Tuberous Sclerosis Complex Market

• The tuberous sclerosis complex (TSC) market size is expected to see steady growth during the forecast period (2024-2034), primarily driven by factors like heightened awareness, improved diagnosis, advancements in medical technology and genetic testing, as well as increased knowledge among healthcare practitioners.
• The tuberous sclerosis complex (TSC) market share has advanced significantly in recent years due to an increased focus on therapeutics that address the various symptoms and risks. Among these, Novartis’s AFINITOR (everolimus), and Nobel Pharma’s HYFTOR (sirolimus topical gel) stand out as prominent mTOR inhibitors. These medications have garnered considerable interest due to their potential in addressing a range of tuberous sclerosis complex symptoms.
• While the tuberous sclerosis complex (TSC) gene is hereditary, nevertheless the disease's clinical signs and symptoms can vary in severity throughout a patient's lifetime and manifest at different ages. Lifelong monitoring and management are crucial to mitigate tuberous sclerosis complex (TSC) impacts, emphasizing the necessity for tailored interventions that align with evolving disease manifestations and improved predictive tools to anticipate disease progression accurately.
• Various emerging therapies hold promise for driving future growth in the tuberous sclerosis complex (TSC) treatment market. These include drugs like NPC-12Y gel (Nobel Pharma), Basimglurant (NOE-101) (Noema Pharma), and others that are under investigation for their potential to prevent tuberous sclerosis complex (TSC).

DelveInsight’s comprehensive report titled “Tuberous Sclerosis Complex (TSC) Market Insights, Epidemiology, and Market Forecast – 2034” offers a detailed analysis of the tuberous sclerosis complex (TSC). The report presents historical and projected epidemiological data covering the total diagnosed prevalent cases of tuberous sclerosis complex (TSC), and treated cases of tuberous sclerosis complex (TSC). In addition to epidemiology, the market report encompasses various aspects related to the patient population. These aspects include the diagnosis process, prescription patterns, physician perspectives, market accessibility, treatment options, and prospective developments in the market across seven major markets: the United States, EU4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan, spanning from 2020 to 2034.

The report analyzes the existing treatment practices and unmet medical requirements in tuberous sclerosis complex (TSC). It evaluates the market potential and identifies potential business prospects for enhancing therapies or interventions. This valuable information enables stakeholders to make well-informed decisions regarding product development and strategic planning for the market.

Tuberous Sclerosis Complex (TSC) Overview

Tuberous sclerosis complex (TSC), also known as tuberous sclerosis, is a rare genetic disease that causes non-cancerous (benign) tumors to grow in the brain and several areas of the body, including the spinal cord, nerves, eyes, lungs, heart, kidneys, and skin.

It is caused by genetic mutations in either the TSC1 or TSC2 gene. Only one of the genes needs to be affected for TSC to be present. A mutation of either one of these genes leads to abnormal development and exponential growth of cells within the body. The TSC1 gene produces a protein called hamartin.

Symptoms range from brain-related (seizures, mental disabilities), skin (white patches, rash, nodules), kidney (affected function), heart (benign tumors), and eye (lesions) to developmental and behavioral issues (learning disabilities, ASD, ADHD, aggression).

Tuberous Sclerosis Complex (TSC) Diagnosis and Treatment Algorithm

Diagnosis of tuberous sclerosis complex (TSC) relies on identifying characteristic symptoms, detailed patient and family history, thorough clinical evaluation, and specialized tests. Hypopigmented macules on the skin along with seizures or autism serve as diagnostic clues for tuberous sclerosis.

Treatment strategies for tuberous sclerosis complex (TSC) are tailored to address the symptoms and risks most relevant to the age of the patient and may include mTOR inhibitors such as AFINITOR (everolimus), and HYFTOR (sirolimus topical gel, and others. Regular surveillance and lifelong management are essential for patients with tuberous sclerosis complex (TSC) to minimize the impacts of the condition.

Tuberous Sclerosis Complex (TSC) Epidemiology

The epidemiology section on the tuberous sclerosis complex (TSC) market report offers information on the patient populations, including historical and projected trends for each of the seven major markets. Examining key opinion leader views from physicians or clinical experts can assist in identifying the reasons behind historical and projected trends. The prevalence patient pool, their trends, and the underlying assumptions are all included in this section of the report.

This section also presents the data with relevant tables and graphs, offering a clear and concise view of the prevalence of tuberous sclerosis complex (TSC). Additionally, the report discloses the assumptions made during the analysis, ensuring data interpretation and presentation transparency. This epidemiological data is valuable for understanding the disease burden and its impact on the patient population across various regions.

Key Findings

• According to the National Organization for Rare Disorders (NORD), tuberous sclerosis complex (TSC) is a rare genetic condition affecting around 1 in every 6,000 newborns in the US, with an estimated 40,000 to 80,000 individuals in the country living with tuberous sclerosis complex (TSC). In Europe, the prevalence is approximated to range from 1 in 25,000 to 1 in 11,300 individuals.
• As per the information available on the National Health Insurance Data of France, the prevalence of tuberous sclerosis complex (TSC) complex was 5 per 100,000 population.
• According to Orphanet, the estimated prevalence of tuberous sclerosis complex (TSC) in the UK is approximately 1 in every 20,000 to 25,000 individuals.

Tuberous Sclerosis Complex (TSC) Market Outlook.

The market for tuberous sclerosis complex (TSC) treatments has seen significant growth in recent years, driven by increased awareness, advancements in medical technology, and emerging therapies. Currently, the focus of the tuberous sclerosis complex (TSC) market largely centers on symptom alleviation and enhancing patient quality of life with the use of anti-seizure drugs such as phenobarbital, phenytoin, clonazepam, and others. However, satisfactory relief is often not achieved with a single medication, necessitating a combination of treatment modalities is required. Despite the US FDA approvals for drugs such as AFINITOR (everolimus) and EPIDIOLEX (cannabidiol), which target seizures in tuberous sclerosis complex (TSC) patients, there's still a significant gap in treatments addressing the root cause of the disease and providing lasting benefits.

There persists a notable gap for more efficacious treatments addressing the root cause of the condition, offering sustained benefits over the long term. Thus, the demand for innovative therapies that not only manage symptoms but also target the underlying pathophysiology of tuberous sclerosis complex (TSC) remains a critical unmet need in the market, presenting significant opportunities for further research and development in this area. 

However, some of the emerging treatments such as NPC-12Y gel (Nobel pharma), NOE-101 (Marinus pharmaceutical), and others hold promise in meeting this unmet need and represent opportunities for further research and development in the field.

 Tuberous Sclerosis Complex (TSC) Recent Developments

• In January 2025, NATCO Pharma received FDA approval for its generic Everolimus tablets (2mg, 3mg, 5mg), a version of AFINITOR DISPERZ® by Novartis. The drug is indicated for treating subependymal giant cell astrocytoma (SEGA) in patients aged one and older with tuberous sclerosis complex (TSC). The product will be launched in the U.S. by marketing partner Breckenridge Pharmaceutical.

Tuberous Sclerosis Complex (TSC) Drug Chapters

Marketed Tuberous Sclerosis Complex (TSC) Drugs

HYFTOR/RAPALIMUS (sirolimus): Nobel pharma

HYFTOR, an mTOR (mammalian target of rapamycin) inhibitor immunosuppressant was approved in April 2022 by the US FDA indicated for the treatment of facial angiofibroma associated with tuberous sclerosis in adults and pediatric patients 6 years of age and older. The US FDA has granted an Orphan Drug Designation (ODD) with 7-year market exclusivity in the US. Additionally, the drug candidate was granted SAKIGAKE fast-track designation and an ODD, it has been also approved in Japan (2018) under the brand name RAPALIMUS gel 0.2%, indicated for skin lesions associated with tuberous sclerosis complex (TSC), has enabled noninvasive treatment.

Note: Detailed marketed therapies assessment will be provided in the final report...

Emerging Tuberous Sclerosis Complex (TSC) Drugs

In recent years, there has been significant progress in understanding the underlying molecular mechanisms of tuberous sclerosis complex (TSC), which has led to the development of targeted therapies aimed at treating the disorder at its root cause. Some of the drugs in the pipeline include NPC-12Y gel (Nobelpharma), Ganaxolone (Marinus Pharmaceuticals), Basimglurant (NOE-101) (Noema Pharma), and others.

Ganaxolone: Marinus Pharmaceuticals

Ganaxolone, a positive allosteric modulator of GABAA receptors, is an investigational product being developed in intravenous and oral formulations intended to maximize therapeutic reach to adult and pediatric patient populations in both acute and chronic care. It exhibits anti-seizure and anti-anxiety activity via its effects on synaptic and extra synaptic GABAA. The drug was recently granted a new method of use patent by the USPTO for ganaxolone in TSC, expiring in 2040. The drug is currently being assessed in the global Phase III TrustTSC trial, and is enrolling patients with topline data anticipated mid-2024.

Basimglurant (NOE-101): Noema Pharma

Basimglurant (NOE-101) is a highly selective, potent, and cell-penetrant negative allosteric modulator of mGlu5 receptors for the treatment of seizures associated with tuberous sclerosis complex (TSC). It acts as a mGluR5 inhibitor that effectively blocks seizures in a transgenic animal model of tuberous sclerosis complex (TSC). Additionally, basimglurant is also being investigated for the management of pain associated with trigeminal neuralgia (TN).

Currently, the drug candidate is being investigated in a Phase IIb trial to evaluate its effectiveness in seizure control in children, adolescents, and young adults with tuberous sclerosis complex (TSC).

Note: Detailed emerging therapies assessment will be provided in the final report...

Tuberous Sclerosis Complex (TSC) Market Segmentation

DelveInsight’s ‘Tuberous Sclerosis Complex (TSC) - Market Insights, Epidemiology, and Market Forecast – 2034’ report provides a detailed outlook of the current and future tuberous sclerosis complex (TSC) market, segmented within countries, by therapies, and by classes. Further, the market of each region is then segmented by each therapy to provide a detailed view of the current and future market share of all therapies.

Tuberous Sclerosis Complex (TSC) Market Size by Countries

The Tuberous Sclerosis Complex (TSC) market size is assessed separately for various countries, including the United States, EU4 (Germany, France, Italy, and Spain), the UK, and Japan. In 2023, the United States held a significant share of the overall 7MM (Seven Major Markets) tuberous sclerosis complex (TSC) market, primarily attributed to the country's higher prevalence of the condition and the elevated cost of the available treatments. This dominance is projected to persist, especially with the potential early introduction of new products.

Tuberous Sclerosis Complex (TSC) Market Size by Therapies

Tuberous Sclerosis Complex (TSC) Market Size by Therapies is categorized into current and emerging markets for the study period 2020–2034. One of the emerging drugs anticipated to launch during the forecast period is Basimglurant (NOE-101) under the developmental pipeline of Noema Pharma.

Note: Detailed market segment assessment will be provided in the final report.

Tuberous Sclerosis Complex (TSC) Drug Uptake

This section focuses on the sales uptake of potential tuberous sclerosis complex (TSC) drugs that have recently been launched or are anticipated to be launched in the tuberous sclerosis complex (TSC) market between 2020 and 2034. It estimates the market penetration of Tuberous Sclerosis Complex (TSC) drugs for a given country, examining their impact within and across classes and segments. It also touches upon the financial and regulatory decisions contributing to the probability of success (PoS) of the drugs in the tuberous sclerosis complex (TSC) market.

The emerging tuberous sclerosis complex (TSC) therapies are analyzed based on various attributes such as safety and efficacy in randomized clinical trials, order of entry and other market dynamics, and the unmet need they fulfill in the tuberous sclerosis complex (TSC) market.

Note: Detailed assessment of drug uptake and attribute analysis will be provided in the full report on Tuberous Sclerosis Complex (TSC)....

Tuberous Sclerosis Complex (TSC) Market Access and Reimbursement

DelveInsight’s ‘Tuberous Sclerosis Complex (TSC) – Market Insights, Epidemiology, and Market Forecast – 2034’ report provides a descriptive overview of the market access and reimbursement scenario of Tuberous Sclerosis Complex (TSC).

This section includes a detailed analysis of the country-wise healthcare system for each therapy, enlightening the market access, reimbursement policies, and health technology assessments.

KOL Views

To keep up with current tuberous sclerosis complex (TSC) market trends and fill gaps in secondary findings, we interview KOLs and SMEs working in the tuberous sclerosis complex (TSC) domain. Their opinion helps understand and validate current and emerging therapies and treatment patterns or tuberous sclerosis complex (TSC) market trends. This will support the clients in potential upcoming novel treatments by identifying the overall scenario of the market and the tuberous sclerosis complex (TSC) unmet needs.

Tuberous Sclerosis Complex (TSC): KOL Insights

DelveInsight’s analysts connected with 50+ KOLs to gather insights; however, interviews were conducted with 15+ KOLs in the 7MM. These KOLs were from organizations, institutes, and hospitals such as the Connecticut Children’s Medical Center in the US, Kanazawa Medical University Hospital in Japan, McGovern Medical School in the US, the University of Texas Health Science Center in the US, Massachusetts General Hospital in the US, and Montefiore Medical Center in the US, among others.

“Tuberous sclerosis complex frequently impacts the brain, leading to the formation of non-cancerous growths on the brain's outer layer, known as cortical tubers. People affected by tuberous sclerosis complex commonly exhibit a range of behavioral patterns referred to as TSC-associated neuropsychiatric disorders (TAND).”

Note: Detailed assessment of KOL Views will be provided in the full report on Tuberous Sclerosis Complex (TSC)...

Competitive Intelligence Analysis

We conduct a Competitive and Market Intelligence analysis of the tuberous sclerosis complex (TSC) Market, utilizing various Competitive Intelligence tools such as SWOT analysis and Market entry strategies. The inclusion of these analyses is contingent upon data availability, ensuring a comprehensive and well-informed assessment of the market landscape and competitive dynamics.

Tuberous Sclerosis Complex (TSC) Pipeline Development Activities

The report offers an analysis of therapeutic candidates in Phase II and III stages and examines companies involved in developing targeted therapeutics for tuberous sclerosis complex (TSC). It provides valuable insights into the advancements and progress of potential treatments in clinical development for this condition.

Pipeline Development Activities

The report covers information on collaborations, acquisition and merger, licensing, patent details, and other information for emerging tuberous sclerosis complex (TSC) therapies.

Tuberous Sclerosis Complex (TSC) Report Insights

• Tuberous Sclerosis Complex (TSC) Patient Population
• Therapeutic Approaches
• Tuberous Sclerosis Complex (TSC) Pipeline Analysis
• Tuberous Sclerosis Complex (TSC) Market Size and Trends
• Tuberous Sclerosis Complex (TSC) Market Opportunities
• Impact of Upcoming Therapies

Tuberous Sclerosis Complex (TSC) Report Key Strengths

• 11 Years Forecast
• The 7MM Coverage
• Tuberous Sclerosis Complex (TSC) Epidemiology Segmentation
• Key Cross Competition
• Highly Analyzed Tuberous Sclerosis Complex (TSC) Market
• Tuberous Sclerosis Complex (TSC) drug uptake

Tuberous Sclerosis Complex (TSC) Report Assessment

• Tuberous Sclerosis Complex (TSC) Current Treatment Practices
• Unmet Needs
• Tuberous Sclerosis Complex (TSC) Pipeline Product Profiles
• Tuberous Sclerosis Complex (TSC) Market Attractiveness

Key Questions

• How common is tuberous sclerosis complex (TSC)?
• What are the key findings of tuberous sclerosis complex (TSC) epidemiology across the 7MM, and which country will have the highest number of patients during the study period (2020–2034)?
• What are the currently available treatments for tuberous sclerosis complex (TSC)?
• What are the disease risk, burden, and unmet needs of tuberous sclerosis complex (TSC)?
• At what CAGR is the tuberous sclerosis complex (TSC) market and its epidemiology is expected to grow in the 7MM during the forecast period (2024–2034)?
• How would the unmet needs impact the tuberous sclerosis complex (TSC) market dynamics and subsequently influence the analysis of the related trends?
• What would be the forecasted patient pool of tuberous sclerosis complex (TSC) in the 7MM covering the United States, EU4 (Germany, France, Italy, and Spain), the UK, and Japan?
• Among EU4 and the UK, which country will have the highest number of patients during the forecast period (2024–2034)?
• How many companies are currently developing therapies for the treatment of tuberous sclerosis complex (TSC)?

Frequently Asked Questions

1. Is tuberous sclerosis complex (TSC) hereditary?

Yes, tuberous sclerosis complex (TSC) is indeed hereditary. It's caused by mutations in either the TSC1 gene on chromosome 9 or the TSC2 gene on chromosome 16.

2. Can tuberous sclerosis complex (TSC) be cured?

There is currently no cure for tuberous sclerosis complex (TSC), but treatments are available to manage symptoms and improve quality of life.

3. Are there any clinical trials for tuberous sclerosis complex (TSC) treatments?

Research and clinical trials are ongoing to explore potential treatments for tuberous sclerosis complex (TSC). Some experimental treatments, such as the activation of ATP-sensitive potassium channels, have shown promise in slowing disease progression.

4. How is the market size estimated in the forecast report?

The market size is estimated through data analysis, statistical modeling, and expert opinions. It may consider factors such as prevalence cases, treatment costs, revenue generated, and market trends.

5. Is there an analysis of the market’s competitive landscape in the report?

The market forecast report will likely offer insights into key market players, their product offerings, partnerships, and strategies, providing stakeholders with a comprehensive understanding of the competitive dynamics in the tuberous sclerosis complex (TSC) market.