Tuberous Sclerosis Treatment Market Size, Share, Industry, Forecast and Outlook (2024-2031)

Global Tuberous Sclerosis Treatment Market is segmented By Drug Class (Antiepileptic Drugs, Benzodiazepines, Adrenocorticotropic Agents, mTOR Kinase Inhibitor) By Route of Administration (Oral, Topical, Injection, Others) By End Users (Hospitals, Clinics, Ambulatory Surgical Centers, Others) and By Region (North America, Europe, South America, Asia Pacific, Middle East, and Africa) – Share, Size, Outlook, and Opportunity Analysis, 2024-2031

Market Overview

Global Tuberous Sclerosis Treatment Market reached US$ YY billion in 2022 and is expected to reach US$ YY billion by 2031 growing with a CAGR of YY% during the forecast period 2024-2031.

Tuberous sclerosis complex causes non-cancerous tumors that grow throughout the body like on the skin and vital organs including the brain, kidney, lung, and heart. The global tuberous sclerosis treatment market has witnessed significant growth in recent years due to the adoption of mTOR kinase inhibitors. For instance, oral mTOR inhibitors shows effective in the treatment of a range of TSC-related manifestations.

Furthermore, significant growth drivers such as increasing collaborations, and research and developments give rise to future market growth. Due to the massive rise in technological advancements in countries like the U.S. and Canada, the market is experiencing a rise in demand from North America. Significant key players like Novartis AG, GW Pharmaceuticals, Takeda Pharmaceuticals, and others are actively operating in the market.

Market Scope

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Market Dynamics

Increasing Research and Developments  for Tuberous Sclerosis Treatment 

The global tuberous sclerosis treatment market has witnessed significant growth due to various recent research and developments. Tuberous sclerosis is usually diagnosed on the basis of medical history and clinical and imaging findings. Also, advanced research and developments in treating tuberous sclerosis help to accelerate market growth.

For instance, in June 2023, Ganaxolone is under clinical development by Marinus Pharmaceuticals and is currently in Phase III for Tuberous Sclerosis.Ganaxolone nanoparticle formulation is under development for the treatment of seizures associated with CDKL5 deficiency disorder (CDD), status epilepticus (SE), and tuberous sclerosis complex.

Rising FDA Approvals of Drugs for Tuberous Sclerosis Treatment 

The increase in drug approvals by regulatory bodies helps to drive market growth. Tuberous sclerosis is characterized by the growth of numerous non-cancerous tumors in many parts of the body like the brain, kidneys, heart, skin, and other organs. Tuberous sclerosis is generally caused by mutations in the TSC1 or TSC2 gene. 

For instance,in August 2022, FDA approved HYFTOR topical treatment for facial angiofibroma associated with tuberous sclerosis. HYFTOR is the first and only topical medication approved by the FDA to treat facial angiofibroma associated with tuberous sclerosis in adults and children 6 years of age and older and received Orphan Drug status for this indication.

Complications Associated with Tuberous Sclerosis are Expected to Hamper the Market Growth 

Tuberous sclerosis treatment can result in a range of associated health problems including epilepsy, learning abilities, autistic spectrum disorder, breathing difficulties, and skin abnormalities such as patches of light-colored or thickened skin. Thus, owing to the above factors the market is expected to hamper over the forecast period.

Segment Analysis

The global tuberous sclerosis treatment market is segmented based on drug class, route of administration, end users and region.

Antiepileptic drugs  from the Drug Class Segment Accounted for Approximately 38.5% of the Global Tuberous Sclerosis Treatment Market Share 

Antiepileptic drugs (AEDs) play a significant role in managing epilepsy in individuals with tuberous sclerosis complex (TSC). The choice of specific AED(s) for treating seizures in patients with TSC depends on the patient's seizure type(s), epilepsy syndrome(s), other involved organ systems, age of the patient, and AED side effect profiles and formulations available.

For instance, in January 2024, Akumentis Healthcare launched Clasepi, a DCGI-approved CBD drug designed to treat epilepsy in patients aged 1 year and older. The CBD is specifically formulated to address seizures linked to Lennox-Gastaut Syndrome, Dravet Syndrome, or Tuberous Sclerosis Complex.

Geographical Penetration

North America Accounted for Approximately 38.4% of the Market Share in 2022

North America has maintained a significant proportion of the tuberous sclerosis market in recent years, and this trend is anticipated to continue throughout the forecast period due to the factors such as an increase in the prevalence of rare diseases, increasing research and developments, and technological advancements in tuberous sclerosis treatment.

For instance, according to TSC Alliance Organisation,it is estimated that at least two children are born with tuberous sclerosis complex. Research suggests that the prevalence of tuberous sclerosis-affected births is one in 6,000. Globally, there are close to one million individuals living with TSC, with around 50,000 residing in the United States.

Competitive Landscape

The major global players in the market include Novartis AG, GW Pharmaceuticals, Takeda Pharmaceuticals, Aucta Pharmaceuticals, Ovid Therapeutics, Pfizer Inc., Shijiazhuang Yiling Pharmaceutical Co, Par Pharmaceuticals, Marinus Pharmaceuticals, Longboard Pharmaceuticals among others.

COVID-19 Impact Analysis

The COVID-19 pandemic has had a significant impact on thetuberous sclerosis treatment market. During the height of the pandemic, many healthcare systems were overwhelmed, and non-urgent medical procedures and screenings were postponed or canceled. This led to delays in tuberous sclerosis treatment research. Clinical trials are vital for developing new treatments and improving outcomes in the tuberous sclerosis treatment market. 

However, the pandemic disrupted the conduct of clinical trials, with many sites temporarily halting enrollment or modifying protocols to prioritize patient safety. This has led to delays in the completion of trials and the availability of new therapies. Moreover, the focus of research and development efforts shifted during the pandemic, with a significant emphasis on developing vaccines and treatments for COVID-19. Therefore, owing to the above factors, the tuberous sclerosis treatment market is expected to be moderately affected over the forecast period.

Key Developments

• In February 2023, National Institute for Health and Clinical Excellence recommends Epidyolex for the treatment of tuberous sclerosis complex. Epidyolex contains an isolated form of CBD and is approved for use in three rare forms of epilepsy.
• In July 2023, a project has been launched led by researchers of Kings College London that will focus on rare diseases like tuberous sclerosis that are connected to the part of the body called the ‘mTOR pathway’. The mTOR Pathway Diseases Node is made up of different researchers, industry groups, and charities.
• In August 2021, Orion Corporation collaborated with Marinus to obtain exclusive rights for marketing both oral and intravenous (IV) dose formulations of ganaxolone in the European Economic Area (EEA), UK, and Switzerland for CDD, tuberous sclerosis complex (TSC), and refractory status epilepticus.

Why Purchase the Report?

• To visualize the global tuberous sclerosis treatment market segmentation based on the drug class, route of administration, end users and region as well as understand key commercial assets and players.
• Identify commercial opportunities by analyzing trends and co-development. 
• Excel data sheet with numerous data points of global tuberous sclerosis treatment market-level with all segments.
• PDF report consists of a comprehensive analysis after exhaustive qualitative interviews and an in-depth study.
• Product mapping available as excel consisting of key products of all the major players.

The global tuberous sclerosis treatment market report would provide approximately 69 tables, 70 figures, and 195 Pages.

Target Audience 2024

• Manufacturers/ Buyers
• Industry Investors/Investment Bankers
• Research Professionals
• Emerging Companies