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Dravet syndrome is a rare, severe genetic epilepsy beginning in infancy, with global incidence estimated at 1 in 15,400 to 1 in 40,900 live births. Its overall prevalence ranges between 1.5 and 6.5 per 100,000 individuals, underscoring its orphan-disease status and diagnostic challenges. This low-frequency yet high-burden profile continues to shape research priorities and therapeutic innovation. These epidemiological insights form a critical foundation for understanding treatment needs within the Dravet syndrome pipeline.
Major companies involved in the Dravet syndrome pipeline analysis include Stoke Therapeutics, Inc., UCB BIOSCIENCES, Inc., and others.
Leading drugs currently in the pipeline include ETX101 and others.
Growth in the Dravet syndrome pipeline is driven by accelerating progress in genetic correction therapies, particularly candidates like antisense oligonucleotides designed to restore NaV1.1 expression. The FDA’s Breakthrough Therapy Designation for zorevunersen underscores rising confidence in disease-modifying approaches, encouraging intensified investment and development of next-generation precision therapeutics.
The Dravet Syndrome Pipeline Analysis Report by Expert Market Research gives comprehensive insights into Dravet syndrome therapeutics currently undergoing clinical trials. It covers various aspects related to the details of each of these drugs under development for Dravet syndrome. The Dravet syndrome report assessment includes the analysis of over 100 pipeline drugs and 50+ companies. The Dravet syndrome pipeline landscape will include an analysis based on efficacy and safety measure outcomes published for the trials, including their adverse effects on patients suffering from the condition, and alignment with Dravet syndrome treatment guidelines to ensure optimal care practices.
The assessment part will include a detailed analysis of each drug, drug class, clinical studies, phase type, drug type, route of administration, and ongoing product development activities related to Dravet syndrome.

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The Dravet syndrome pipeline continues to advance as research focuses on addressing the disorder’s underlying genetic cause rather than only managing seizures. Current treatments rely on anti-seizure medications, yet unmet needs remain significant. In December 2024, Stoke Therapeutics received FDA Breakthrough Therapy Designation for zorevunersen, a proprietary antisense oligonucleotide designed to upregulate NaV1.1 protein expression by leveraging the wild-type SCN1A gene. Early clinical data show sustained seizure reduction and cognitive improvement, positioning zorevunersen as a promising first disease-modifying therapy in ongoing trials.
Dravet syndrome is a rare but severe genetic epilepsy starting in infancy. Global estimates suggest DS occurs in approximately 1 in 15,400 to 1 in 40,900 live births, reflecting its low incidence but genetic origin. Among the general population, its prevalence ranges between 1.5 and 6.5 per 100,000 individuals, depending on study methodology and region. This rarity underscores DS’s classification as an orphan disease and highlights diagnostic challenges. Accurate epidemiologic tracking remains essential as new therapies emerge, to ensure timely diagnosis and optimize resource allocation worldwide.
This section of the report covers the analysis of Dravet syndrome drug candidates based on several segmentations, including:
By Phase
The pipeline assessment report covers 50+ drug analyses based on phase:
By Drug Class
The Dravet syndrome pipeline analysis report covers 50+ drug analyses based on drug classes:
By Route of Administration
The pipeline assessment report covers 50+ drug analyses based on the route of administration.
The report covers phase I, phase II, phase III, phase IV, and early-phase drugs. The coverage includes an in-depth analysis of each drug across these phases. According to EMR analysis, phase II accounts for 30% of total Dravet syndrome clinical trials, representing the largest share. Phase III follows with 35%, reflecting strong late-stage activity, while phase I contributes 20% and phase IV represents 15% of ongoing efforts. Early-phase programs remain limited, highlighting the current focus on mid- to late-stage development.
The drug molecule categories covered under the Dravet syndrome pipeline analysis include recombinant fusion proteins, small molecules, monoclonal antibodies, peptides, and polymers. The Dravet syndrome report provides a comparative assessment of these classes across all clinical phases, highlighting ongoing advancements in therapeutic innovation. In addition, in July 2022, the U.S. FDA expanded approval of DIACOMIT (stiripentol) for patients with Dravet syndrome as young as six months old when administered with clobazam, reflecting continued progress in broadening treatment access for younger patient populations.
The EMR report for the Dravet syndrome pipeline covers the profile of key companies involved in clinical trials and their drugs under development. It provides a detailed Dravet syndrome therapeutic assessment, analyzing the competitive dynamics of the clinical trial landscape. Below is the list of a few players involved in Dravet syndrome clinical trials:
This section covers the detailed analysis of each drug under multiple phases, including phase I, phase II, phase III, phase IV, and emerging drugs for Dravet syndrome. It includes product description, trial ID, study type, drug class, mode of administration, and recruitment status of Dravet syndrome drug candidates.
ETX101 is an investigational gene-regulation therapy designed for Dravet syndrome and belongs to the class of genetic neurodevelopmental disorder therapeutics. It works by increasing the expression of the SCN1A gene in inhibitory interneurons, aiming to restore Nav1.1 channel activity and reduce the frequency and severity of seizures. ETX101 uses an AAV-based delivery system to target specific neuronal populations. Encoded Therapeutics, the company developing ETX101, focuses on precision gene therapies that modulate gene expression rather than replace defective genes.
STK-001 is an antisense oligonucleotide (ASO) therapy candidate for Dravet syndrome and belongs to the class of oligonucleotide-based therapeutics. It functions by increasing production of the Nav1.1 protein from the healthy SCN1A allele, improving inhibitory neuronal function and stabilizing seizure activity. By enhancing endogenous gene expression, STK-001 aims to address the underlying pathology rather than only symptoms. Stoke Therapeutics, the developer, specializes in RNA-based medicines that boost protein output through their proprietary TANGO platform.
LP352 is an oral, centrally acting serotonin 5-HT2c receptor superagonist belonging to the small-molecule therapeutic class. It is designed to modulate neuronal excitability by selectively activating pathways that reduce seizure activity in Dravet syndrome and other developmental and epileptic encephalopathies. LP352 aims to improve seizure control with fewer off-target effects due to its high receptor specificity. Longboard Pharmaceuticals, the company behind LP352, focuses on next-generation neurology therapeutics derived from advanced serotonin receptor modulation technologies.
*Please note that this is only a partial list; the complete list of drugs will be available in the full report.*
The Dravet Syndrome Pipeline Analysis Report provides a strategic overview of the latest and future landscape of treatments for Dravet syndrome. It provides necessary information for making informed investment decisions along with research, development, and strategic planning efforts. The stakeholders will benefit from the essential insights into Dravet syndrome collaborations, regulatory environments, and potential growth opportunities.
*While we strive to always give you current and accurate information, the numbers depicted on the website are indicative and may differ from the actual numbers in the main report. At Expert Market Research, we aim to bring you the latest insights and trends in the market. Using our analyses and forecasts, stakeholders can understand the market dynamics, navigate challenges, and capitalize on opportunities to make data-driven strategic decisions.*
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