Jammu Journal

Dravet Syndrome Market

The Dravet Syndrome market growth is driven by factors like increase in the prevalence of Dravet Syndrome, investments in research and development, entry of emerging therapies during the study period 2019-2032.

The Dravet Syndrome market report also offers comprehensive insights into the Dravet Syndrome market size, share, Dravet Syndrome epidemiology, emerging therapies, market drivers and barriers, ongoing clinical trials, key collaboration in the space, market uptake by key therapies and companies actively pushing Dravet Syndrome market size growth forward. 

Some of the key highlights from the Dravet Syndrome Market Insights Report:

• Several key pharmaceutical companies, including Takeda, Epygenix Therapeutics, Stoke Therapeutics, Longboard Pharmaceuticals, Encoded Therapeutics, Eisai, Virpax Pharmaceuticals, and others, are developing novel products to improve the Dravet Syndrome treatment outlook. 
• The total Dravet Syndrome market size will include the market size of the potential upcoming therapies and current treatment regimens in the seven major markets.
• As per DelveInsight analysis, the Dravet Syndrome market is anticipated to witness growth at a considerable CAGR

Strategise your business goals by understanding market dynamics @ Dravet Syndrome Market Landscape

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.

Do you know the treatment paradigms for different countries? Download our Dravet Syndrome Market Sample Report

Dravet Syndrome Epidemiology Segmentation 

DelveInsight’s Dravet Syndrome market report is prepared on the basis of epidemiology model. It offers comprehensive insights to the Dravet Syndrome historical patient pools and forecasted Dravet Syndrome patients. The report provides in-depth data of various subtypes and for the same epidemiology is segmented further. The Dravet Syndrome Market report proffers epidemiological analysis for the study period 2019-32 in the 7MM segmented into:

• Dravet Syndrome Prevalence 
• Age-Specific Dravet Syndrome Prevalence 
• Gender-Specific Dravet Syndrome Prevalence 
• Diagnosed and Treatable Cases of Dravet Syndrome

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Dravet Syndrome Treatment Market 

The Dravet Syndrome market outlook of the report helps to build a detailed comprehension of the historic, current, and forecasted Dravet Syndrome market trends by analyzing the impact of current Dravet Syndrome therapies on the market, unmet needs, drivers and barriers, and demand for better technology.

This segment gives a thorough detail of Dravet Syndrome market trend of each marketed drug and late-stage pipeline therapy by evaluating their impact based on the annual cost of therapy, inclusion and exclusion criteria, mechanism of action, compliance rate, growing need of the market, increasing patient pool, covered patient segment, expected launch year, competition with other therapies, brand value, their impact on the market and view of the key opinion leaders. The calculated Dravet Syndrome market data are presented with relevant tables and graphs to give a clear view of the market at first sight.

According to DelveInsight, the Dravet Syndrome market in 7MM is expected to witness a major change in the study period 2019-2032.

Dravet Syndrome Emerging Drugs

Soticlestat : Takeda

Soticlestat is a potent, highly selective, oral, first-in-class inhibitor of the enzyme cholesterol 24-hydroxylase (CH24H), with the potential to reduce seizure susceptibility and improve seizure control. CH24H is predominantly expressed in the brain, where it converts cholesterol into 24S-hydroxycholesterol (24HC) to adjust the homeostatic balance of brain cholesterol. 24HC is a positive allosteric modulator of the NMDA receptor and modulates glutamatergic signaling associated with epilepsy. Glutamate is one of the main neurotransmitters in the brain and has been shown to play a role in the initiation and spread of seizure activity. Recent literature indicates that CH24H is involved in over-activation of the glutamatergic pathway through modulation of the NMDA channel and that increased expression of CH24H can disrupt the reuptake of glutamate by astrocytes, resulting in epileptogenesis and neurotoxicity. Inhibition of CH24H by soticlestat reduces the neuronal levels of 24HC and may improve the excitatory/inhibitory balance of NMDA channel activity. It is being investigated by Ovid and Takeda for the treatment of rare developmental and epileptic encephalopathies (DEEs), a group of highly refractory epilepsy syndromes including Dravet Syndrome and LGS. Soticlestat is in the Phase III stage of development for the treatment of Dravet Syndrome.

EPX-100: Epygenix Therapeutics

EPX-100 is a repurposed antihistamine that was used in the past to treat itchiness. The medication can suppress seizures via modulation of serotonin (5-HT) signaling pathways, which is different from its anti-histaminic properties. EPX-100 was a powerful suppressor of spontaneous convulsive behavior and electrographic seizures in zebrafish disease models for Dravet syndrome. EPX-100’s antiepileptic action is not through a histaminergic mechanism of action but acts via modulation of serotonin (5-HT) signaling pathways. Currently, the drug is in Phase II trial as a single pivotal study to compare the efficacy of EPX-100 against placebo as adjunctive therapy in patients with Dravet Syndrome.

STK-001: Stoke Therapeutics

STK-001 is an investigational new medicine for the treatment of Dravet syndrome. It is a proprietary antisense oligonucleotide (ASO) and has the potential to be the first disease-modifying therapy to address the genetic cause of Dravet syndrome. The drug is designed to upregulate NaV1.1 protein expression by leveraging the non-mutant copy of the SCN1A gene to restore physiological NaV1.1 levels, thereby reducing both the occurrences of seizures and significant non-seizure comorbidities. This RNA-based approach is not gene therapy but rather RNA modulation, as it does not manipulate nor insert genetic deoxyribonucleic acid (DNA). The drug is currently in Phase II clinical trial for the treatment of patients with Dravet Syndrome.

LP352: Longboard Pharmaceuticals

LP352 is an oral, centrally acting 5-HT2c superagonist in development for the potential treatment of seizures associated with DEEs such as Dravet syndrome, LGS, tuberous sclerosis complex (TSC), CDKL5 deficiency disorder (CDD), and other epileptic disorders. LP352 is designed to modulate GABA and, as a result, suppress the central hyperexcitability that is characteristic of seizures. LP352 is the only 5-HTc receptor superagonist being dose-optimized for the refractory epilepsy population. LP352 also demonstrated statistically significant reductions in both epileptiform event frequency and duration – reducing the frequency of epileptiform events by 85% and the cumulative duration of epileptiform events by 84% in the SCN1lab zebrafish model of Dravet syndrome.

Dravet Syndrome Key Companies

• Takeda
• Epygenix Therapeutics
• Stoke Therapeutics
• Longboard Pharmaceuticals
• Encoded Therapeutics
• Eisai
• Virpax Pharmaceuticals

For more information, visit Dravet Syndrome Market Analysis, Patient Pool, and Emerging Therapies

Scope of the Dravet Syndrome Market Report:

• 11 Years Forecast
• 7MM Coverage 
• Descriptive overview of Dravet Syndrome, causes, signs and symptoms, diagnosis, treatment
• Comprehensive insight into Dravet Syndrome epidemiology in the 7MM
• Dravet Syndrome marketed and emerging therapies 
• Dravet Syndrome companies
• Dravet Syndrome market drivers and barriers 

Key Questions Answered in the Dravet Syndrome Market Report 2032:

• What was the Dravet Syndrome market share distribution in 2019, and how would it appear in 2032?
• What is the total Dravet Syndrome market size and the market size by therapy across the 7MM for the study period (2019-32)?
• What are the important findings from 7MM, and which country will have the greatest Dravet Syndrome market size from 2019-32?
• During the study period (2019-2032), at what CAGR is the Dravet Syndrome market projected to expand at 7MM?

Table of Contents:

1 Dravet Syndrome Market Key Comprehensive Insights 

2 Dravet Syndrome Market Report Introduction

3 Competitive Intelligence Analysis for Dravet Syndrome

4 Dravet Syndrome Market Analysis Overview at a Glance

5 Executive Summary of Dravet Syndrome

6 Dravet Syndrome Epidemiology and Market Methodology

7 Dravet Syndrome Epidemiology and Patient Population

8 Dravet Syndrome Patient Journey

9 Dravet Syndrome Treatment Algorithm, Dravet Syndrome Current Treatment, and Medical Practices

10 Key Endpoints in Dravet Syndrome Clinical Trials

11 Dravet Syndrome Marketed Therapies 

12 Dravet Syndrome Emerging Therapies

13 Dravet Syndrome: 7 Major Market Analysis

14 Attribute analysis

15 Access and Reimbursement Overview of Dravet Syndrome

16 Dravet Syndrome Market Key Opinion Leaders Reviews

18 Dravet Syndrome Market Drivers

19 Dravet Syndrome Market Barriers

20 SWOT Analysis

21 Disclaimer

22 DelveInsight Capabilities

23 About DelveInsight

Related Reports:

Dravet Syndrome Epidemiology 2032

DelveInsight’s “Dravet Syndrome – Epidemiology Forecast to 2032” report delivers an in-depth understanding of the disease, historical and forecasted Dravet Syndrome epidemiology in the 7MM, i.e., the United States, EU5 (Germany, Spain, Italy, France, and the United Kingdom), and Japan.

Dravet Syndrome Pipeline 2023

“Dravet Syndrome Pipeline Insights, 2023” report by DelveInsight outlines comprehensive insights of present clinical development scenarios and growth prospects across the Dravet Syndrome market. A detailed picture of the Dravet Syndrome pipeline landscape is provided, which includes the disease overview and Dravet Syndrome treatment guidelines. 

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