Dravet 綜合徵 (DS) 市場：初步調查（競爭信息、市場分析和 2032 年預測）

本報告調查了全球 Dravet 綜合徵 (DS) 市場，並提供了市場概況、2032 年預測、當前治療和未滿足的需求、國家趨勢、市場驅動因素和限制等。

目錄

執行摘要

• 主要調查結果

Dravet 綜合徵 (DS) 疾病的背景

• Dravet 綜合徵 (DS) 的定義
• 症狀和原因
• SCN1A基因
• 診斷
• 鑑別診斷

到 2032 年的流行病學和預測

• 主要調查結果
• 方法和數據源

Dravet 綜合徵（DS）流行病學和模型參數的主要來源

• 巴西
• 墨西哥

當前的治療和醫療實踐

• 治療和醫療實踐
• Dravet 綜合徵 (DS) 癲癇發作的治療
• 自 2017 年以來獲得美國 FDA 批准的治療 Dravet 綜合徵的藥物
• 大麻二酚治療 Dravet 綜合徵 (DS)

未滿足的需求

治療章節

新療法

Dravet 綜合症 (DS) - 價格和贖回

未來治療範式

• Situation Syndrome (DS) 競爭對手情況和預期批准
• 未來的治療算法和競爭對手定位
• 新療法的重要數據摘要

當前和新療法的年度費用

市場展望

到 2032 年的國家市場預測

各國市場預測

• 巴西
• 墨西哥

市場促進因素和限制

報告調查方法

附錄

Growth of the incidence DS population will contribute to increasing sales in the DS therapy market over our forecast period. An estimated annual growth rate of ~1% in the diagnosed incident DS population across the major pharmaceutical markets will contribute to increasing DS therapy sales. In 2018, the drug-treated cases of Dravet Syndrome (DS) in Brazil & Mexico were 8,392 cases. These cases are expected to increase to 11,335 cases in 2032 owing to changes in population demographics and increasing cases of DS.

The continuous involvement of supportive treatments including antiepileptic therapies for the management of DS patients will contribute to the overall growth of the DS therapy market. We forecast that total sales of the total care market will reach $276 Million by 2032.

Dravet syndrome is a severe form of epilepsy characterized by frequent, prolonged seizures often triggered by high body temperature (hyperthermia), developmental delay, speech impairment, ataxia, hypotonia, sleep disturbances, and other health problems. DS is considered an epileptic encephalopathy due to seizures. In addition, it is considered a "channelopathy" because the effects of the mutation on the sodium channel appear to contribute to the disorder independently of the seizures.

Dravet Syndrome - DS Epidemiology

We estimate the total number of Dravet Syndrome (DS) prevalent cases in the Brazil and Mexico of 1:20,000 or 0.005%. There is a significant amount of shortage of large-scale epidemiological reports. The rarity of the disease further compounds the problem at hand as there exists a lack of firmly established and specific diagnostic criteria and some of the studies may have chances of biasness depending on the geographical area studied. There also exists methodological challenges for researchers due to measurements involving a small sample population.

According to the KOLs View, approximately 50% of the cases of Dravet Syndrome (DS) are diagnosed. Patients with Dravet syndrome may be misdiagnosed with myoclonic atonic epilepsy, Lennox-Gastaut syndrome, myoclonic epilepsy of infancy, genetic epilepsy with febrile seizures plus, atypical febrile seizures, and mitochondrial disorders. Additionally, some children may be diagnosed with focal epilepsy.

Dravet Syndrome -DS Market Forecast

The Dravet Syndrome (DS) therapy market is expected to experience high growth throughout our study period, increasing from $XX billion in 2018 to $XX billion in 2032, representing ~1% annual growth. Primary drivers of this growth will be uptake of the novel upcoming treatment like Soticlestat (Takeda/Ovid Therapeutics), Lorcaserin (Eisai/Arena Pharmaceuticals), STK 001 (Stoke Therapeutics, Inc), EPX 100 (Epygenix Therapeutics), ETX101 (Encoded Therapeutics).

Most interviewed experts believe that patients will benefit from the upcoming therapies based on targeting CH24H inhibitors, selective serotonin 5-HT2c receptor, NAV1.1 Voltage-gated Sodium Channel Modulators and Specific GABAA Receptor have the potential to increase the therapeutic options for Dravet Syndrome (DS) patients. Expected launch of late-stage product such as Soticlestat, Lorcaserin and STK 001 are likely to change the current treatment paradigm for Dravet Syndrome (DS) and these approved therapies will be more preferrable in the future.

Report Highlights

• Dravet Syndrome-DS Current Market Trends

• Dravet Syndrome-DS Current & Forecasted Cases across Brazil & Mexico

• Dravet Syndrome-DS Market Opportunities And Sales Potential for Agents

• Dravet Syndrome-DS Patient-based Market Forecast to 2032

• Dravet Syndrome-DS Untapped Business Opportunities

• Dravet Syndrome-DS Product Positioning Vis-a-vis Competitors' Products

• Dravet Syndrome-DS KOLs Insight

Table of Contents

Executive Summary

• Key Findings

Dravet Syndrome (DS) Disease Background

• Dravet Syndrome (DS) Definition
• Symptoms & Causes
• SCN1A Gene
• Diagnosis
• Differential Diagnosis

Epidemiology Estimated and Forecast to 2032

• Key Findings
• Methods and data Sources
  • Country Specific Prevalent cases of Dravet Syndrome (DS) (Brazil and Mexico)
  • Country Specific Diagnosed and Drug Treated Cases of Dravet Syndrome (DS)

Key Sources for Dravet Syndrome (DS) Epidemiology and Model Parameters

• Brazil
  • Brazil Prevalent cases of Dravet Syndrome (DS)
  • Brazil Diagnosed and Drug Treated Cases of Dravet Syndrome (DS)
• Mexico
  • Mexico Prevalent cases of Dravet Syndrome (DS)
  • Mexico Diagnosed and Drug Treated Cases of Dravet Syndrome (DS)

Current Therapies and Medical Practice

• Treatments & Medical Practices
• Treatment of Seizures in Dravet Syndrome (DS)
• Approved Therapies for Dravet Syndrome by U.S. FDA since 2017
• Cannabidiol in the treatment of Dravet Syndrome (DS)

Unmet Needs

Therapies Chapters

• Fenfluramine (Zogenix)
• Epidiolex (Jazz Pharmaceuticals plc/GW Pharmaceuticals)
• Stiripentol (Biocodex)

Emerging Therapies

• Pipeline Overview
• Therapeutic Developments Pipeline for Dravet Syndrome (DS)
  • Product Analysis
    • Soticlestat (Takeda/Ovid Therapeutics)
      • Product Profile
      • Clinical Development
      • Sales & Market Opportunity by 2032
    • Lorcaserin (Eisai/Arena Pharmaceuticals)
      • Product Profile
      • Clinical Development
      • Sales & Market Opportunity by 2032
    • STK 001 (Stoke Therapeutics)
      • Product Profile
      • Clinical Development
      • Sales & Market Opportunity by 2032
    • EPX 100 (Epygenix Therapeutics)
      • Product Profile
      • Clinical Development
      • Sales & Market Opportunity by 2032
    • EPX 200 (Epygenix Therapeutics)
      • Product Profile
      • Sales & Market Opportunity by 2032
    • EPX 300 (Epygenix Therapeutics)
      • Product Profile
      • Sales & Market Opportunity by 2032
    • ETX101 (Encoded Therapeutics)
      • Product Profile
      • Sales & Market Opportunity by 2032
    • Huperzine A (Supernus Pharmaceuticals/Biscayne Neurotherapeutics)
      • Product Profile
    • LP352 (Longboard Pharmaceuticals/Arena Pharmaceuticals)
      • Product Profile
    • NT-102 (Neuroene Therapeutics)
      • Product Profile
    • NCT10004 (Engrail Therapeutics/NeuroCycle Therapeutics)
      • Product Profile
    • BMB-101 (Bright Minds Biosciences)
      • Product Profile
    • OPK88001 (CAMP4 Therapeutics/OPKO Health)
      • Product Profile

Dravet Syndrome (DS)- Pricing & Reimbursement

Future Treatment Paradigm

• Dravet Syndrome (DS) Competitor Landscape and Approvals Anticipated
• Future Treatment Algorithms and Competitor Positioning
• Key Data Summary for Emerging Treatment

Annual Cost of Current & Emerging Therapies

Market Outlook

• Key Findings

Country Specific Market Forecast to 2032

• Total Market for Dravet Syndrome (DS) 2018-2032 (USD Million)
• Total Market for Dravet Syndrome (DS) by Therapies 2018-2032 (USD Million)

Market Forecast by Country

• Brazil
  • Brazil Market for Dravet Syndrome (DS) 2018-2032 (USD Million)
  • Brazil Market for Dravet Syndrome (DS) by Therapies 2018-2032 (USD Million)
• Mexico
  • Mexico Market for Dravet Syndrome (DS) 2018-2032 (USD Million)
  • Mexico Market for Dravet Syndrome (DS) by Therapies 2018-2032 (USD Million)

Market Drivers and Constraints

• What Factors Are Driving the Market for Dravet Syndrome (DS)?
• What Factors Are Constraining the Market for Dravet Syndrome (DS)?

Report Methodology

Appendix