病於兒童，其特徵為藥物難治性癲癇、發育停滯或倒退以及認知障礙。與傳統癲癇不同，DEEs透過潛在的遺傳或結構性病因以及持續且破壞性的腦電活動本身損害神經發育。 Dravet症候群、雷葛氏症候群和SCN2A/8A基因突變等典型症候群通常透過先進的多基因檢測確診，這些檢測取代了以往廣泛的診斷猜測。雖然標準的頻譜抗癲癇藥物（如芬氟拉明和大麻二酚）可以緩解基本症狀，但該領域正積極向基因型特異性精準藥理學時代邁進。目前的治療趨勢以針對中樞神經系統的標靶療法為基礎，這些療法旨在解決特定的離子通道突變。一項突破性進展是reltrigine，它是一種選擇性持續鈉電流抑制劑，針對SCN2A和SCN8A DEE，目前正在接受FDA優先審評，預計將於2026年12月27日獲批。同時，針對多種DEE的III期EMERALD試驗也取得了顯著進展。反義寡核苷酸（ASO）研發管線也取得了顯著成功，包括用於治療Dravet症候群的zolevnersen（STK-001）和elsnersen，後者最近在早期發作的SCN2A-DEE患者中顯示出與安慰劑相比，每月癲癇發作頻率降低了77%。這些標靶分子療法展現了首個真正意義上的緩解疾病潛力，能夠穩定癲癇發作並延緩潛在的認知功能進展。

主要亮點

• 在德國，預計到 2025 年，DEE 病例數將增加到約 16,265 例，到 2035 年將增加到 16,395 例。
• DEE 是一組嚴重的神經系統疾病，其特徵是難以控制的癲癇發作和發育障礙。
• 基因檢測技術的日益普及使得早期診斷和選擇精準的治療方法成為可能。
• 持續性癲癇發作和認知障礙帶來的負擔仍然存在巨大的未滿足需求。
• 新型基因標靶治療和疾病修正治療正在拓展未來的治療選擇。

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
• 發布日期等的預測
• 臨床試驗現況分析
• 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
• 特徵和水平
• 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
• 學位旨在解決重大未滿足的需求
• 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

• Longboard Pharmaceuticals
• Praxis Precision Medicines
• Neurocrine Biosciences
• Bright Minds Biosciences Pty Ltd.
• Encoded Therapeutics

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

Developmental and Epileptic Encephalopathies (DEEs) Emerging Therapy and TPP Insights

Thelansis's "Developmental and Epileptic Encephalopathies (DEEs) 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.

Developmental and Epileptic Encephalopathies (DEEs) Overview

Developmental and epileptic encephalopathies (DEEs) are a heterogeneous group of severe, pediatric-onset neurodevelopmental syndromes characterized by drug-resistant epilepsy, developmental stagnation or regression, and cognitive deficits. Unlike classic epilepsies, DEEs damage neurodevelopment through both the underlying genetic or structural etiology and the continuous, disruptive electrographic activity itself. Syndromic archetypes like Dravet, Lennox-Gastaut, and SCN2A/8A mutations are routinely confirmed via advanced multigene panels, which have replaced broad diagnostic guesswork. While standard broad-spectrum antiseizure medications (such as fenfluramine and cannabidiol) provide baseline symptom mitigation, the field has aggressively moved into an era of genotype-specific precision pharmacology. The current landscape is anchored by targeted central nervous system therapeutics designed to address specific ion channel variants. A landmark shift involves relutrigine, a selective inhibitor of persistent sodium currents under FDA Priority Review with a target action date of December 27, 2026, for SCN2A and SCN8A DEEs, complemented by its broad-DEE Phase 3 EMERALD trial. Concurrently, the antisense oligonucleotide (ASO) pipeline has achieved dramatic success, led by zorevunersen (STK-001) for Dravet syndrome and elsunersen, which recently demonstrated a 77% placebo-adjusted monthly seizure reduction in early-onset SCN2A-DEE. These targeted molecular frameworks offer the first true disease-modifying prospects to stabilize both seizure activity and underlying cognitive trajectories.

Key Highlights

• In Germany, the DEE prevalence patient pool is projected to increase from approximately 16,265 cases in 2025 to 16,395 by 2035.
• DEE represents a group of severe neurological disorders characterized by refractory seizures and developmental impairment.
• Genetic testing is increasingly enabling earlier diagnosis and precision treatment selection.
• Significant unmet need remains due to persistent seizure burden and cognitive dysfunction.
• Emerging gene-targeted and disease-modifying therapies are expanding future treatment opportunities.

Market Overview

• The Spain DEE market is projected to grow from approximately $180 MN to $480 MN by 2035.
• Market growth is supported by:
• Increasing adoption of novel anti-seizure medications
• Expansion of precision medicine and genetic therapies
• Market expansion is driven by high unmet need and increasing treatment intensity.
• Future growth will depend on gene therapies and innovative disease-modifying treatment approaches.

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

• Longboard Pharmaceuticals
• Praxis Precision Medicines
• Neurocrine Biosciences
• Bright Minds Biosciences Pty Ltd.
• Encoded Therapeutics

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)