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

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The Fragile X syndrome (FXS) market size in the 2MM was valued at $21.9 million in 2020. The market is projected to grow at a CAGR of over 7% during the forecast period. Since there are currently no approved therapies available, prescribed treatments are exclusively off-label drugs that target individual symptoms of the disease. Despite behavioral and educational interventions, patients with FXS continue to present with significant impairments in their functioning throughout life. It is suggested that an FXS-specific treatment that targets the underlying brain deficits could treat cognition and language impairments. The introduction of pipeline agents is a major driver of growth within the FXS market.

Overview of the FXS market

Overview of the FXS market

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What are the market dynamics in the FXS market?

FXS is a lifelong disorder associated with a spectrum of developmental and behavioral issues, including psychiatric symptoms like anxiety, attention deficit hyperactivity disorder [ADHD], and poor eye contact. Neurologic symptoms like seizures and movement disorders also indicate FXS. 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.

The high failure rate associated with FXS drug development is due to the heterogeneity of the behavioral phenotypes and severity in patients. In 2020, in the 2MM, the sex-specific diagnosed prevalent cases of FXS were higher in men than women. Throughout the forecast period, it is observed that the symptom occurs more commonly in men than in women.

FXS is an active area of preclinical research and the identification of new potential drug targets using fmr1 K/O mice continues to strengthen the molecular basis of FXS. The orphan drug designation granted by the FDA and EMA provides companies and drug developers with additional benefits such as tax credits, reduced clinical testing fees, and additional patent exclusivity. During the 2020 base year, the atypical antipsychotics drug class contributed the highest sales to the FXS market.

What is the regional and country-level outlook of the FXS market?

In 2020, the US had the highest number of diagnosed prevalent cases of FXS, followed by Germany. The dominance of the US within the 2MM is due to its larger FXS population and higher drug prices.

What are the classifications of the FXS market?

An FXS patient’s set of symptoms can greatly change over time. They can range from mild anxiety or features of autism spectrum disorder (ASD) that affect communication and social interaction to more severe behavioral issues and cognitive deficits. However, there is not a system to grade the severity of behavioral symptoms such as hyperactivity and mood disorders including aggression and anxiety.

What are the current treatment options in the FXS market?

There are currently no approved therapies available for FXS. Prescribed treatment consists exclusively of off-label drugs that target individual symptoms of the disease. Some FXS patients require treatment with several different products to help alleviate their unique set of symptoms. The physician’s choice of treatment is heavily influenced by the tolerability of the drugs, side-effect profile, and the types and severities of symptoms. As such, first-line treatments vary widely among patients. Additionally, acute medications such as benzodiazepines and lithium may be used on an as-needed basis such as in distress crisis.

Patients with FXS benefit from educational and behavioral interventions alongside pharmacological therapy, and this can be tailored to the individual’s specific set of cognitive and learning deficits. As such, individualized education plans, speech-language therapy, occupational therapy, or behavioral therapy are often recommended to support the management of FXS symptoms. Early intervention is important as it aims to minimize symptoms and/or employ coping strategies to give patients the greatest chance of developing a full range of skills and the best opportunity to learn in a school environment. Many physicians avoid prescribing pharmacologic treatment for patients younger than five years old, as medication may have counterproductive effects on brain development during this time.

Who are the major players in the FXS market?

Some of the key players in the FXS market are Acadia Pharmaceuticals, Advanz Pharma Corp, Boehringer Ingelheim, Confluence Pharmaceuticals, Eli Lilly, GlaxoSmithKline, H. Lundbeck, Janssen, Neuren Pharmaceuticals, Novartis, Otsuka Pharmaceuticals, Sanofi, and Takeda.

FXS market, by major players

FXS market, by major players

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Market report scope

Market size (2020) $21.9 million
Growth rate CAGR of >7%
Forecast period 2020 to 2030
Key players Acadia Pharmaceuticals, Advanz Pharma Corp, Boehringer Ingelheim, Confluence Pharmaceuticals, Eli Lilly, GlaxoSmithKline, H. Lundbeck, Janssen, Neuren Pharmaceuticals, Novartis, Otsuka Pharmaceuticals, Sanofi, and Takeda


This report provides:

  • 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 including 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 a qualitative analysis of its implications.

Key Highlights

Report deliverables include a PowerPoint report and Excel-based forecast model

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.

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 counterstrategies 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.

Acadia Pharmaceuticals
Advanz Pharma Corp
Boehringer Ingelheim
Confluence Pharmaceuticals
Eli Lilly
H. Lundbeck
Neuren Pharmaceuticals
Otsuka Pharmaceuticals
Tetra Therapeutics
Zynerba Pharmaceuticals

Table of Contents

Table of Contents (PowerPoint Deck)

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

10 Contact Us

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