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Fragile X Syndrome Market Size and Trend Report including Epidemiology and Pipeline Analysis, Competitor Assessment, Unmet Needs, Clinical Trial Strategies and Forecast, 2021 - 2030

  • ID: 5562914
  • Drug Pipelines
  • February 2022
  • Region: Global
  • 60 Pages
  • GlobalData
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Fragile X syndrome (FXS) (ICD-10: Q99.2) is an X-linked genetic condition and is the most common cause of inherited learning disability. FXS is caused by expansion of a cytosine, guanine, and guanine (CGG) trinucleotide repeats in the 5’ untranslated region of the fragile X mental retardation 1 (FMR1) gene. This expansion results in transcriptional silencing of the FMR1 gene with a subsequent reduction or absence of fragile X mental retardation protein (FMRP) expression. FMRP is an important mediator in dendritic maturity and synaptic plasticity.

FXS is a lifelong disorder associated with a spectrum of developmental and behavioral issues, including psychiatric symptoms (anxiety, attention deficit hyperactivity disorder [ADHD], and poor eye contact) and neurologic symptoms (seizures and movement disorders). Other common features of the syndrome include characteristic physical attributes (large and prominent ears, long face, and flat feet) as well as deafness, due to the high recurrence of ear infections. The severity of these symptoms is dependent on the amount of FMRP protein that is produced, which in turn is dependent on both the number of CGG repeats as well as the degree of methylation in the FMR1 gene. As females have two X chromosomes and thus two copies of the FMR1 gene, females often have milder symptoms than males.

The two clinically and commercially relevant segments considered for the classification of FXS in this report are segmented into three groups by age: pediatric patients aged 0-11 years, adolescent patients aged 12-17, and adult patients aged 18+.

This report covers 2MM (US and Germany) and provides an Excel-based forecast model for the Fragile X Syndrome market through 2030.

Key Highlights

  • Forecasts include two countries
  • Forecasts cover three time points: base year, 5-year, and 10-year
  • There are currently no approved therapies available for FXS, so prescribed treatments are exclusively off-label products that target individual symptoms of the disease. The most frequently used drug classes are: selective serotonin reuptake inhibitors (SSRIs), psychostimulants, antipsychotic agents, anticonvulsant agents, and antihypertensive anxiolytics.
  • The current treatment options that target FXS symptoms of irritability and aggression are often sedating and diminish patients’ interaction with their environment. This prevents disruptive behavior but negatively affects the patients’ quality of life. There is a need for interventions that suppress aggressive behavior without side effects.
  • Products in Phase I and II stages of development account for 89.7% of the FXS pipeline, while limited development is seen in Phase III trials with only one drug currently in development.
  • The FXS market will exhibit significant growth between 2020 and 2030, driven by the entry of four pipeline agents. Notably, the sales of high-priced pipeline agents, Zygel, zatolmilast, and trofinetide represent the most impactful drivers of growth.


  • Overview of FXS, including epidemiology, symptoms, diagnosis, and disease management.
  • Annualized FXS therapeutics market revenue, cost of therapy per patient, and treatment usage patterns in three patient segments (pediatric patients aged 0-11 years, adolescent patients aged 12-17, and adult patients aged 18+) forecast from 2020 to 2030.
  • Key topics covered include strategic competitor assessment, market characterization, unmet needs, clinical trial mapping, and implications of these factors for the FXS therapeutics market.
  • Pipeline analysis: comprehensive data assessing emerging trends and mechanisms of action under development for FXS treatment. The most promising candidates in Phase III and Phase IIb development are profiled.
  • Analysis of the current and future market competition in the global FXS therapeutics market. Insightful review of the key industry drivers, restraints and challenges. Each trend is independently researched to provide qualitative analysis of its implications.

Reasons to Buy

  • Develop and design your in-licensing and out-licensing strategies through a review of pipeline products and technologies, and by identifying the companies with the most robust pipeline.
  • Develop business strategies by understanding the trends shaping and driving the global FXS therapeutics market.
  • Drive revenues by understanding the key trends, innovative products and technologies, market segments, and companies likely to impact the global FXS therapeutics market in the future.
  • Formulate effective sales and marketing strategies by understanding the competitive landscape and by analyzing the performance of various competitors.
  • Identify emerging players with potentially strong product portfolios and create effective counter-strategies to gain a competitive advantage.
  • Organize your sales and marketing efforts by identifying the market categories and segments that present maximum opportunities for consolidations, investments and strategic partnerships.

Table of Contents

1 Executive Summary
1.1 Executive Summary
2 Disease Overview
2.1 Overview of Fragile X Syndrome
2.2 Fragile X Syndrome SWOT Analysis
2.3 Classification of Fragile X Syndrome
3 Epidemiology
3.1 Diagnosed Prevalent Cases of Fxs, Men and Women, 2020-30
3.2 Sources and Methodology
4 Current Treatment Options
4.1 Treatment Paradigm
4.2 Current Treatment Options
4.3 Product Profile: Ssris (Fluoxetine, Sertraline, Citalopram, Escitalopram)
4.4 Product Profile: Other Antidepressants (Venlafaxine, Bupropion, Trazadone)
4.5 Product Profile: Psychostimulants (Methylphenidate Hydrochloride)
4.6 Product Profile: Atypical Antipsychotics (Aripiprazole, Risperidone)
4.7 Product Profile: Anticonvulsants (Lamotrigine, Carbamazepine, Sodium Valproate)
4.8 Product Profile: Antihypertensive Anxiolytics (Clonidine, Guanfacine)
4.9 Product Profile: Benzodiazepines (Clonazepam, Diazepam)
4.10 Product Profile: Lithium (Lithium Carbonate)
5 Unmet Needs and Opportunities
5.1 Unmet Needs in Fragile X Syndrome
6 R&D Strategies
6.1 Trends in Deal-Making in Fxs
6.2 Trends in Deal-Making in Fxs
7 Pipeline Assessment
7.1 Fragile X Syndrome Pipeline Overview
7.2 Late-Stage Pipeline Agents
7.3 Product Profile: Zynerba Pharmaceuticals’ Zygel
7.4 Product Profile: Tetra Therapeutics’ Zatolmilast
7.5 Product Profile: Confluence Pharmaceuticals’ Acamprosate Calcium Sr
7.6 Product Profile: Neuren Pharmaceuticals’ and Acadia Pharmaceuticals’ Trofinetide
8 Market Outlook
8.1 Fragile X Syndrome Market Forecast
8.2 Market Drivers and Barriers
9 Appendix
9.1 Bibliography
9.2 Primary Research: Kol Information
9.3 About the Authors

Companies Mentioned

A selection of companies mentioned in this report includes:

  • Acadia Pharmaceuticals
  • Advanz Pharma Corp
  • Boehringer Ingelheim
  • Confluence Pharmaceuticals
  • Eli Lilly
  • GlaxoSmithKline
  • H. Lundbeck
  • Janssen
  • Neuren Pharmaceuticals
  • Novartis
  • Otsuka Pharmaceuticals
  • Sanofi
  • Takeda
  • Viatris
  • Tetra Therapeutics
  • Zynerba Pharmaceuticals