
Dravet Syndrome- Pipeline Insight, 2024
Description
Dravet Syndrome- Pipeline Insight, 2024
DelveInsight’s, “Dravet Syndrome- Pipeline Insight, 2024” report provides comprehensive insights about 12+ companies and 16+ pipeline drugs in Dravet Syndrome pipeline landscape. It covers the pipeline drug profiles, including clinical and nonclinical stage products. It also covers the therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.
Geography Covered
- Global coverage
Dravet Syndrome: Overview
Dravet Syndrome is a severe and progressive genetic epilepsy characterized by frequent, prolonged, and refractory seizures that usually begin within the first year of life. Dravet Syndrome is classified as a developmental and epileptic encephalopathy due to the developmental delays and cognitive impairment, in addition to seizure activity, that stem from the genetic mutation that causes the disease. The voltage-gated sodium channel is responsible for the initiation of action potentials and, therefore, is involved in neuronal excitability. The alpha subunit has four homologous domains with six transmembrane segments each that form the voltage sensor and ion-conducting pore. Mutations cause either a gain or a loss of function. Initially, researchers could not explain how loss-of-function mutations could lead to seizures. A mouse model of DS showed selective loss of sodium current in the hippocampal γ-aminobutyric acid–mediated inhibitory interneurons. Failure of inhibition leading to excitation is a proposed pathogenesis of this mutation in DS.
Approximately 85% of those diagnosed with Dravet Syndrome have a mutation of the SCN1A gene. Dravet Syndrome is not usually caused by an inherited mutation. In 90% of these patients, the mutation is not found in the patient’s parents Dravet Syndrome can be diagnosed, if a patient exhibits specific symptoms, genetic testing may be done via an epilepsy panel, to look for SCN1A and other genes commonly associated with epilepsy. However, the effects of Dravet syndrome are not limited to seizures, developmental delays, movement and balance issues and also language and speech disturbances. Dravet syndrome has a high rate of premature death due to the severity of this type of epilepsy. Up to 20% of children and adolescents living with Dravet die before adulthood.
The correct diagnosis of DS and appropriate follow-up are typically delayed. The EEG is normal at onset, and neuroimaging reveals no structural lesion. Early development is normal, but signs of regression appear in the second year of life and are often accompanied by convulsive status epilepticus, alternating hemiconvulsions, and myoclonic seizures. The diagnosis can be confirmed by genetic testing that is now available and shows mutations within the SCN1A gene. Early recognition and diagnosis of DS and management with appropriate anticonvulsants and a treatment plan may reduce the seizure burden and improve the long-term developmental outcome.
""Dravet Syndrome- Pipeline Insight, 2024"" report by DelveInsight outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Dravet Syndrome pipeline landscape is provided which includes the disease overview and Dravet Syndrome treatment guidelines. The assessment part of the report embraces, in depth Dravet Syndrome commercial assessment and clinical assessment of the pipeline products under development. In the report, detailed description of the drug is given which includes mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, Dravet Syndrome collaborations, licensing, mergers and acquisition, funding, designations and other product related details.
Report Highlights
- The companies and academics are working to assess challenges and seek opportunities that could influence Dravet Syndrome R&D. The therapies under development are focused on novel approaches to treat/improve Dravet Syndrome.
This segment of the Dravet Syndrome report encloses its detailed analysis of various drugs in different stages of clinical development, including phase II, I, preclinical and Discovery. It also helps to understand clinical trial details, expressive pharmacological action, agreements and collaborations, and the latest news and press releases.
Dravet Syndrome Emerging Drugs
- Soticlestat : Takeda
- STK-001: Stoke Therapeutics
- LP352: Longboard Pharmaceuticals
Further product details are provided in the report……..
Dravet Syndrome: Therapeutic Assessment
This segment of the report provides insights about the different Dravet Syndrome drugs segregated based on following parameters that define the scope of the report, such as:
- Major Players in Dravet Syndrome
- There are approx. 12+ key companies which are developing the therapies for Dravet Syndrome. The companies which have their Dravet Syndrome drug candidates in the most advanced stage, i.e. phase III include, Takeda.
- Phases
- Late stage products (Phase III)
- Mid-stage products (Phase II)
- Early-stage product (Phase I) along with the details of
- Pre-clinical and Discovery stage candidates
- Discontinued & Inactive candidates
- Route of Administration
- Oral
- Intravenous
- Subcutaneous
- Parenteral
- Topical
- Molecule Type
- Recombinant fusion proteins
- Small molecule
- Monoclonal antibody
- Peptide
- Polymer
- Gene therapy
- Product Type
Dravet Syndrome: Pipeline Development Activities
The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Dravet Syndrome therapeutic drugs key players involved in developing key drugs.
Pipeline Development Activities
The report covers the detailed information of collaborations, acquisition and merger, licensing along with a thorough therapeutic assessment of emerging Dravet Syndrome drugs.
Dravet Syndrome Report Insights
- Dravet Syndrome Pipeline Analysis
- Therapeutic Assessment
- Unmet Needs
- Impact of Drugs
- Pipeline Product Profiles
- Therapeutic Assessment
- Pipeline Assessment
- Inactive drugs assessment
- Unmet Needs
Current Treatment Scenario and Emerging Therapies:
- How many companies are developing Dravet Syndrome drugs?
- How many Dravet Syndrome drugs are developed by each company?
- How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Dravet Syndrome?
- What are the key collaborations (Industry–Industry, Industry–Academia), Mergers and acquisitions, licensing activities related to the Dravet Syndrome therapeutics?
- What are the recent trends, drug types and novel technologies developed to overcome the limitation of existing therapies?
- What are the clinical studies going on for Dravet Syndrome and their status?
- What are the key designations that have been granted to the emerging drugs?
- Takeda
- Epygenix Therapeutics
- Stoke Therapeutics
- Longboard Pharmaceuticals
- Encoded Therapeutics
- Eisai
- Virpax Pharmaceuticals
- Soticlestat
- EPX-100
- STK-001
- LP352
- ETX101
- Lorcaserin
- VRP324
Table of Contents
60 Pages
- Introduction
- Executive Summary
- Dravet Syndrome: Overview
- Causes
- Mechanism of Action
- Signs and Symptoms
- Diagnosis
- Disease Management
- Pipeline Therapeutics
- Comparative Analysis
- Therapeutic Assessment
- Assessment by Product Type
- Assessment by Stage and Product Type
- Assessment by Route of Administration
- Assessment by Stage and Route of Administration
- Assessment by Molecule Type
- Assessment by Stage and Molecule Type
- Dravet Syndrome– DelveInsight’s Analytical Perspective
- Late Stage Products (Phase III)
- Comparative Analysis
- Soticlestat : Takeda
- Product Description
- Research and Development
- Product Development Activities
- Drug profiles in the detailed report…..
- Mid Stage Products (Phase II)
- Comparative Analysis
- EPX-100: Epygenix Therapeutics
- Product Description
- Research and Development
- Product Development Activities
- Drug profiles in the detailed report…..
- Early Stage Products (Phase I)
- Comparative Analysis
- Drug name: Company name
- Product Description
- Research and Development
- Product Development Activities
- Drug profiles in the detailed report…..
- Preclinical and Discovery Stage Products
- Comparative Analysis
- Drug name: Company name
- Product Description
- Research and Development
- Product Development Activities
- Drug profiles in the detailed report…..
- Inactive Products
- Comparative Analysis
- Dravet Syndrome Key Companies
- Dravet Syndrome Key Products
- Dravet Syndrome- Unmet Needs
- Dravet Syndrome- Market Drivers and Barriers
- Dravet Syndrome- Future Perspectives and Conclusion
- Dravet Syndrome Analyst Views
- Dravet Syndrome Key Companies
- Appendix
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