Tuberous sclerosis, caused by mutations in the genes TSC1 and TSC2, affects around 1 in 6000 to 1 in 10,000 live births. The overall prevalence of this rare genetic disorder is approximately 1 in 20,000. Currently, there is no cure for tuberous sclerosis and the available treatments are symptomatic and supportive including early development interventions for reducing the risk of developmental delays in the affected individual. This indicates the need for effective drugs for tuberous sclerosis that can address the root cause and offer long-term relief.
The Tuberous Sclerosis Drug Pipeline Insight Report by Expert Market Research gives comprehensive insights into tuberous sclerosis drugs currently undergoing clinical trials. It covers various aspects related to the details of each of these drugs under development for tuberous sclerosis. The report includes the analysis of over 100 pipeline drugs and 50+ companies. The tuberous sclerosis 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 tuberous sclerosis.
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 tuberous sclerosis.
Tuberous sclerosis or tuberous sclerosis complex refers to a rare condition that causes the development of non-cancerous tumors in many parts of the body. This rare multisystem autosomal dominant genetic disease is lifelong, with the prognosis for the patient highly variable and often depending on the severity of the symptoms. Common neurological symptoms of tuberous sclerosis include brain tumors, seizures, developmental delays, and behavioral problems, among others.
In April 2022, the U.S. Food and Drug Administration (FDA) granted approval to the use of HYFTOR™ (sirolimus topical gel) 0.2% for the treatment of facial angiofibromas (facial tumors) in adults and children (aged 6 years and above) affected by tuberous sclerosis. The topical gel HYFTOR, developed by Noblepharma, is the first FDA-approved treatment for facial angiofibromas associated with tuberous sclerosis complex. Further, the rising focus on the development of next-generation mTOR inhibitors and the growing interest in gene therapy to alter the disease progression are likely to positively influence the pipeline landscape of tuberous sclerosis in the coming years.
This section of the report covers the analysis of vasculitis drugs based on several segmentations including:
By Phase
By Molecule Type
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 clinical trials for tuberous sclerosis.
The drug molecules categories covered under tuberous sclerosis pipeline analysis include bispecific antibodies, peptides, small molecules, and gene therapy. The treatment depends on the type and severity of symptoms, the age of the patient, the affected organ, and the presence of genetic mutations, among others. The report provides a comparative analysis of the drug classes for each drug in various phases of clinical trials for tuberous sclerosis.
The EMR report for the tuberous sclerosis drug pipeline 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 tuberous sclerosis clinical trials:
The leading drugs in the pipeline are as follows:
The study is designed to assess the behavioral and other co-occurring outcomes of Epidolex (cannabidol oral solution) as an add-on treatment in patients with tuberous sclerosis complex who experience seizures. The trial is sponsored by Jazz Pharmaceuticals and is currently under phase IV.
The objective of the study is to investigate the long-term safety outcomes of everolimus in around 206 patients affected by tuberous sclerosis and refractory seizures. The trial is sponsored by Novartis Pharmaceuticals and is currently under phase III.
Sponsored by Marinus Pharmaceuticals, the placebo-controlled study of adjunctive ganaxolone therapy is currently under phase III. The randomized study has an estimated 128 children and adult participants with tuberous sclerosis complex-related epilepsy.
This Phase II double-blind, randomized, placebo-controlled study is conducted by Noema Pharma AG and aims to evaluate the efficacy and safety of basimglurant (NOE-101) in facilitating effective seizure control in participants with tuberous sclerosis complex.
*Please note that this is only a partial list; the complete list of drugs will be available in the full report.*
The Tuberous Sclerosis Drug Pipeline Insight Report provides a strategic overview of the latest and future landscape of treatments for tuberous sclerosis. 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 market trends, regulatory environments, and potential growth opportunities within tuberous sclerosis pipeline insights.
*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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