1. Key Insights

2. Fragile X Syndrome (FXS): Market Overview at a Glance

• 2.1. Total Market Share (%) Distribution of FXS in 2017
• 2.2. Total Market Share (%) Distribution of FXS in 2028

3. Disease Background and Overview: Fragile X Syndrome (FXS)

• 3.1. Introduction
• 3.2. Genomics
• 3.3. Symptoms
• 3.4. Clinical Features
• 3.5. Diagnosis
• 3.6. Fragile X and Newborn Screening
  • 3.6.1. Recommendations for Diagnostic Testing
  • 3.6.2. Differential Diagnosis

4. Epidemiology and Patient Population: Fragile X Syndrome

• 4.1. Key Findings
• 4.2. Total Diagnosed Prevalent cases of Fragile X Syndrome in 7MM
• 4.3. United States
  • 4.3.1. Assumptions and Rationale
  • 4.3.2. Total Diagnosed Prevalent Cases of Fragile X Syndrome (FXS) in the United States
  • 4.3.3. Gender-Specific Cases of Fragile X Syndrome (FXS) in the United States
  • 4.3.4. Age-Specific Cases of Fragile X Syndrome (FXS) in the United States
• 4.4. EU5
• 4.5. Assumptions and Rationale
• 4.6. Germany
  • 4.6.1. Total Diagnosed Prevalent Cases of Fragile X Syndrome (FXS) in Germany
  • 4.6.2. Gender-Specific Cases of Fragile X Syndrome (FXS) in Germany
  • 4.6.3. Age-Specific Cases of Fragile X Syndrome (FXS) in Germany
• 4.7. France
  • 4.7.1. Total Diagnosed Prevalent Cases of Fragile X Syndrome (FXS) in France
  • 4.7.2. Gender-Specific Cases of Fragile X Syndrome (FXS) in France
  • 4.7.3. Age-Specific Cases of Fragile X Syndrome (FXS) in France
• 4.8. Italy
  • 4.8.1. Total Diagnosed Prevalent Cases of Fragile X Syndrome (FXS) in Italy
  • 4.8.2. Gender-Specific Cases of Fragile X Syndrome (FXS) in Italy
  • 4.8.3. Age-Specific Cases of Fragile X Syndrome (FXS) in Italy
• 4.9. Spain
  • 4.9.1. Total Diagnosed Prevalent Cases of Fragile X Syndrome (FXS) in Spain
  • 4.9.2. Gender-Specific Cases of Fragile X Syndrome (FXS) in Spain
  • 4.9.3. Age-Specific Cases of Fragile X Syndrome (FXS) in Spain
• 4.10. United Kingdom (UK)
  • 4.10.1. Total Diagnosed Prevalent Cases of Fragile X Syndrome in the United Kingdom
  • 4.10.2. Gender-Specific Cases of Fragile X Syndrome in the United Kingdom
  • 4.10.3. Age-Specific Cases of Fragile X Syndrome (FXS) in the United Kingdom
• 4.11. Japan
  • 4.11.1. Assumptions and Rationale
  • 4.11.2. Total Diagnosed Prevalent Cases of Fragile X Syndrome (FXS) in Japan
  • 4.11.3. Gender-Specific Cases of Fragile X Syndrome (FXS) in Japan
  • 4.11.4. Age-Specific Cases of Fragile X Syndrome (FXS) in Japan

5. Current Treatment

6. Unmet Need

7. Emerging Drugs

• 7.1. Key Cross Competition
• 7.2. Cannabidiol: Zynerba Pharmaceuticals
  • 7.2.1. Product Description
  • 7.2.2. Other Developmental Activities
  • 7.2.3. Clinical Development
  • 7.2.4. Safety and Efficacy
  • 7.2.5. Product Profile
• 7.3. Acamprosate: Confluence Pharmaceuticals
  • 7.3.1. Product Description
  • 7.3.2. Other Developmental Activities
  • 7.3.3. Clinical Development
  • 7.3.4. Safety and Efficacy
  • 7.3.5. Product Profile
• 7.4. BPN14770: Tetra Therapeutics
  • 7.4.1. Product Description
  • 7.4.2. Other Developmental Activities
  • 7.4.3. Clinical Development
  • 7.4.4. Safety and Efficacy
  • 7.4.5. Product Profile
• 7.5. OV101: Ovid Therapeutics
  • 7.5.1. Product Description
  • 7.5.2. Other Developmental Activities
  • 7.5.3. Clinical Development
  • 7.5.4. Safety and Efficacy
  • 7.5.5. Product Profile
• 7.6. Trofinetide: Neuren Pharmaceuticals
  • 7.6.1. Product Description
  • 7.6.2. Other Developmental Activities
  • 7.6.3. Clinical Development
  • 7.6.4. Safety and Efficacy
  • 7.6.5. Product Profile

8. Market Size: Fragile X Syndrome

• 8.1. Key Findings
• 8.2. Total Market Size of Fragile X Syndrome in 7MM
• 8.3. Total Market Size of Fragile X Syndrome by Therapies in 7MM

9. Market Outlook: 7 MM

• 9.1. United States
  • 9.1.1. The total market size of Fragile X Syndrome in the US
  • 9.1.2. Total Market Size of Fragile X Syndrome by Therapies in the US
• 9.2. EU5
• 9.3. Germany
  • 9.3.1. The total market size of Fragile X Syndrome in Germany
  • 9.3.2. Total Market Size of Fragile X Syndrome by Therapies in Germany
• 9.4. France
  • 9.4.1. The total market size of Fragile X Syndrome in France
  • 9.4.2. Total Market Size of Fragile X Syndrome by Therapies in France
  • 9.4.3. Italy
  • 9.4.4. The total market size of Fragile X Syndrome in Italy
  • 9.4.5. Total Market Size of Fragile X Syndrome by Therapies in Italy
• 9.5. Spain
  • 9.5.1. The total market size of Fragile X Syndrome in Spain
  • 9.5.2. Total Market Size of Fragile X Syndrome by Therapies in Spain
• 9.6. United Kingdom
  • 9.6.1. The total market size of Fragile X Syndrome in the UK
  • 9.6.2. Total Market Size of Fragile X Syndrome by Therapies in the UK
• 9.7. Japan
  • 9.7.1. The total market size of Fragile X Syndrome in Japan
  • 9.7.2. Total Market Size of Fragile X Syndrome by Therapies in Japan

10. Market Drivers

11. Market Barriers

12. KOL Views

13. Appendix

• 13.1. Report Methodology

14. DelveInsight Capabilities

15. Disclaimer

16. About DelveInsight

DelveInsight’s ‘Fragile X Syndrome (FXS)- Market Insights, Epidemiology, and Market Forecast-2028’ report deliver an in-depth understanding of the disease, historical & forecasted epidemiology as well as the market trends of Fragile X Syndrome in the United States, EU5 (Germany, Spain, Italy, France and the United Kingdom), and Japan. The Report provides the current treatment practices, emerging drugs, market share of the individual therapies, the current and forecasted market size of Fragile X Syndrome from 2017 to 2028 segmented by seven major markets.
Fragile X Syndrome is a genetic disease due to a CGG trinucleotide expansion, called as full mutation (greater than 200 CGG repeats), in the fragile X mental retardation 1 gene locus Xq27.3. This leads to a hyper-methylated region in the gene promoter therefore silencing it and lowering the expression levels of the fragile X mental retardation 1, a protein involved in synaptic plasticity and maturation. It is considered as the most common inherited cause of intellectual disability.
Individuals with Fragile X Syndrome are present with intellectual disability, autism, hyperactivity, long face, large or prominent ears, and macroorchidism at puberty and thereafter. Most of the young children with Fragile X Syndrome will present with language delay, sensory hyperarousal and anxiety.
Geography Covered
• The United States
• EU5 (Germany, France, Italy, Spain, and the United Kingdom)
• Japan
Study Period: 2017–2028
Fragile X Syndrome - Disease Understanding and Treatment Algorithm
Fragile X Syndrome causes intellectual disability, behavioral and learning challenges and various physical characteristics. Though Fragile X Syndrome occurs in both genders, males are more frequently affected than females, and generally with greater severity. Life expectancy is not affected in people with Fragile X Syndrome because there are usually no life-threatening health concerns associated with the condition.
As one of the most common cause of inherited intellectual disability, Fragile X Syndrome occurs in 1 in every 4000 males and 1 in every 8000 females. The average age of male Fragile X Syndrome diagnosis is 35-37 months and females Fragile X Syndrome diagnosis is 42 months.
Fragile X Syndrome Epidemiology
The Fragile X Syndrome epidemiology division provides insights about historical and current patient pool and forecasted trend for every 7 major countries. It helps to recognize the causes of current and forecasted trends by exploring numerous studies and views of key opinion leaders (KOL).
The disease epidemiology covered in the report provides historical as well as forecasted epidemiology (Total Diagnosed Prevalence of Fragile X Syndrome in 7MM, Gender-Specific Prevalence of Fragile X Syndrome in 7MM, and Age-Specific Prevalence of Fragile X Syndrome), scenario of Fragile X Syndrome in the 7MM covering United States, EU5 countries (Germany, Spain, Italy, France and United Kingdom) and Japan from 2017–2028.
According to DelveInsight, the total prevalent cases of Fragile X Syndrome in the 7MM was found to be 117,962 in 2017. The diagnosed prevalence of Fragile X Syndrome in the 7MM is expected to increase during the study period of 2017-2028 due to increase in the awareness of the condition.
Fragile X Syndrome Drug Chapters
This segment of the Fragile X Syndrome report encloses the detailed analysis of marketed drugs and late stage (Phase-III and Phase-II) pipeline drugs. It also helps to understand the clinical trial details, expressive pharmacological action, agreements and collaborations, approval and patent details, advantages and disadvantages of each included drug and the latest news and press releases.
In the absence of any approved therapy for the treatment for Fragile X Syndrome, the current market size of Fragile X Syndrome is mainly dependent on various specialized therapies, such as speech and language therapy, occupational therapy, physical therapy and drug therapies. The most frequently prescribed therapeutic drug classes are psychotropic drugs, such as antidepressants, stimulants, tranquilizers, and antipsychotics, that are often used to treat disorders such as hyperactivity, anxiety, and inattention.
Interventional services, like speech-language therapy, occupational therapy, physical therapy, special education services, and behavior management are commonly utilized to address specific behaviors and developmental issues, and comorbid conditions, such as ASD, anxiety, attention-deficit/hyperactivity disorder (ADHD), seizures, and others.
Antipsychotic drugs generally are helpful in clinical settings to target irritability, aggression, mood instability, and perseverative behaviors in both male individuals and female individuals with Fragile X Syndrome. Risperidone is the most frequently used antipsychotic drug in the past and was effective clinically, with high response rates for aggressive behavior in older male individuals with Fragile X Syndrome and other irritable, aberrant, and undesired behaviors in young boys with Fragile X Syndrome and autistic traits. Aripiprazole is the second most frequently used atypical antipsychotic agent used in Fragile X Syndrome, with the highest overall response rate for an individual-antipsychotic and the lowest concern about weight gain.
However, these pharmacologic treatments target only the behavioral symptoms and not the cognitive/language impairments or the underlying brain deficits.
Several companies are working robustly on many new therapies, such as BPN14770 (Tetra Therapeutics), Cannabidiol (Zynerba Pharmaceuticals), OV101 (Ovid Therapeutics), Acamprosate (Confluence Pharmaceuticals), and Trofinetide (Neuren Pharmaceuticals) and others. These all are anticipated to enter the Fragile X Syndrome market by 2028.
Fragile X Syndrome Market Outlook
The Fragile X Syndrome market outlook of the report helps to build the detailed comprehension of the historic, current and forecasted trend of the market by analyzing the impact of current therapies on the market, unmet needs, drivers and barriers and demand of better technology.
According to DelveInsight, the market of Fragile X Syndrome in 7MM was found to be approximately USD 33.51 Million in 2017.
Fragile X Syndrome market size in the United States accounted for approx. 66% of the total market in 2017. The US led the market in 2017, followed by the EU5 and Japan respectively.
Fragile X Syndrome Drugs Uptake
This section focusses on the rate of uptake of the potential drugs recently launched in the market or will get launched in the market during the study period from 2017-2028. The analysis covers market uptake by drugs; patient uptake by therapies and sales of each drug.
Currently, the market of Fragile X Syndrome depends on off-label psychotropic drugs, such as antidepressants, stimulants, tranquilizers, antipsychotics and others.
Various therapies are in the late stage of development by various companies such as BPN14770 (Tetra Therapeutics), Cannabidiol (Zynerba Pharmaceuticals), OV101 (Ovid Therapeutics), Acamprosate (Confluence Pharmaceuticals), and Trofinetide (Neuren Pharmaceuticals) for patients with Fragile X Syndrome. All these therapies are expected to enter the market during the forecast period 2019-2028.
Fragile X Syndrome Report Insights
• Patient Population of Fragile X Syndrome
• Therapeutic Approaches
• Pipeline Analysis
• Market Size and Trends
• Market Opportunities
• Impact of upcoming Therapies
Fragile X Syndrome Report Key Strengths
• 10 Year Forecast
• 7MM Coverage
• Epidemiology Segmentation
• Drugs Uptake
• Highly Analyzed Market
• Key Cross Competition
Fragile X Syndrome Report Assessment
• Current Treatment Practices
• Unmet Needs
• Detailed Pipeline Product Profiles
• Market Attractiveness
• Market Drivers and Barriers
Key Benefits
• This DelveInsight report will help to develop Business Strategies by understanding the trends shaping and driving Fragile X Syndrome market
• Organize sales and marketing efforts by identifying the best opportunities for Fragile X Syndrome market
• To understand the future market competition in the Fragile X Syndrome market