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Tuberous Sclerosis - Pipeline Insight, 2021

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    Clinical Trials

  • 60 Pages
  • March 2021
  • Region: Global
  • DelveInsight
  • ID: 5306341
This “Tuberous Sclerosis - Pipeline Insight, 2021,” report provides comprehensive insights about 7+ companies and 7+ pipeline drugs in Tuberous Sclerosis pipeline landscape. It covers the pipeline drug profiles, including clinical and nonclinical stage products. It also covers the therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.

Geography Covered

  • Global coverage

Tuberous Sclerosis Understanding

Tuberous Sclerosis: Overview

Tuberous Sclerosis is a rare form of cancer affecting the peritoneum, the thin membrane surrounding your abdominal organs. The causes of Tuberous Sclerosis are unknown. Primary Tuberous Sclerosis is more common in women than in men. The symptoms of Tuberous Sclerosis resemble those of ovarian cancer. Many of these symptoms are due to buildup of fluid (ascites) in the abdomen. Tuberous Sclerosis symptoms may include: nausea, loss of appetite, vomiting, abnormal vaginal bleeding, rectum bleeding, feeling of fullness after breath and shortness of breath. Physical exam, Ultrasound, medical history are some procedures followed up for the diagnosis of Tuberous Sclerosis. The treatment usually depends upon the size, location and stage of cancer.

"Tuberous Sclerosis - Pipeline Insight, 2021" report outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Tuberous Sclerosis pipeline landscape is provided which includes the disease overview and Tuberous Sclerosis treatment guidelines. The assessment part of the report embraces, in depth Tuberous Sclerosis commercial assessment and clinical assessment of the pipeline products under development. In the report, detailed description of the drug is given which includes mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, Tuberous Sclerosis collaborations, licensing, mergers and acquisition, funding, designations and other product related details.

Report Highlights

The companies and academics are working to assess challenges and seek opportunities that could influence Tuberous Sclerosis R&D. The therapies under development are focused on novel approaches to treat/improve Tuberous Sclerosis.

Tuberous Sclerosis Emerging Drugs Chapters

This segment of the Tuberous Sclerosis report encloses its detailed analysis of various drugs in different stages of clinical development, including phase II, I, preclinical and Discovery. It also helps to understand clinical trial details, expressive pharmacological action, agreements and collaborations, and the latest news and press releases.

Tuberous Sclerosis Emerging Drugs

GWP42003-P: GW Research Ltd

GWP42003-P also known as epidiolex is an oral cannabidiol solution that GW Pharmaceuticals developed. Epidiolex is a plant-derived cannabidiol, a non-psychoactive marijuana extract. It is being studied in phase III stage for the treatment of patients with Tuberous sclerosis.

Ganaxolone: Marinus Pharmaceuticals

Ganaxolone is a GABAA receptor modulator that acts by regulating brain activity. GABA is the chief inhibitory neurotransmitter in the brain. Ganaxolone binds to one of the receptor subtypes, the GABAA receptor. When this receptor is activated, it increases the movement of chloride ions through a pore on the cell surface. This action changes the positive and negative charges inside and outside the cell, which inhibits the firing of the neuron. The drug is being evaluated in phase II stage for the treatment of Tuberous sclerosis.

Tuberous Sclerosis: Therapeutic Assessment

This segment of the report provides insights about the different Tuberous Sclerosis drugs segregated based on following parameters that define the scope of the report, such as:

Major Players in Tuberous Sclerosis

There are approx. 7+ key companies which are developing the therapies for Tuberous Sclerosis. The companies which have their Tuberous Sclerosis drug candidates in the most advanced stage, i.e. phase III include, GW Research Ltd.


This report covers around 7+ products under different phases of clinical development like

  • Late stage products (Phase III)
  • Mid-stage products (Phase II)
  • Early-stage product (Phase I) along with the details of
  • Pre-clinical and Discovery stage candidates
  • Discontinued & Inactive candidates
  • Route of Administration

Tuberous Sclerosis pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as

  • Oral
  • Subcutaneous
  • Intravenous
  • Intramuscular
  • Molecule Type

Products have been categorized under various Molecule types such as

  • Bispecific Antibody
  • Peptides
  • Small molecule
  • Gene therapy
  • Product Type

The drugs have been categorized under various product types like Mono, Combination and Mono/Combination.

Tuberous Sclerosis: Pipeline Development Activities

The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Tuberous Sclerosis therapeutic drugs key players involved in developing key drugs.

Pipeline Development Activities

The report covers the detailed information of collaborations, acquisition and merger, licensing along with a thorough therapeutic assessment of emerging Tuberous Sclerosis drugs.

Tuberous Sclerosis Report Insights

  • Tuberous Sclerosis Pipeline Analysis
  • Therapeutic Assessment
  • Unmet Needs
  • Impact of Drugs

Tuberous Sclerosis Report Assessment

  • Pipeline Product Profiles
  • Therapeutic Assessment
  • Pipeline Assessment
  • Inactive drugs assessment
  • Unmet Needs

Key Questions Answered

Current Treatment Scenario and Emerging Therapies:

  • How many companies are developing Tuberous Sclerosis drugs?
  • How many Tuberous Sclerosis drugs are developed by each company?
  • How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Tuberous Sclerosis?
  • What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the Tuberous Sclerosis therapeutics?
  • What are the recent trends, drug types and novel technologies developed to overcome the limitation of existing therapies?
  • What are the clinical studies going on for Tuberous Sclerosis and their status?
  • What are the key designations that have been granted to the emerging drugs?

Table of Contents


Executive Summary

Tuberous Sclerosis: Overview
  • Causes
  • Mechanism of Action
  • Signs and Symptoms
  • Diagnosis
  • Disease Management

Pipeline Therapeutics
  • Comparative Analysis

Therapeutic Assessment
  • Assessment by Product Type
  • Assessment by Stage and Product Type
  • Assessment by Route of Administration
  • Assessment by Stage and Route of Administration
  • Assessment by Molecule Type
  • Assessment by Stage and Molecule Type

Tuberous Sclerosis - Analytical Perspective

In-depth Commercial Assessment
  • Tuberous Sclerosis companies’ collaborations, Licensing, Acquisition -Deal Value Trends

Tuberous Sclerosis Collaboration Deals
  • Company-Company Collaborations (Licensing/Partnering) Analysis
  • Company-University Collaborations (Licensing/Partnering) Analysis

Late Stage Products (Phase III)
  • Comparative Analysis

GWP42003-P: GW Research Ltd
  • Product Description
  • Research and Development
  • Product Development Activities

Mid Stage Products (Phase II)
  • Comparative Analysis

Ganaxolone: Marinus Pharmaceuticals
  • Product Description
  • Research and Development
  • Product Development Activities

Preclinical Stage Products
  • Comparative Analysis

Drug Name: Company Name
  • Product Description
  • Research and Development
  • Product Development Activities

Inactive Products
  • Comparative Analysis

Tuberous Sclerosis Key Companies

Tuberous Sclerosis Key Products

Tuberous Sclerosis- Unmet Needs

Tuberous Sclerosis- Market Drivers and Barriers

Tuberous Sclerosis- Future Perspectives and Conclusion

Tuberous Sclerosis Analyst Views

Tuberous Sclerosis Key Companies

AppendixList of Tables
Table 1 Total Products for Tuberous Sclerosis
Table 2 Late Stage Products
Table 3 Mid Stage Products
Table 4 Early Stage Products
Table 5 Pre-clinical & Discovery Stage Products
Table 6 Assessment by Product Type
Table 7 Assessment by Stage and Product Type
Table 8 Assessment by Route of Administration
Table 9 Assessment by Stage and Route of Administration
Table 10 Assessment by Molecule Type
Table 11 Assessment by Stage and Molecule Type
Table 12 Inactive ProductsList of Figures
Figure 1 Total Products for Tuberous Sclerosis
Figure 2 Late Stage Products
Figure 3 Mid Stage Products
Figure 4 Early Stage Products
Figure 5 Preclinical and Discovery Stage Products
Figure 6 Assessment by Product Type
Figure 7 Assessment by Stage and Product Type
Figure 8 Assessment by Route of Administration
Figure 9 Assessment by Stage and Route of Administration
Figure 10 Assessment by Molecule Type
Figure 11 Assessment by Stage and Molecule Type
Figure 12 Inactive Products

Companies Mentioned

A selection of companies mentioned in this report includes:

  • Novartis
  • Marinus Pharmaceuticals
  • Aucta Pharmaceuticals, Inc
  • Dermatology Specialties Limited Partnership
  • Ovid Therapeutics
  • GW Research Ltd