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 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.
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.
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.
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.
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Report Coverage
The Tuberous Sclerosis Drug Pipeline Insight Report by the publisher 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 Drug Pipeline Outlook
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.
Tuberous Sclerosis - Drug Pipeline Therapeutic Assessment
This section of the report covers the analysis of vasculitis drugs based on several segmentations including:By Phase
- Late-Stage Products (Phase 3 and Phase 4)
- Mid-Stage Products (Phase 2)
- Early-Stage Products (Phase I)
- Preclinical and Discovery Stage Products
By Molecule Type
- Bispecific Antibody
- Small Molecule
- Peptides
- Gene Therapy
By Route of Administration
- Oral
- Parenteral
- Others
Tuberous Sclerosis - Pipeline Assessment Segmentation, By Phases
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 analysis, phase II covers a major share of the total clinical trials for tuberous sclerosis.Tuberous Sclerosis - Pipeline Assessment Segmentation, By Drug Classes
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.Tuberous Sclerosis Clinical Trials Therapeutic Assessment - Competitive Dynamics
The 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:- Jazz Pharmaceuticals
- Novartis Pharmaceuticals
- GRIN Therapeutics, Inc.
- Noema Pharma AG
- Marinus Pharmaceuticals
- Neurim Pharmaceuticals Ltd.
- Aadi Bioscience, Inc.
- Takeda
- Reveal Pharmaceuticals Inc.
- Nobelpharma
Tuberous Sclerosis - Emerging Drugs Profile
The leading drugs in the pipeline are as follows:
Drug: Cannabidiol Oral Solution [Epidiolex]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.
Drug: Everolimus
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.Drug: Ganaxalone
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.Drug: Basimglurant (NOE-101
)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.
Reasons To Buy This 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.Key Questions Answered in the Tuberous Sclerosis - Pipeline Insight Report
- What is the current landscape of tuberous sclerosis pipeline drugs?
- How many companies are developing tuberous sclerosis drugs?
- How many phase III and phase IV drugs are currently present in tuberous sclerosis pipeline drugs?
- Which companies/institutions are leading the tuberous sclerosis drug development?
- What is the efficacy and safety profile of tuberous sclerosis pipeline drugs?
- What are the opportunities and challenges present in the tuberous sclerosis drug pipeline landscape?
- Which company is conducting major trials for tuberous sclerosis drugs?
- What geographies are covered for clinical trials in tuberous sclerosis?
- What are emerging trends in tuberous sclerosis clinical trials?
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Table of Contents
1 Preface
3 Overview of Tuberous Sclerosis Pipeline
4 Patient Profile: Tuberous Sclerosis Pipeline
5 Tuberous Sclerosis Pipeline: Epidemiology Snapshot
6 Tuberous Sclerosis Pipeline: Market Dynamics
7 Tuberous Sclerosis Pipeline: Key Facts Covered
8 Tuberous Sclerosis Pipeline, Drug Pipeline Assessment
9 Drug Pipeline Comparative Analysis
10 Tuberous Sclerosis Drug Pipeline - Late-Stage Products (Phase III and IV) (Top Drugs)
11 Tuberous Sclerosis Drug Pipeline - Mid-Stage Products (Phase II) (Top Drugs)
12 Tuberous Sclerosis Drug Pipeline - Early-Stage Products (Phase I) (Top Drugs)
13 Tuberous Sclerosis Drug Pipeline - Preclinical and Discovery Stage Products (Top Drugs)
14 Tuberous Sclerosis, Key Drug Pipeline Companies
