Product Code: MM202045
Due to various unmet needs in treatment, several emerging drugs like Trofinetide (Neuren Pharmaceuticals Limited/Acadia Pharmaceuticals), ANAVEX 2-73 (Anavex Life Sciences), Triheptanoin (Ultragenyx Pharmaceutical), Tianeptine (AMO Pharma), and much more could be the solution which would also significantly increase the market size of Rett Syndrome.
Rett syndrome (RTT) is a severe neurodevelopmental disorder that mostly affects girls. It is characterized by normal early growth and development followed by a slowing of development, loss of purposeful use of the hands, distinctive hand movements, slowed brain and head growth, problems with walking, seizures, and intellectual disability. Currently, there is no cure for Rett Syndrome (RTT). However, there is a variety of off-label therapies are aimed at symptomatic and supportive care through a multidisciplinary approach which may improve the potential for movement, communication, and social participation.
The main goals of treatment are to relieve patients from seizure disorders, behavioral alterations, sleep disorders, breathing irregularities, cardiac dysfunction (prolonged QT interval), gastrointestinal dysfunction, and bone fractures. Many people with Rett syndrome live well into adulthood, although they may require constant care and supervision. However, there is an increased risk of sudden death in people with Rett syndrome. Approximately one-quarter of deaths in Rett are sudden and unexpected. This may be due, in part, to heart irregularities, specifically a prolonged QT interval and T-wave abnormalities.
Rett Syndrome - Prevalence (Epidemiology)
The total prevalent cases in the G7 countries are anticipated to rise to 40,654 cases by 2032 for the study period (2018-2032). As per estimates, the United States accounted for the highest prevalence of Rett Syndrome (RTT) cases in 2018 which was 16,580 cases, and expected to increase by 2032 with an impressive CAGR for the study period 2018-2032. Among the EU5, Germany had the highest Rett Syndrome (RTT) cases, followed by France, Italy, the UK, and Spain. Japan is reported to have the highest number of cases after the United States.
In the United States, Rett syndrome impacts approximately 6,000 to 9,000 patients. Typically, with symptoms presenting between six to 18 months of age, patients experience a period of rapid decline with loss of purposeful hand use (fine motor skills), development of hand stereotypies, absent or impaired mobility (gross motor skills), loss of communication skills (including eye contact) and inability to independently conduct activities of daily living.
Rett Syndrome Current Market Size & Forecast Trends
An estimated annual growth rate of 0.4% in the Prevalent Rett syndrome (RTT) population across the major pharmaceutical markets will contribute to increasing RTT therapy sales. In 2018, there were approximately 38,444 prevalent cases of RTT in the major markets; we expect the number of cases will exceed in 2032 owing to changes in population demographics and increasing cases of RS patients' group.
Due to the rareness of the RTT, the treatment cost and travel cost also play an important role which directly affects the affected role and their family which is unable to manage the disease symptoms. There is a high need for effective medical treatment options for Rett syndrome, which can provide a better treatment option. With pivotal Phase III trials studying ANAVEX 2-73 and Trofinetide, we forecast that the first approved therapy for Rett Syndrome (RTT) will likely be available in 2022.
In the 2018-2032 forecast period, we expect a greater uptake of ANAVEX2-73-Anavex, followed by trofinetide-Acadia Pharmaceuticals, Triheptanoin (UX-007) -Ultragenyx in the sequence in the US market for the treatment of the Rett Syndrome by 2032. Other potential upcoming therapies like AMO04-AMO Pharma, BHV5000-Biohaven, NLX101-Neurolixis, TSHA-102-Taysha Gene Therapies, VYNT0126-Vyant Bio, and many others.
Report Highlights:
- Rett Syndrome - Current Market Trends
- Rett Syndrome - Current & Forecasted Cases across the G7 Countries
- Rett Syndrome - Market Opportunities And Sales Potential for Agents
- Rett Syndrome - Patient-based Market Forecast to 2032
- Rett Syndrome - Untapped Business Opportunities
- Rett Syndrome - Product Positioning Vis-a-vis Competitors' Products
- Rett Syndrome - KOLs Insight
Table of Contents
Executive Summary
Rett Syndrome (RTT) Disease Background
- Rett Syndrome (RTT) Definition
- Stages
- Symptoms & Causes
- Pathophysiology
- Diagnosis
Epidemiology Estimated and Forecast to 2032
- Key Findings
- Methods and Data Sources
- Country Specific Prevalent cases of Rett Syndrome (RTT) (US, Germany, France, Italy, Spain, UK, and Japan)
- Country Specific Diagnosed and Drug Treated Cases of Rett Syndrome (RTT)
Key Sources for Rett Syndrome (RTT) Epidemiology and Model Parameters
- The United States
- The United States Prevalent cases of Rett Syndrome (RTT)
- The United States Diagnosed and Drug Treated Cases of Rett Syndrome (RTT)
- Germany
- Germany Prevalent cases of Rett Syndrome (RTT)
- Germany Diagnosed and Drug Treated Cases of Rett Syndrome (RTT)
- France
- France Prevalent cases of Rett Syndrome (RTT)
- France Diagnosed and Drug Treated Cases of Rett Syndrome (RTT)
- Italy
- Italy Prevalent cases of Rett Syndrome (RTT)
- Italy Diagnosed and Drug Treated Cases of Rett Syndrome (RTT)
- Spain
- Spain Prevalent cases of Rett Syndrome (RTT)
- Spain Diagnosed and Drug Treated Cases of Rett Syndrome (RTT)
- The United Kingdom
- United Kingdom Prevalent cases of Rett Syndrome (RTT)
- The United Kingdom Diagnosed and Drug Treated Cases of Rett Syndrome (RTT)
- Japan
- Japan Prevalent cases of Rett Syndrome (RTT)
- Japan Diagnosed and Drug Treated Cases of Rett Syndrome (RTT)
Current Therapies and Medical Practice
- Key Findings
- Treatment Guidelines
Unmet Needs
Emerging Therapies
- Key Findings
- Pipeline Overview
Notable Developments in the Rett Syndrome (RTT) space
Unmet Needs
Emerging Therapies
- Pipeline Overview
- Therapeutic Developments Pipeline for Rett Syndrome (RTT)
- Product Analysis
- Trofinetide (Neuren Pharmaceuticals Limited/Acadia Pharmaceuticals)
- Product Profile
- Clinical Development
- Sales & Market Opportunity by 2032
- ANAVEX 2-73 (Anavex Life Sciences)
- Product Profile
- Clinical Development
- Sales & Market Opportunity by 2032
- Triheptanoin (Ultragenyx Pharmaceutical)
- Product Profile
- Clinical Development
- Sales & Market Opportunity by 2032
- Tianeptine (AMO Pharma)
- Product Profile
- Clinical Development
- BVH-5000 (Biohaven Pharmaceuticals)
- NLX-101 (Neurolixis)
- TSHA 102 (Taysha Gene Therapies)
- VYNT-0126 (Vyant Bio, Inc. (Formerly Known as Cancer Genetics, Inc.))
- Arvisol (Echo Pharmaceuticals)
- STRX 230 (Sarepta Therapeutics/StrideBio)
- REL-1017 (Relmada Therapeutics)
- Sigma 1 receptor ligand (Epigen Biosciences)
- ACTX-101 (Alcyone Therapeutics)
- OAV201 (Novartis Gene Therapies) (Discontinued)
- Gilenya (Novartis)
Rett Syndrome (RTT)- Cost Burden & Prescriptions survey
Future Treatment Paradigm
- Rett Syndrome (RTT) Competitor Landscape and Approvals Anticipated
- Future Treatment Algorithms and Competitor Positioning
- Key Data Summary for Emerging Treatment
Annual Cost of Current & Emerging Therapies
Market Outlook
- Key Findings
- Country-Specific Market Forecast to 2032
- G7 total Market for Rett Syndrome (RTT) 2018-2032 (USD Million)
- G7 total Market for Rett Syndrome (RTT) by Therapies 2018-2032 (USD Million)
Market Forecast by Country
- The United States
- United States Market for Rett Syndrome (RTT) 2018-2032 (USD Million)
- United States Market for Rett Syndrome (RTT) by Therapies 2018-2032 (USD Million)
- Germany
- Germany Market for Rett Syndrome (RTT) 2018-2032 (USD Million)
- Germany Market for Rett Syndrome (RTT) by Therapies 2018-2032 (USD Million)
- France
- France Market for Rett Syndrome (RTT) 2018-2032 (USD Million)
- France Market for Rett Syndrome (RTT) by Therapies 2018-2032 (USD Million)
- Italy
- Italy Market for Rett Syndrome (RTT) 2018-2032 (USD Million)
- Italy Market for Rett Syndrome (RTT) by Therapies 2018-2032 (USD Million)
- Spain
- Spain Market for Rett Syndrome (RTT) 2018-2032 (USD Million)
- Spain Market for Rett Syndrome (RTT) by Therapies 2018-2032 (USD Million)
- The United Kingdom
- United Kingdom Market for Rett Syndrome (RTT) 2018-2032 (USD Million)
- United Kingdom Market for Rett Syndrome (RTT) by Therapies 2018-2032 (USD Million)
- Japan
- Japan Market for Rett Syndrome (RTT) 2018-2032 (USD Million)
- Japan Market for Rett Syndrome (RTT) by Therapies 2018-2032 (USD Million)
Market Drivers and Constraints
- What Factors Are Driving the Market for Rett Syndrome (RTT)?
- What Factors Are Constraining the Market for Rett Syndrome (RTT)?
Appendix