Rett Syndrome Market

Market Overview:

The 7 major Rett syndrome markets reached a value of US$ 182.6 Million in 2024. Looking forward, IMARC Group expects the 7MM to reach US$ 419.4 Million by 2034, exhibiting a growth rate (CAGR) of 7.85% during 2024-2034.

The Rett syndrome market has been comprehensively analyzed in IMARC's new report titled "Rett Syndrome Market: Epidemiology, Industry Trends, Share, Size, Growth, Opportunity, and Forecast 2024-2034". Rett syndrome is a rare inherited neurological and developmental illness that causes severe impairments in the brain. The disorder is associated with many mutations in the MECP2 gene, which encodes the methyl-CpG binding protein-2 (MECP2) essential for the normal function of nerve cells. These variations result in a progressive loss of language and motor skills. The symptoms associated with Rett syndrome include a wide-based gait, walking on the toes, slowed head growth, teeth grinding, difficulty chewing, diminished eye contact, apraxia, loss of muscle tone, distinctive hand movements, etc. Individuals suffering from the disorder may also experience sleep problems, seizures, cognitive disabilities, scoliosis, and breathing difficulties. The diagnosis of this disease is based on identifying underlying symptoms and evaluating the patient's physical and neurological status. A blood or genetic workup is utilized to determine the presence of gene mutations that cause Rett syndrome. Additionally, the healthcare provider may perform various procedures, such as neuroimaging and electrocardiography, to confirm a diagnosis.

The rising prevalence of inherited disorders due to gene mutations, DNA abnormalities, and chromosomal damage is primarily driving the Rett syndrome market. Apart from this, the widespread adoption of effective drugs, such as anticonvulsants and sedatives, to produce calmness, control seizures, and relieve symptoms in patients is also creating a positive outlook for the market. Additionally, the inflating application of novel diagnostic procedures, including denaturing high-performance liquid chromatography and direct sequencing analysis, which identify the gene mutations and aid in the early diagnosis of the ailment, is further propelling the market growth. Moreover, the emerging popularity of occupational and physical therapies that help improve fine motor skills, restore muscle strength, and enhance mobility in patients is acting as another significant growth-inducing factor. Besides this, the increasing utilization of nutritional interventions, such as supplements or dietary changes, to manage symptoms related to feeding difficulties or gastrointestinal issues is also bolstering the market growth. Furthermore, the escalating demand for insulin-like growth factors owing to their numerous advantages, including inhibition of neuroinflammation, decreased overactivity of microglia, correcting deficits in synaptic functions, etc., is expected to drive the Rett syndrome market in the coming years.

IMARC Group's new report provides an exhaustive analysis of the Rett syndrome market in the United States, EU4 (Germany, Spain, Italy, and France), United Kingdom, and Japan. This includes treatment practices, in-market, and pipeline drugs, share of individual therapies, market performance across the seven major markets, market performance of key companies and their drugs, etc. The report also provides the current and future patient pool across the seven major markets. According to the report, the United States has the largest patient pool for Rett syndrome and also represents the largest market for its treatment. Furthermore, the current treatment practice/algorithm, market drivers, challenges, opportunities, reimbursement scenario, unmet medical needs, etc., have also been provided in the report. This report is a must-read for manufacturers, investors, business strategists, researchers, consultants, and all those who have any kind of stake or are planning to foray into the Rett syndrome market in any manner.

Recent Developments:

• In April 2024, Acadia Pharmaceuticals Inc. announced that Health Canada had accepted its New Drug Submission (NDS) for trofinetide to treat Rett syndrome. Health Canada had granted Priority Review for Acadia’s submission.
• February 2024, Taysha Gene Therapies, Inc. published improvements to its TSHA-102 clinical trial for the treatment of Rett syndrome. The Independent Data Monitoring Committee (IDMC) granted the Company's request to proceed with dosage escalation in the REVEAL Phase 1/2 adolescent and adult trial, allowing for early advancement to the high dose cohort of 1x1015 total vg. The IDMC also approved the dosing of the second patient in cohort one (low dosage) of 5.7x1014 total vg in the REVEAL Phase 1/2 pediatric study. The Company recently announced the expansion of its ongoing REVEAL Phase 1/2 teenage and adult study in Canada to the U.S. after the submission of the adolescent and adult trial protocol to the U.S. Food and Drug Administration.
• In January 2024, Anavex Life Sciences Corp. reported topline results from the randomized, double-blind, placebo-controlled Phase 2/3 EXCELLENCE clinical trial, which assessed the clinical efficacy, safety, and tolerability of 30 mg ANAVEX 2-73 in 92 pediatric patients with Rett syndrome aged 5 to 17 years. Participants were randomized 2:1 (ANAVEX 2-73 [62 patients] vs. placebo [30 patients]) for 12 weeks, including a safety checkup at week 16. Additionally, Anavex received positive Real World Evidence (RWE) input from Rett syndrome patients under Compassionate Use Authorization.
• In November 2023, Neurogene Inc. revealed that the first two young female patients with Rett syndrome have been dosed in its ongoing Phase 1/2 trial of NGN-401. NGN-401 is an investigational adeno-associated virus (AAV) gene therapy candidate for Rett syndrome, specifically designed and delivered to enhance therapeutic effectiveness while avoiding transgenic overexpression toxicity.

Key Highlights:

• Rett syndrome affects around one in every 10,000 girls in the United States by the age of twelve.
• The global prevalence of Rett syndrome is estimated to be 7.1 cases per 100,000 females.
• Scoliosis is the most common orthopedic co-morbidity, occurring by age 15 in nearly 75% of people with Rett syndrome.
• Rett syndrome almost predominantly affects girls.
• 70% of children with Rett syndrome live to at least 50 years of age.

Drugs:

Daybue (trofinetide) is the first and only FDA-approved medication for Rett syndrome in adults and pediatric patients aged two years and older. Daybue is available as an oral solution at a concentration of 200 mg/mL.

ANAVEX 2-73 (blarcamesine) is an orally available, small molecule designed to help normalize brain signaling in Rett syndrome patients. The drug acts by activating the sigma-1 receptor (SIGMAR1), which is essential for maintaining cellular homeostasis and fostering neuroplasticity.

TSHA-102 is a self-complementary AAV9-based gene transfer therapy in clinical trials for Rett syndrome. TSHA-102 makes use of a unique miRNA-Responsive Auto-Regulatory Element (miRARE) technology that is intended to regulate MECP2 levels in the CNS on a cell-by-cell basis while avoiding overexpression risks.

Time Period of the Study

• Base Year: 2024
• Historical Period: 2019-2024
• Market Forecast: 2024-2034
   

Countries Covered

• United States
• Germany
• France
• United Kingdom
• Italy
• Spain
• Japan
   

Analysis Covered Across Each Country

• Historical, current, and future epidemiology scenario
• Historical, current, and future performance of the Rett syndrome market
• Historical, current, and future performance of various therapeutic categories in the market
• Sales of various drugs across the Rett syndrome market
• Reimbursement scenario in the market
• In-market and pipeline drugs

Competitive Landscape:

This report also provides a detailed analysis of the current Rett syndrome marketed drugs and late-stage pipeline drugs.

In-Market Drugs

• Drug Overview
• Mechanism of Action
• Regulatory Status
• Clinical Trial Results
• Drug Uptake and Market Performance
   

Late-Stage Pipeline Drugs

• Drug Overview
• Mechanism of Action
• Regulatory Status
• Clinical Trial Results
• Drug Uptake and Market Performance
   

*Kindly note that the drugs in the above table only represent a partial list of marketed/pipeline drugs, and the complete list has been provided in the report.

Key Questions Answered in this Report:

Market Insights

• How has the Rett syndrome market performed so far and how will it perform in the coming years?
• What are the markets shares of various therapeutic segments in 2024 and how are they expected to perform till 2034?
• What was the country-wise size of the Rett syndrome market across the seven major markets in 2024 and what will it look like in 2034?
• What is the growth rate of the Rett syndrome market across the seven major markets and what will be the expected growth over the next ten years?
• What are the key unmet needs in the market?
   

Epidemiology Insights

• What is the number of prevalent cases (2018-2034) of Rett syndrome across the seven major markets?
• What is the number of prevalent cases (2018-2034) of Rett syndrome by age across the seven major markets?
• What is the number of prevalent cases (2018-2034) of Rett syndrome by gender across the seven major markets?
• What is the number of prevalent cases (2018-2034) of Rett syndrome by type across the seven major markets?
• How many patients are diagnosed (2018-2034) with Rett syndrome across the seven major markets?
• What is the size of the Rett syndrome patient pool (2019-2024) across the seven major markets?
• What would be the forecasted patient pool (2024-2034) across the seven major markets?
• What are the key factors driving the epidemiological trend of Rett syndrome?
• What will be the growth rate of patients across the seven major markets?
   

Rett Syndrome: Current Treatment Scenario, Marketed Drugs and Emerging Therapies

• What are the current marketed drugs and what are their market performance?
• What are the key pipeline drugs and how are they expected to perform in the coming years?
• How safe are the current marketed drugs and what are their efficacies?
• How safe are the late-stage pipeline drugs and what are their efficacies?
• What are the current treatment guidelines for Rett syndrome drugs across the seven major markets?
• Who are the key companies in the market and what are their market shares?
• What are the key mergers and acquisitions, licensing activities, collaborations, etc. related to the Rett syndrome market?
• What are the key regulatory events related to the Rett syndrome market?
• What is the structure of clinical trial landscape by status related to the Rett syndrome market?
• What is the structure of clinical trial landscape by phase related to the Rett syndrome market?
• What is the structure of clinical trial landscape by route of administration related to the Rett syndrome market?