Dravet Syndrome: Drug Development Pipeline Study, H1 2018 - Product Image

Dravet Syndrome: Drug Development Pipeline Study, H1 2018

  • ID: 4463286
  • Report
  • Region: Global
  • 46 Pages
  • VPA Research
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10 Drugs have been Assigned with Orphan Drug Designation

FEATURED COMPANIES

  • Biscayne Pharmaceuticals
  • Epygenix Therapeutics
  • INSYS Therapeutics Inc
  • OPKO Health Inc
  • Ovid Therapeutics; Takeda
  • Xenon Pharmaceuticals Inc
  • MORE

The most comprehensive pipeline review “Dravet Syndrome - Drug Development Pipeline Study” provides in-depth analysis and update information on Dravet Syndrome pipeline drugs. Current status of all the drugs in the Dravet Syndrome pipeline is provided in the research work.
 
Previously known as severe myoclonic epilepsy of infancy (SMEI), the neurological disease is attracting wide interest from pharmaceutical companies over the past five years. In particular, with 10 drugs receiving orphan drug status, the prominence of Dravet Syndrome pipeline is robust.

The report provides detailed analysis of Dravet Syndrome pipeline including companies involved, drugs in different stages of development, drug development details across regions (North America, Europe, Asia Pacific and Rest of the World). Further, the Dravet Syndrome report identifies aspects important in modifying industry market scenarios, increasing opportunities and list of potential companies that impact the Dravet Syndrome industry.

Excerpts from this Report:

Dravet syndrome is a genetically- modified seizure condition associated with extended, frequent refractory seizures. The condition is also called as severe myoclonic epilepsy of infancy (SMEI). Dravet Syndrome occurs due to defects in SCN1A genes in brain and causes cognitive and behavioral disorders and around 15- 20% death rate.

Seizures start at the age of one, and are frequently associated with febrile convulsions. Subsequently, multiple types of seizures occur such as epilepticus. After the age 2, baby shows signs of deteriorating cognitive and behavioral skills, resulting in long term disability.

US FDA has not approved any drugs and EMA has approved only one drug. Treatment options include Clobazam plus valporate plus stiripentol, which offers only a modest seizure control.

Lack of approved complete treatments for the disease has spurred interest among pharmaceutical companies, in specific over the past few years. As of February 2018, the Dravet syndrome pipeline consists of at least 15 drug candidates, of which 4 are in late stage. Of this, 10 drugs have been given Orphan Drug Designations. This research work reviews the current status of Dravet Syndrome drug development pipeline.

Dravet Syndrome pipeline comprises of 15 drugs under development as of February 2018. Of these, 4 drugs are in Phase 1 and 4 are in phase 2. Further, 3 drugs remain in Phase 3 while one drug has moved into Pre-registration phase. In addition, 3 drugs are in Pre-Clinical Stage.

23 companies/ institutions are developing the pipeline for Dravet Sysndrome.

Scope:

  • All latest industry developments and their impact on projects and companies
  • Complete overview of global Dravet Syndrome therapy options and treatments
  • The pipeline study reviews drug details for Dravet Syndrome by developing companies, institutions, mechanism of action, Target, Route, Phase (pre-clinical/ Discovery, Phase 1, Phase 2, Phase 3, Pre-Registration, IND/ NDA) and molecule
  • Mergers, Acquisitions, licensing, co-development and project acquisition news for Dravet Syndrome market
  • Dravet Syndrome drugs being developed in combination with other drugs are also detailed
  • Company wise drugs listed

Products Mentioned:

  • AntagoNAT
  • Ataluren (Translarna™)
  • BIS-001
  • Clobazam
  • Epidiolex
  • EPX 100
  • EPX 200
  • EPX 300
  • Nav1.6 sodium channel
  • PRAX 330
  • SAGE-217
  • SGE-516
  • Synthetic cannabidiol
  • TAK 935
  • ZX-008
READ MORE
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FEATURED COMPANIES

  • Biscayne Pharmaceuticals
  • Epygenix Therapeutics
  • INSYS Therapeutics Inc
  • OPKO Health Inc
  • Ovid Therapeutics; Takeda
  • Xenon Pharmaceuticals Inc
  • MORE

1.1 List of Figures
1.2 List of Tables

2 Executive Summary
2.1 Disease Overview
2.2 Pipeline Snapshot
2.3 Pipeline Drugs by Phase
2.4 Pipeline Drugs by Company
2.5 Pipeline Drugs by Mechanism Of Action

3 Dravet Syndrome - Company wise Pipeline Analysis
3.1 Biscayne Pharmaceuticals Inc Pipeline, H1 2018
3.2 Epygenix Therapeutics Inc Pipeline, H1 2018
3.3 Gw Pharmaceuticals Plc Pipeline, H1 2018
3.4 Insys Therapeutics Inc Pipeline, H1 2018
3.5 Lundbeck Inc Pipeline, H1 2018
3.6 Opko Health Inc Pipeline, H1 2018
3.7 Praxis Precision Medicines Pipeline, H1 2018
3.8 Ptc Therapeutics Inc Pipeline, H1 2018
3.9 Sage Therapeutics Inc Pipeline, H1 2018
3.10 Takeda Pharmaceutical Company Ltd Pipeline, H1 2018
3.11 Xenon Pharmaceuticals Inc Pipeline, H1 2018
3.12 Zogenix Inc Pipeline, H1 2018

4 Dravet Syndrome Pipeline Drug Snapshots
4.1 BIS - 001 Drug Details
4.1.1 Snapshot
4.1.2 Drug Overview
4.1.3 Mechanism of Action
4.1.4 Current Status
4.1.5 Pre - Clinical/ Discovery Phase Overview
4.1.6 Phase 1 Overview
4.2 Epidiolex Drug Details
4.2.1 Snapshot
4.2.2 Drug Overview
4.2.3 Current Status
4.2.4 Mechanism of Action
4.2.5 Phase 3 overview
4.3 Synthetic Cannabidiol Drug Details
4.3.1 Snapshot
4.3.2 Drug Overview
4.3.3 Current Status
4.3.4 Mechanism of Action
4.3.5 Phase 3 Overview
4.4 Antagonat Drug Details
4.4.1 Snapshot
4.4.2 Drug Overview
4.4.3 Current Stauts
4.4.4 Mechanism of Action
4.4.5 Pre - clinical Trial Details
4.4.6 Phase 2 Trial details
4.5 Ataluren Drug Details
4.5.1 Snapshot
4.5.2 Drug Overview
4.5.3 Current Status
4.5.4 Phase 2 Trial Details
4.6 SAGE - 217 Drug Details
4.6.1 Snapshot
4.6.2 Drug Overview
4.6.3 Mechanism of Action
4.6.4 Current Status
4.6.5 Pre - clinical Stage
4.7 SGE - 516 Drug Details
4.7.1 Snapshot
4.7.2 Drug Overview
4.7.3 Mechanism of Action
4.7.4 Current Status
4.7.5 Pre - Clinical Study
4.8 NAV1.6 Sodium Channel Drug Details
4.8.1 Snapshot
4.8.2 Drug Overview
4.8.3 Current Status
4.8.4 Mechanism of Action
4.8.5 Pre - Clinical Phase
4.9 ZX - 008 Drug Details
4.9.1 Snapshot
4.9.2 Drug Overview
4.9.3 Current Status
4.9.4 Mechanism of Action
4.9.5 Ongoing Trials
4.10 TAK 935 Drug Details
4.10.1 Snapshot
4.10.2 Drug Overview
4.10.3 Current Status
4.10.4 Mechanism of Action
4.10.5 Pre - clinical phase
4.11 EPX 300 Drug Details
4.11.1 Snapshot
4.11.2 Drug Overview
4.11.3 Mechanism of Action
4.11.4 Current Status
4.11.5 Pre Clinical Phase Details
4.12 EPX – 100 Drug Details
4.12.1 Snapshot
4.12.2 Drug Overview
4.12.3 Mechanism of Action
4.12.4 Current Status
4.13 EPX - 200 Drug Details
4.13.1 Snapshot
4.13.2 Drug Overview
4.14 Clobazam Drug Details
4.14.1 Snapshot
4.14.2 Drug Overview
4.14.3 Mechanism of Action
4.14.4 Current Status

5 Recent Developments in Thyroid Cancer Pipeline
5.1 Zogenix, Inc Completes Enrollment Of Its Phase 3 Trial - January 2018
5.2 Ema Accepts Epidiolex’ Marketing Authorization Application For Review - February 2018
5.3 Zx008 Shows Successful Results In Phase 3 Trial - December 2017
5.4 Epygenix Awarded Orphan Drug Designation For Its Epx - 300 Drug - August 2017

6 Appendix
6.2 Sources And Research Methodology

1.1 List of Figures
Figure 1: Dravet Syndrome Pipeline by Phase, H1 - 2018
Figure 2: Dravet Syndrome Pipeline by Companies, H1 - 2018
Figure 3: Company wise Pipeline Drug Phases, H1 - 2018
Figure 4: Dravet Syndrome Pipeline by Mechanism of Action, H1 - 2018
Figure 5: Mechanism wise Pipeline Drug Phases, H1 - 2018

1.2 List of Tables
Table 1: Dravet Syndrome Pipeline by Phase, H1 - 2018
Table 2: Dravet Syndrome Pipeline by Companies, H1 - 2018
Table 3: Dravet Syndrome Pipeline by Mechanism of Action, H1 - 2018
Table 4: Biscayne Pharmaceuticals Inc - Dravet Syndrome Pipeline, H1 - 2018
Table 5: Epygenix Therapeutics Inc - Dravet Syndrome Pipeline, H1 - 2018
Table 6: GW Pharmaceuticals Plc - Dravet Syndrome Pipeline, H1 - 2018
Table 7: INSYS Therapeutics Inc - Dravet Syndrome Pipeline, H1 - 2018
Table 8: Lundbeck Inc - Dravet Syndrome Pipeline, H1 - 2018
Table 9: OPKO Health Inc - Dravet Syndrome Pipeline, H1 - 2018
Table 10: Praxis Precision Medicines - Dravet Syndrome Pipeline, H1 - 2018
Table 11: PTC Therapeutics Inc - Dravet Syndrome Pipeline, H1 - 2018
Table 12: Sage Therapeutics Inc - Dravet Syndrome Pipeline, H1 - 2018
Table 13: Takeda Pharmaceutical Company Ltd - Dravet Syndrome Pipeline, H1 - 2018
Table 14: Xenon Pharmaceuticals Inc - Dravet Syndrome Pipeline, H1 - 2018
Table 15: Zogenix Inc - Dravet Syndrome Pipeline, H1 - 2018 21

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FEATURED COMPANIES

  • Biscayne Pharmaceuticals
  • Epygenix Therapeutics
  • INSYS Therapeutics Inc
  • OPKO Health Inc
  • Ovid Therapeutics; Takeda
  • Xenon Pharmaceuticals Inc
  • MORE

Dravet syndrome is a genetically - modified seizure condition associated with extended, frequent refractory seizures. The condition is also called as severe myoclonic epilepsy of infancy (SMEI). Dravet Syndrome occurs due to defects in SCN1A genes in brain and causes cognitive and behavioral disorders and around 15- 20% death rate.

Seizures start at the age of one, and are frequently associated with febrile convulsions. Subsequently, multiple types of seizures occur such as epilepticus. After the age 2, baby shows signs of deteriorating cognitive and behavioral skills, resulting in long term disability.

US FDA has not approved any drugs and EMA has approved only one drug. Treatment options include Clobazam plus valporate plus stiripentol, which offers only a modest seizure control.

Lack of approved complete treatments for the disease has spurred interest among pharmaceutical companies, in specific over the past few years. As of February 2018, the Dravet syndrome pipeline consists of at least 15 drug candidates, of which 4 are in late stage. Of this, 10 drugs have been given Orphan Drug Designations. This research work reviews the current status of Dravet Syndrome drug development pipeline.

Dravet Syndrome pipeline comprises of 15 drugs under development as of February 2018. Of these, 4 drugs are in Phase 1 and 4 are in phase 2. Further, 3 drugs remain in Phase 3 while one drug has moved into Pre-registration phase. In addition, 3 drugs are in Pre-Clinical Stage.

23 companies/ institutions are developing the pipeline for Dravet Sysndrome.

Note: Product cover images may vary from those shown
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  • Biscayne Pharmaceuticals
  • Brabant Pharma Limited
  • California Pacific Medical Center
  • Epygenix Therapeutics
  • GW Pharmaceuticals; University of Alabama; University of Utah
  • Harvard University
  • INSYS Therapeutics Inc
  • Lundbeck Inc
  • Northwestern University Feinberg School of Medicine; Praxis Precision Medicines
  • OPKO Health Inc
  • OVATION Pharmaceuticals
  • Ovid Therapeutics; Takeda
  • PTC Therapeutics; Sanofi Genzyme
  • Sage Therapeutics Inc
  • The University of Montreal Hospital Research Center; University of Chicago
  • University of California; University of Colorado
  • Xenon Pharmaceuticals Inc
  • Zogenix Inc
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