Developmental and epileptic encephalopathies (DEEs) include EEG abnormalities, developmental delays, and early-onset epilepsy are the hallmarks of severe illnesses. These disorders are frequently brought on by genetic abnormalities that alter brain anatomy, synaptic transmission, or neuronal signaling. Dravet syndrome and West syndrome are examples of DEEs, in which seizures exacerbate developmental deficits. Surgery, antiepileptic medications, ketogenic diets, and precision medicine are all used in treatment. DEEs have a dismal prognosis and are frequently resistant to conventional treatments, even with interventions. The Developmental and Epileptic Encephalopathy pipeline analysis by Expert Market Research focuses on various treatment options for this disease.
Major companies involved in the developmental and epileptic encephalopathy pipeline analysis include Neurocrine Biosciences, and Takeda among others.
Leading drugs currently in the pipeline include NBI-921352, and REGN5381, others.
Increased investment in research and development, along with regulatory support, is accelerating clinical trials and new treatment approvals.
The Developmental and Epileptic Encephalopathy Pipeline Analysis Report by Expert Market Research gives comprehensive insights into developmental and epileptic encephalopathy therapeutics currently undergoing clinical trials. It covers various aspects related to the details of each of these drugs under development for Developmental and Epileptic Encephalopathy therapeutics. The developmental and epileptic encephalopathy report assessment includes the analysis of over 50 pipeline drugs and 25+ companies. The developmental and epileptic encephalopathy 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 developmental and epileptic encephalopathy 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 developmental and epileptic encephalopathy.
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Genetic abnormalities affecting ion channels, synaptic proteins, and neuronal signaling pathways are part of the pathophysiology of developmental and epileptic encephalopathy (DEE). These disturbances impede synaptic transmission, plasticity, and neuronal excitability by causing aberrant cortical and subcortical connections. Defects in metabolism or structural brain abnormalities may also be involved. By changing intracortical connections throughout crucial developmental stages, the ensuing epileptiform activity intensifies cognitive delays, leading to extensive cortical dysfunction and serious neurodevelopmental abnormalities.
Developmental and epileptic encephalopathy (DEE) is treated using a variety of strategies depending on the type of seizure and its underlying cause. Anti-seizure drugs are common tactics, however, many DEEs are resistant to drugs. Vagus nerve stimulation (VNS), ketogenic diets, and surgical procedures for focal seizures are alternatives. Emerging alternatives include gene-targeted medicines, repurposed medications like fenfluramine, and precision therapeutics like cannabidiol. These seek to enhance general quality of life and seizure control.
Each illness and population has a different prevalence of developmental and epileptic encephalopathy (DEE). DEE has a cumulative incidence of 169 per 100,000 children with infantile epileptic spasms syndrome is the most prevalent at 58.2 per 100,000 children. Other syndromes such as Lennox-Gastaut syndrome and epilepsy with myoclonic-atonic seizures are less common. Genetic factors that contribute significantly to the prevalence of DEE include mutations in SCN1A and CDKL5.
This section of the report covers the analysis of developmental and epileptic encephalopathy drug candidates based on several segmentations, including:
By Phase
By Drug Class
By 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 covers a major share of the total developmental and epileptic encephalopathy clinical trials.
In the developmental and epileptic encephalopathy pipeline, majority of the candidates are in Phase II demonstrating progress toward potential treatments.
The drug molecule categories covered under the developmental and epileptic encephalopathy pipeline analysis include monoclonal antibody, peptides, small molecule and gene therapy. The developmental and epileptic encephalopathy report provides a comparative analysis of the drug classes for each drug in various phases of clinical trials for Developmental and Epileptic Encephalopathy.
The EMR report for the developmental and epileptic encephalopathy pipeline analysis covers the profile of key companies involved in clinical trials and their drugs under development. Below is the list of a few players involved in developmental and epileptic encephalopathy 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 developmental and epileptic encephalopathy. It includes product description, trial ID, study type, drug class, mode of administration, and recruitment status of developmental and epileptic encephalopathy drug candidates.
NBI-921352, developed by Neurocrine Biosciences, is an investigational selective NaV1.6 inhibitor aimed at treating SCN8A developmental and epileptic encephalopathy (SCN8A-DEE), a rare and severe form of epilepsy. A Phase 2 randomized, double-blind, placebo-controlled study is underway to assess its efficacy, safety, and pharmacokinetics as adjunctive therapy in patients aged 2 to 21 with SCN8A-DEE. The trial began in January 2022 and is expected to complete by December 2025.
Soticlestat, developed by Takeda, is an investigational, first-in-class inhibitor of cholesterol 24-hydroxylase (CH24H). It is an enzyme primarily expressed in the brain that catabolizes cholesterol to 24-S hydroxycholesterol (24HC), resulting in a reduction in glutamatergic hyperexcitability. In the Phase 2 ELEKTRA study, soticlestat achieved its primary endpoint, demonstrating a statistically significant reduction in seizure frequency in children with Dravet syndrome (DS) or Lennox-Gastaut syndrome (LGS).
*Please note that this is only a partial list; the complete list of drugs will be available in the full report.*
The Developmental and Epileptic Encephalopathy Drug Pipeline Analysis Report provides a strategic overview of the latest and future landscape of treatments for Developmental and Epileptic Encephalopathy. 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 developmental and epileptic encephalopathy collaborations, regulatory environments, and potential growth opportunities.
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*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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