脆弱性X症候群 (FXS)：市場分析、疫学、市場予測 (2030年まで)

世界の主要7ヶ国における脆弱性X症候群 (FXS) の市場規模は、2017年には3,380万米ドルに達しました。

当レポートでは、世界の主要7カ国 - 米国、英国、ドイツ、フランス、イタリア、スペイン、日本 - における脆弱性X症候群 (FXS) の疫学的動向と今後の見通しについて分析、疾患の概要や市場の基本構造、現在の治療法とアンメットニーズ、有病者数の動向見通し、年齢別・男女別・国別の詳細動向、上市済み／治験中の治療薬のプロファイル、世界全体／各国の市場規模、主な市場促進・抑制要因、といった情報を取りまとめてお届けいたします。

第1章 主な考察

第2章 脆弱性X症候群 (FXS)：患者の概略

• FXSの患者数：シェア分布 (2017年)
• FXSの患者数：シェア分布 (2030年)

第3章 エグゼクティブサマリー

第4章 SWOT分析

第5章 疾患の背景と概要：脆弱X症候群 (FXS)

• イントロダクション
• ゲノミクス
• 症状
• 臨床的特徴
• 診断
• FXSと新生児スクリーニング
  • 診断テストの推奨
  • 鑑別診断

第6章 認可済み施設

第7章 疫学と患者数：脆弱X症候群 (FXS)

• 主な調査結果
• 脆弱X症候群：主要7ヶ国で診断された有病例の合計
• 米国
  • 仮定と根拠
  • FXSの受診済み有病者総数
  • FXSの受診済み有病者数：男女別
  • FXSの受診済み有病者数：年齢別
• 欧州主要5ヶ国 (EU5)
• ドイツ
• フランス
• イタリア
• スペイン
• 英国
• 日本
  • 前提と根拠

第8章 現在の治療慣行

第9章 アンメットニーズ

第10章 新規の治療薬

• 主なクロス競合
• Zygel (カンナビジオール／ZYN002)：Zynerba Pharmaceuticals
• Acamprosate：Confluence Pharmaceuticals
• BPN14770：Tetra Therapeutics
• OV101（ガボクサドール）：Ovid Therapeutics
• トロフィタイド：Neuren Pharmaceuticals

第11章 その他の製品

• Metadoxine（MG01CI）：Alcobra Ltd./Arcturus Therapeutics
• Ganaxolone：Marinus Pharmaceuticals

第12章 市場規模：脆弱性X症候群 (FXS)

• 主な調査結果
• 主要7ヶ国の市場規模：全体的傾向
• 主要7ヶ国の市場規模：治療薬別

第13章 属性分析

第14章 コンジョイント分析

第15章 市場の展望：主要7ヶ国の場合

• 米国
  • FXSの市場規模：全体
  • FXSの市場規模：治療薬別
• 欧州主要5ヶ国 (EU5)
• ドイツ
• フランス
• イタリア
• スペイン
• 英国
• 日本

第16章 市場アクセスと償還

第17章 ケーススタディ

第18章 市場促進要因

第19章 市場抑制要因

第20章 業界の有識者 (KOL) の見解

第21章 付録

第22章 分析手法

第23章 DelveInsightのサービス内容

第24章 免責事項

第25章 DelveInsightについて

List of Tables

• Table 1: Summary of Fragile X Syndrome Market, Epidemiology, and Key Events (2017-2030)
• Table 2: Individuals for Whom Testing Should be Considered for FXS diagnosis
• Table 3: Total Diagnosed Prevalent cases of Fragile X Syndrome in the 7MM (2017-2030)
• Table 4: Total Diagnosed Prevalent Cases of Fragile X Syndrome (FXS)in the United States (2017-2030)
• Table 5: Gender-specific Cases of Fragile X Syndrome in the United States (2017-2030)
• Table 6: Age-specific Cases of Fragile X Syndrome in the United States (2017-2030)
• Table 7: Total Diagnosed Prevalent Cases of FXS in Germany (2017-2030)
• Table 8: Gender-specific Cases of Fragile X Syndrome in Germany (2017-2030)
• Table 9: Age-specific Cases of Fragile X Syndrome in Germany (2017-2030)
• Table 10: Total Diagnosed Prevalent Cases of FXS in France (2017-2030)
• Table 11: Gender-specific Cases of Fragile X Syndrome in France (2017-2030)
• Table 12: Ages-specific Cases of Fragile X Syndrome in France (2017-2030)
• Table 13: Total Diagnosed Prevalent Cases of FXS in Italy (2017-2030)
• Table 14: Gender-specific Cases of Fragile X Syndrome in Italy (2017-2030)
• Table 15: Age-specific Cases of Fragile X Syndrome in Italy (2017-2030)
• Table 16: Total Diagnosed Prevalent Cases of FXS in Spain (2017-2030)
• Table 17: Gender-specific Cases of Fragile X Syndrome in Spain (2017-2030)
• Table 18: Age-specific Cases of Fragile X Syndrome in Spain (2017-2030)
• Table 19: Total Diagnosed Prevalent Cases of FXS in the UK (2017-2030)
• Table 20: Gender-specific Cases of Fragile X Syndrome in the UK (2017-2030)
• Table 21: Age-specific Cases of Fragile X Syndrome in the UK (2017-2030)
• Table 22: Total Diagnosed Prevalent Cases of FXS in Japan (2017-2030)
• Table 23: Gender-specific Cases of Fragile X Syndrome in Japan (2017-2030)
• Table 24: Age-specific Cases of Fragile X Syndrome in Japan (2017-2030)
• Table 25: Summary of medical problems and management
• Table 26: Comparison of Emerging Drugs
• Table 27: Zygel, Clinical Trial Description, 2020
• Table 28: Acamprosate, Clinical Trial Description, 2020
• Table 29: BPN14770, Clinical Trial Description, 2020
• Table 30: OV101; Clinical Trial Description, 2020
• Table 31: Trofinetide, Clinical Trial Description, 2020
• Table 32: Metadoxine, Clinical Trial Description, 2020
• Table 33: Ganaxolone, Clinical Trial Description, 2020
• Table 34: Total Market Size of Fragile X Syndrome in the 7MM, in USD Million (2017-2030)
• Table 35: Total Market Size of FXS by Therapies in the 7MM, in USD Million (2017-2030)
• Table 36: Conjoint Analysis for Zygel
• Table 37: Conjoint Analysis for Acamprosate
• Table 38: Conjoint Analysis for BPN14770
• Table 39: Conjoint Analysis for OV101
• Table 40: Conjoint Analysis for Trofinetide
• Table 41: Total Market size of Fragile X Syndrome in the US (2017-2030)
• Table 42: Total Market Size of FXS by Therapies in the US, in USD Million (2017-2030)
• Table 43: Total Market size of Fragile X Syndrome in Germany (2017-2030)
• Table 44: Total Market Size of FXS by Therapies in Germany, in USD Million (2017-2030)
• Table 45: Total Market size of Fragile X Syndrome in France (2017-2030)
• Table 46: Total Market Size of FXS by Therapies in France, in USD Million (2017-2030)
• Table 47: Total Market size of Fragile X Syndrome in Italy (2017-2030)
• Table 48: Total Market Size of FXS by Therapies in Italy, in USD Million (2017-2030)
• Table 49: Total Market size of Fragile X Syndrome in Spain (2017-2030)
• Table 50: Total Market Size of FXS by Therapies in Spain, in USD Million (2017-2030)
• Table 51: Total Market size of Fragile X Syndrome in the UK (2017-2030)
• Table 52: Total Market Size of FXS by Therapies in the UK, in USD Million (2017-2030)
• Table 53: Total Market size of Fragile X Syndrome in Japan (2017-2030)
• Table 54: Total Market Size of FXS by Therapies in Japan, in USD Million (2017-2030)

List of Figures

• Figure 1: Fragile X Syndrome (FXS) SWOT Analysis
• Figure 2: Behavioral features of FXS
• Figure 3: Physical features of FXS
• Figure 4: Use of molecular techniques for FXS diagnosis
• Figure 5: Total Diagnosed Prevalent cases of Fragile X Syndrome in the 7MM (2017-2030)
• Figure 6: Total Diagnosed Prevalent Cases of Fragile X Syndrome (FXS) in the United States (2017-2030)
• Figure 7: Gender-specific Cases of Fragile X Syndrome (FXS) in the United States (2017-2030)
• Figure 8: Age-specific Cases of Fragile X Syndrome (FXS) in the United States (2017-2030)
• Figure 9: Total Diagnosed Prevalent Cases of FXS in Germany (2017-2030)
• Figure 10: Gender-specific Cases of Fragile X Syndrome (FXS) in Germany (2017-2030)
• Figure 11: Age-specific Cases of Fragile X Syndrome (FXS) in Germany (2017-2030)
• Figure 12: Total Diagnosed Prevalent Cases of FXS in France (2017-2030)
• Figure 13: Gender-specific Cases of Fragile X Syndrome (FXS) in France (2017-2030)
• Figure 14: Age-specific Cases of Fragile X Syndrome (FXS) in France (2017-2030)
• Figure 15: Total Diagnosed Prevalent Cases of FXS in Italy (2017-2030)
• Figure 16: Gender-specific Cases of Fragile X Syndrome (FXS) in Italy (2017-2030)
• Figure 17: Age-specific Cases of Fragile X Syndrome (FXS) in Italy (2017-2030)
• Figure 18: Total Diagnosed Prevalent Cases of FXS in Spain (2017-2030)
• Figure 19: Gender-specific Cases of Fragile X Syndrome (FXS) in Spain (2017-2030)
• Figure 20: Age-specific Cases of Fragile X Syndrome (FXS) in Spain (2017-2030)
• Figure 21: Total Diagnosed Prevalent Cases of FXS in the UK (2017-2030)
• Figure 22: Gender-specific Cases of Fragile X Syndrome (FXS) in the UK (2017-2030)
• Figure 23: Age-specific Cases of Fragile X Syndrome (FXS) in the UK (2017-2030)
• Figure 24: Total Diagnosed Prevalent Cases of FXS in Japan (2017-2030)
• Figure 25: Gender-specific Cases of Fragile X Syndrome (FXS) in Japan (2017-2030)
• Figure 26: Age-specific Cases of Fragile X Syndrome (FXS) in Japan (2017-2030)
• Figure 27: Unmet Needs
• Figure 28: Future Needs
• Figure 29: Total Market Size of Fragile X Syndrome in the 7MM in USD Million (2017-2030)
• Figure 30: Total Market Size of FXS by Therapies in the 7MM in USD Million (2017-2030)
• Figure 31: Market size of Fragile X Syndrome in the US (2017-2030)
• Figure 32: Total Market Size of FXS by Therapies in the US, in USD Million (2017-2030)
• Figure 33: Market size of Fragile X Syndrome in Germany (2017-2030)
• Figure 34: Total Market Size of FXS by Therapies in Germany, in USD Million (2017-2030)
• Figure 35: Market size of Fragile X Syndrome in France (2017-2030)
• Figure 36: Total Market Size of FXS by Therapies in France, in USD Million (2017-2030)
• Figure 37: Market size of Fragile X Syndrome in Italy (2017-2030)
• Figure 38: Total Market Size of FXS by Therapies in Italy, in USD Million (2017-2030)
• Figure 39: Market size of Fragile X Syndrome in Spain (2017-2030)
• Figure 40: Total Market Size of FXS by Therapies in Spain, in USD Million (2017-2030)
• Figure 41: Market size of Fragile X Syndrome in the UK (2017-2030)
• Figure 42: Total Market Size of FXS by Therapies in the UK, in USD Million (2017-2030)
• Figure 43: Market size of Fragile X Syndrome in Japan (2017-2030)
• Figure 44: Total Market Size of FXS by Therapies in Japan, in USD Million (2017-2030)
• Figure 45: Market Drivers
• Figure 46: Funding Organizations for FXS
• Figure 47: Market Barriers

DelveInsight's 'Fragile X Syndrome (FXS)-Market Insights, Epidemiology and Market Forecast-2030' report delivers an in-depth understanding of the Fragile X Syndrome (FXS), historical and forecasted epidemiology as well as the Fragile X Syndrome (FXS) market trends in the United States, EU5 (Germany, Spain, Italy, France, and United Kingdom) and Japan.

The Fragile X Syndrome (FXS) market report provides current treatment practices, emerging drugs, and market share of the individual therapies, current and forecasted 7MM Fragile X Syndrome (FXS) market size from 2017 to 2030. The report also covers current Fragile X Syndrome (FXS) treatment practice/algorithm, market drivers, market barriers and unmet medical needs to curate the best of the opportunities and assesses the underlying potential of the market.

Geography Covered:

• The United States
• EU5 (Germany, France, Italy, Spain, and the United Kingdom)
• Japan

Study Period: 2017-2030

Fragile X Syndrome (FXS) Disease Understanding and Treatment Algorithm

Fragile X Syndrome (FXS) Overview

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. It 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 FXS 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 FXS will present with language delay, sensory hyperarousal and anxiety.

FXS causes intellectual disability, behavioral and learning challenges and various physical characteristics. Though FXS occurs in both genders, males are more frequently affected than females, and generally with greater severity. Life expectancy is not affected in people with FXS because there are usually no life-threatening health concerns associated with the condition.

Fragile X Syndrome (FXS) Diagnosis

Initially, the diagnosis of FXS was made through karyotype, which allowed the observation of the distal narrowing of the long arm of the X chromosome in the band 27.3 (Xq27.3-23.8) using the light microscope. The findings of distal constrictions can be done in different chromosomes, and are known as fragile sites, from where FXS is named. Nowadays, there are several molecular tests available for the diagnosis of FXS, which are far more sensitive and specific than the karyotype. Besides allowing the diagnosis of patients with the FM and FXS, these tests allow the identification of carriers of the PM, which are individuals typically with a normal IQ. Still, the female carriers have a high risk of having children with FXS. There are molecular tests that allow the quantification of messenger RNA (mRNA) and FMRP protein, allowing a better understanding of the physiopathology of the disease by correlating the results to the phenotype of the FM and PM patients.

Polymerase chain reaction (PCR) and Southern blot are the routine tests for the DNA diagnosis of FXS, which allow determining the number of CGG repeats and the methylation status of the FMR1 gene. The PCR through the use of specific primers for the FMR1 gene allows the amplification of the region that contains the CGG repeat and, can identify patients with an expanded FMR1 allele particularly in the premutation but also in the full mutation range.

Fragile X Syndrome (FXS) Treatment

There is no single treatment for FXS, but there are treatments that help minimize the symptoms of the condition. Current approaches to therapy for FXS are all symptom-based, and few controlled trials have been performed to determine their effectiveness. Psychopharmacologic intervention should be combined with other supportive strategies, including speech therapy, sensory integration, occupational therapy, individualized educational plans, and tailored behavioral interventions to maximize functioning.

The treatment regimen of FXS include the use of stimulants, selective serotonin reuptake inhibitors (SSRIs), atypical antipsychotics and alpha agonists. Medications such as methylphenidate (Ritalin), guanfacine (Intuniv), clonidine (Catapres), selective serotonin reuptake inhibitor (SSRI), such as sertraline (Zoloft), escitalopram (Lexapro), duloxetine (Cymbalta), and paroxetine (Pail, Pexeva) are used.

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.

It covers the details of conventional and current medical therapies available in the Fragile X Syndrome (FXS) market for the treatment of the various types of Fragile X Syndrome (FXS). It also provides the treatment guidelines and algorithms of the United States and Europe. The DelveInsight's "Fragile X Syndrome (FXS) market report" gives a thorough understanding of Fragile X Syndrome (FXS) by including details such as disease definition, comorbidities, causes, risk factors, pathogenesis, and diagnosis.

Fragile X Syndrome (FXS) Epidemiology

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 Diagnosed Prevalence of Fragile X Syndrome in 7MM, and Age-specific Diagnosed Prevalence of Fragile X Syndrome, the scenario of Fragile X Syndrome in the 7MM covering the United States, EU5 countries (Germany, Spain, Italy, France and the United Kingdom) and Japan from 2017 to 2030.

Key Findings

This section provides glimpses of the Fragile X Syndrome (FXS) epidemiology in the 7MM.

• The prevalent population of Fragile X Syndrome in 7MM is expected to increase at a CAGR of 0.62% for the study period, i.e. 2017-2030.
• Among the gender-specific prevalent cases, in 2017, there were 40,102 cases of Fragile X Syndrome (FXS) in males and 27,552 cases in females, in the US.
• The United States accounts for the highest diagnosed prevalent cases of Fragile X Syndrome with 67,654 cases in 2017.

Country Wise-Fragile X Syndrome (FXS) Epidemiology

The epidemiology segment also provides the Fragile X Syndrome (FXS) epidemiology data and findings across the United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom) and Japan.

Fragile X Syndrome (FXS) Drug Chapters

This segment of the Fragile X Syndrome report encloses the detailed analysis of 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.

Fragile X Syndrome (FSX) Emerging Drugs

Zygel (Cannabidiol/ZYN002): Zynerba Pharmaceuticals

Zygel (Cannabidiol gel [ZYN002]) is the first, and only patent-protected synthetic cannabidiol (CBD) formulated as a permeation-enhanced gel for transdermal delivery. It is a cannabinoid receptor CB1 & CB2 inverse agonist The company is conducting phase II/ III trials for the treatment of FXS, and it is also being studied in children with, osteoarthritis and adult epilepsy patients with focal seizures. The product has received Fast track and Orphan drug designation for the treatment of FXS.

Products detail in the report

Acamprosate: Confluence Pharmaceuticals

Acamprosate is a novel, clinically discovered treatment that acts as an NMDA receptor inhibitor GABA receptor agonist; Glutamaterigic antagonist at mGlur5 receptor. It is designed for the treatment of core social and communication impairments caused by FXS and Autism Spectrum Disorders. The company has targeted important functionalities-talking, thinking, social interaction, and self-care. Currently, the company is conducting phase II/III trial for the treatment of FXS. In March 2015, Acamprosate received Orphan drug designation from both, US FDA and EMA for FXS.

Products detail in the report

BPN14770: Tetra Therapeutics

BPN14770 is a novel therapeutic agent that selectively inhibits phosphodiesterase-4D (PDE4D) to enhance the early and late stages of memory formation. Based on these outcomes, the company is conducting the Phase II trial for the Treatment of FXS and plans to announce top-line results of Phase II trials in 2020. BPN14770 has received Orphan Drug Designation from FDA for the treatment of FXS.

Products detail in the report

OV101 (gaboxadol): Ovid Therapeutics

OV101 also known as gaboxadol, is an oral, first-in-class, potent, δ-selective, extrasynaptic GABAA receptor agonist (δSEGA). Currently, the drug is in the phase II stage of development for the treatment of Fragile X Syndrome. The drug is the only clinically tested δ-selective direct-acting GABAA receptor agonist that can bind to and activate this specific δ-subset of GABA receptors, found in the extrasynaptic space outside of the synapse. In March 2018, Ovid Therapeutics announced that the US FDA had granted Fast Track designation to OV101 for the treatment of FXS.

Products detail in the report

Trofinetide: Neuren Pharmaceuticals

Neuren Pharmaceuticals is developing Trofinetide (NNZ-2566), a nuclear factor (erythroid-derived 2)-like 2 (Nrf2) activator. NNZ2566 normalizes the translocation of Nrf2 to the nucleus, inducing expression of numerous oxidative stress-related genes including NQO1, GST-α1, and EH and has a knockdown effect on E-cadherin. Thus, the drug activates the Nrf2/ARE pathway with a potential benefit across multiple symptoms that could be associated with the pathobiological processes underlying the disease.

Products detail in the report

Fragile X Syndrome (FXS) Market Outlook

FXS is the known leading cause of inherited intellectual disability and an autism spectrum disorder. Currently, there is no approved treatment for FXS. The treatment relies on the associated symptoms of the disease which are typically managed using pharmacologic interventions, such as stimulants for attention deficit and hyperactivity, selective serotonin reuptake inhibitors (SSRIs) for anxiety, antipsychotic drugs for aggression and mood instability, and melatonin for sleep. In boys with FXS, the most frequently used medications are stimulants. These medications are targeted toward the symptoms of hyperactivity, impulsivity, and distractibility and can be quite helpful in these areas. Despite being the most common medication in FXS, the efficacy of these drugs and their side effects vary for each individual. Stimulants may induce irritability and other behavioral problems in children <5 years of age in such cases, non-stimulant medications may be helpful.

The FXS market outlook of the report helps to build a detailed comprehension of the historical, current and forecasted FXS market trends by analyzing the impact of current therapies on the market, unmet needs, drivers and barriers, and demand of better technology.

This segment gives a thorough detail FXS market trend of each marketed drug and late-stage pipeline therapy by evaluating their impact based on the annual cost of therapy, inclusion and exclusion criteria's, mechanism of action, compliance rate, growing need of the market, increasing patient pool, covered patient segment, expected launch year, competition with other therapies, brand value, their impact on the market and view of the key opinion leaders. The calculated market data are presented with relevant tables and graphs to give a clear view of the market at first sight.

According to DelveInsight, Fragile X Syndrome (FXS) 7MM market is expected to change in the study period 2017-2030.

Key Findings

This section includes a glimpse of the Fragile X Syndrome (FXS) 7MM market.

• According to Delveinsight estimates, the United States accounts for the largest market size of Fragile X Syndrome (FXS), in comparison to Japan and EU5 (the United Kingdom, Germany, Italy, France, and Spain).
• The market size of FXS in the seven major markets was USD 33.8 Million in 2017.
• Even with substantial support and therapies, individuals with FXS continue to present with significant impairments in their functioning throughout their life. There remains a great need for safe and effective treatments for FXS, particularly for targeted treatments that surpass symptom management and address the pathophysiologic abnormalities that underlie the most common manifestations.

The United States Market Outlook

This section provides the totalFragile X Syndrome (FXS) market size and market size by therapies in the United States.

EU-5 Market Outlook

The total Fragile X Syndrome (FXS) market size and market size by therapies in Germany, France, Italy, Spain, and the United Kingdom are provided in this section.

Japan Market Outlook

The total Fragile X Syndrome (FXS) market size and market size by therapies in Japan are provided.

Fragile X Syndrome (FXS) Drugs Uptake

This section focusses on the rate of uptake of the potential drugs expected to get launched in the market during the study period 2017-2030. The analysis covers Fragile X Syndrome (FXS) market uptake by drugs; patient uptake by therapies; and sales of each drug.

It helps in understanding the drugs with the most rapid uptake, reasons behind the maximal use of new drugs and allows the comparison of the drugs based on market share and size which again will be useful in investigating factors important in market uptake and in making financial and regulatory decisions.

Fragile X Syndrome (FXS) Development Activities

The report provides insights into different therapeutic candidates in phase II, and phase III stage. It also analyzes key players involved in developing targeted therapeutics.

Pipeline Development Activities

The report covers the detailed information of collaborations, acquisition and merger, licensing and patent details for Fragile X Syndrome (FXS) emerging therapies.

Competitive Intelligence Analysis

We perform competitive and market Intelligence analysis of the Fragile X Syndrome (FXS) market by using various competitive intelligence tools that include-SWOT analysis, PESTLE analysis, Porter's five forces, BCG Matrix, Market entry strategies, etc. The inclusion of the analysis entirely depends upon the data availability.

Scope of the Report:

• The report covers the descriptive overview of Fragile X Syndrome (FXS), explaining its causes, signs and symptoms, pathogenesis and currently available therapies.
• Comprehensive insight has been provided into the fragile X Syndrome (FXS) epidemiology and treatment.
• Additionally, an all-inclusive account of both the current and emerging therapies for Fragile X Syndrome (FXS) is provided, along with the assessment of new therapies, which will have an impact on the current treatment landscape.
• A detailed review of Fragile X Syndrome (FXS) market; historical and forecasted is included in the report, covering the 7MM drug outreach.
• The report provides an edge while developing business strategies, by understanding trends shaping and driving the 7MMFragile X Syndrome (FXS) market.

Report Highlights:

• The market is expected to be driven positively due to funding and cooperation of various organizations, identification of potential targets by animal models, strengthening the molecular basis of FXS. However few factors such as failure of clinical trials, expensive diagnostic procedures might hamper the market.
• The 7MM market is anticipated to increase at a CAGR of 29.83% for the forecasted period (2020-2030).
• In the coming years, Fragile X Syndrome (FXS) market is set to change due to the rising awareness of the disease, and incremental healthcare spending across the world; which would expand the size of the market to enable the drug manufacturers to penetrate more into the market.
• The companies and academics are working to assess challenges and seek opportunities that could influence Fragile X Syndrome (FXS) R&D. The therapies under development are focused on novel approaches to treat/improve the disease condition.
• Major players are involved in developing therapies for Fragile X Syndrome (FXS). Launch of emerging therapies will significantly impact the Fragile X Syndrome (FXS) market. In the present scenario, several companies are actively working in this space while five companies are leading with the major products.
• Our in-depth analysis of the pipeline assets across different stages of development (phase III and phase II), different emerging trends and comparative analysis of pipeline products with detailed clinical profiles, key competitors, launch date along with product development activities will support the clients in the decision-making process regarding their therapeutic portfolio by identifying the overall scenario of the research and development activities.

Fragile X Syndrome (FXS) Report Insights

• Patient Population
• Therapeutic Approaches
• Fragile X Syndrome (FXS) Pipeline Analysis
• Fragile X Syndrome (FXS) Market Size and Trends
• Market Opportunities
• Impact of upcoming Therapies

Fragile X Syndrome (FXS) Report Key Strengths

• 11-year Forecast
• 7MM Coverage
• Fragile X Syndrome (FXS) Epidemiology Segmentation
• Key Cross Competition
• Highly Analyzed Market
• Drugs Uptake

Fragile X Syndrome (FXS) Report Assessment

• Current Treatment Practices
• Unmet Needs
• Pipeline Product Profiles
• Market Attractiveness
• Market Drivers and Barriers

Key Questions

Market Insights:

• What was the Fragile X Syndrome (FXS) market share (%) distribution in 2017 and how it would look like in 2030?
• What would be the Fragile X Syndrome (FXS) total market size as well as market size by therapies across the 7MM during the forecast period (2020-2030)?
• What are the key findings pertaining to the market across the 7MM and which country will have the largest FXS market size during the forecast period (2020-2030)?
• At what CAGR, the Fragile X Syndrome (FXS) market is expected to grow at the 7MM level during the forecast period (2020-2030)?
• What would be the Fragile X Syndrome (FXS) market outlook across the 7MM during the forecast period (2020-2030)?
• What would be the Fragile X Syndrome (FXS) market growth till 2030, and what will be the resultant market size in the year 2030?
• How would the market drivers, barriers and future opportunities affect the market dynamics and subsequent analysis of the associated trends?

Epidemiology Insights:

• What is the disease risk, burden and unmet needs of (FXS)?
• What is the historical Fragile X Syndrome (FXS) patient pool in the United States, EU5 (Germany, France, Italy, Spain, and the UK) and Japan?
• What would be the forecasted patient pool of FXS at the 7MM level?
• What will be the growth opportunities across the 7MM concerning the patient population pertaining to Fragile X Syndrome (FXS)?
• Out of the countries mentioned above, which country would have the highest prevalent population of Fragile X Syndrome (FXS) during the forecast period (2020-2030)?
• At what CAGR the population is expected to grow across the 7MM during the forecast period (2020-2030)?

Current Treatment Scenario, Marketed Drugs and Emerging Therapies:

• What are the current options for the treatment of Fragile X Syndrome (FXS) along with the approved therapy?
• What are the current treatment guidelines for the treatment of Fragile X Syndrome (FXS) in the US and Europe?
• What are theFragile X Syndrome (FXS) marketed drugs and their MOA, regulatory milestones, product development activities, advantages, disadvantages, safety and efficacy, etc.?
• How many companies are developing therapies for the treatment of Fragile X Syndrome (FXS)?
• How many therapies are developed by each company for the treatment of Fragile X Syndrome (FXS)?
• How many emerging therapies are in the mid-stage and late stage of development for the treatment of Fragile X Syndrome (FXS)?
• What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the Fragile X Syndrome (FXS) therapies?
• What are the recent novel therapies, targets, mechanisms of action and technologies developed to overcome the limitation of existing therapies?
• What are the clinical studies going on for Fragile X Syndrome (FXS) and their status?
• What are the key designations that have been granted for the emerging therapies for Fragile X Syndrome (FXS)?
• What are the 7MM historical and forecasted market of Fragile X Syndrome (FXS)?

Reasons to buy:

• The report will help in developing business strategies by understanding trends shaping and driving the Fragile X Syndrome (FXS).
• To understand the future market competition in the Fragile X Syndrome (FXS) market and Insightful review of the key market drivers and barriers.
• Organize sales and marketing efforts by identifying the best opportunities for Fragile X Syndrome (FXS) in the US, Europe (Germany, Spain, Italy, France, and the United Kingdom) and Japan.
• Identification of strong upcoming players in the market will help in devising strategies that will help in getting ahead of competitors.
• Organize sales and marketing efforts by identifying the best opportunities for Fragile X Syndrome (FXS) market.
• To understand the future market competition in the Fragile X Syndrome (FXS) market.

List of Companies

• Zynerba Pharmaceuticals
• Confluence Pharmaceuticals
• Tetra Therapeutics
• Ovid Therapeutics
• Neuren Pharmaceuticals

Table of Contents

1 Key Insights

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

• 2.1 Total Patient Share (%) Distribution of FXS in 2017
• 2.2 Total Patient Share (%) Distribution of FXS in 2030

3 Executive Summary

4 SWOT Analysis

5 Disease Background and Overview: Fragile X Syndrome (FXS)

• 5.1 Introduction
• 5.2 Genomics
• 5.3 Symptoms
• 5.4 Clinical Features
• 5.5 Diagnosis
• 5.6 Fragile X and Newborn Screening
  • 5.6.1 Recommendation for Diagnostic Testing
  • 5.6.2 Differential Diagnosis

6 Recognized Establishments

7 Epidemiology and Patient Population: Fragile X Syndrome

• 7.1 Key Findings
• 7.2 Total Diagnosed Prevalent cases of Fragile X Syndrome in the 7MM
• 7.3 United States
  • 7.3.1 Assumptions and Rationale
  • 7.3.1 Total Diagnosed Prevalent Cases of Fragile X Syndrome (FXS) in the United States
  • 7.3.2 Gender-specific Cases of Fragile X Syndrome (FXS) in the United States
  • 7.3.3 Age-specific Cases of Fragile X Syndrome (FXS) in the United States
• 7.4 EU5
  • 7.4.1 Assumptions and Rationale
• 7.5 Germany
  • 7.5.1 Total Diagnosed Prevalent Cases of Fragile X Syndrome (FXS) in Germany
  • 7.5.2 Gender-specific Cases of Fragile X Syndrome (FXS) in Germany
  • 7.5.3 Age-specific Cases of Fragile X Syndrome (FXS) in Germany
• 7.6 France
  • 7.6.1 Total Diagnosed Prevalent Cases of Fragile X Syndrome (FXS) in France
  • 7.6.2 Gender-specific Cases of Fragile X Syndrome (FXS) in France
  • 7.6.3 Age-specific Cases of Fragile X Syndrome (FXS) in France
• 7.7 Italy
  • 7.7.1 Total Diagnosed Prevalent Cases of Fragile X Syndrome (FXS) in Italy
  • 7.7.2 Gender-specific Cases of Fragile X Syndrome (FXS) in Italy
  • 7.7.3 Age-specific Cases of Fragile X Syndrome (FXS) in Italy
• 7.8 Spain
  • 7.8.1 Total Diagnosed Prevalent Cases of Fragile X Syndrome (FXS) in Spain
  • 7.8.2 Gender-specific Cases of Fragile X Syndrome (FXS) in Spain
  • 7.8.3 Age-specific Cases of Fragile X Syndrome (FXS) in Spain
• 7.9 United Kingdom (UK)
  • 7.9.1 Total Diagnosed Prevalent Cases of Fragile X Syndrome (FXS) in the United Kingdom
  • 7.9.2 Gender-specific Cases of Fragile X Syndrome (FXS) in the United Kingdom
  • 7.9.3 Age-specific Cases of Fragile X Syndrome (FXS) in the United Kingdom
• 7.1 Japan
  • 7.10.1 Assumption and Rationale
  • 7.10.3 Total Diagnosed Prevalent Cases of Fragile X Syndrome (FXS) in Japan
  • 7.10.4 Gender-specific Cases of Fragile X Syndrome (FXS) in Japan
  • 7.10.5 Age-specific Cases of Fragile X Syndrome (FXS) in Japan

8 Current Treatment

9 Unmet Need

10 Emerging Drugs

• 10.1 Key Cross Competition
• 10.2 Zygel (Cannabidiol/ZYN002): Zynerba Pharmaceuticals
  • 10.2.1 Product Description
  • 10.2.2 Other Developmental Activities
  • 10.2.3 Clinical Development
  • 10.2.4 Safety and Efficacy
  • 10.2.5 Product Profile
• 10.3 Acamprosate: Confluence Pharmaceuticals
  • 10.3.1 Product Description
  • 10.3.2 Other Developmental Activities
  • 10.3.3 Clinical Development
  • 10.3.4 Safety and Efficacy
  • 10.3.5 Product Profile
• 10.4 BPN14770: Tetra Therapeutics
  • 10.4.1 Product Description
  • 10.4.2 Other Developmental Activities
  • 10.4.3 Clinical Development
  • 10.4.4 Safety and Efficacy
  • 10.4.5 Product Profile
• 10.5 OV101 (gaboxadol): Ovid Therapeutics
  • 10.5.1 Product Description
  • 10.5.2 Other Developmental Activities
  • 10.5.3 Clinical Development
  • 10.5.4 Safety and Efficacy
  • 10.5.5 Product Profile
• 10.6 Trofinetide: Neuren Pharmaceuticals
  • 10.6.1 Product Description
  • 10.6.2 Other Developmental Activities
  • 10.6.3 Clinical Development
  • 10.6.4 Safety and Efficacy
  • 10.6.5 Product Profile

11 Other Products

• 11.1 Metadoxine (MG01CI): Alcobra Ltd./Arcturus Therapeutics
  • 11.1.1 Product Description
  • 11.1.2 Other Developmental Activities
  • 11.1.3 Clinical Development
  • 11.1.4 Safety and Efficacy
  • 11.1.5 Product Profile
• 11.2 Ganaxolone: Marinus Pharmaceuticals
  • 11.2.1 Product Description
  • 11.2.2 Other Developmental Activities
  • 11.2.3 Clinical Development
  • 11.2.4 Safety and Efficacy
  • 11.2.5 Product Profile

12 Market Size: Fragile X Syndrome

• 12.1 Key Findings
• 12.2 Total Market Size of Fragile X Syndrome in the 7MM
• 12.3 Total Market Size of Fragile X Syndrome by Therapies in the 7MM

13 Attribute Analysis

14 Conjoint Analysis

15 7MM Market Outlook

• 15.1 United States
  • 15.1.1 Total Market size of Fragile X Syndrome in the US
  • 15.1.2 Total Market Size of Fragile X Syndrome by Therapies in the US
• 15.2 EU5
• 15.3 Germany
  • 15.3.1 Total Market size of Fragile X Syndrome in Germany
  • 15.3.2 Total Market Size of Fragile X Syndrome by Therapies in Germany
• 15.4 France
  • 15.4.1 Total Market size of Fragile X Syndrome in France
  • 15.4.2 Total Market Size of Fragile X Syndrome by Therapies in France
• 15.5 Italy
  • 15.5.1 Total Market size of Fragile X Syndrome in Italy
  • 15.5.2 Total Market Size of Fragile X Syndrome by Therapies in Italy
• 15.6 Spain
  • 15.6.1 Total Market size of Fragile X Syndrome in Spain
  • 15.6.2 Total Market Size of Fragile X Syndrome by Therapies in Spain
• 15.7 United Kingdom
  • 15.7.1 Total Market size of Fragile X Syndrome in the UK
  • 15.7.2 Total Market Size of Fragile X Syndrome by Therapies in the UK
• 15.8 Japan
  • 15.8.1 Total Market size of Fragile X Syndrome in Japan
  • 15.8.2 Total Market Size of Fragile X Syndrome by Therapies in Japan

16 Market Access and Reimbursement

17 Case Reports

• 17.1 Fragile X-associated neuropsychiatric disorders
• 17.2 Prepubertal Metformin Treatment in Fragile X Syndrome Alleviated Macroorchidism
• 17.3 Fragile X Associated Primary Ovarian Insufficiency (FXPOI): Case report and literature review

18 Market Drivers

19 Market Barriers

20 KOL Views

21 Appendix

22 Report Methodology

23 DelveInsight Capabilities

23 Disclaimer

25 About DelveInsight