Fragile X Syndrome (FXS) Market to Register Immense Growth During the Forecast Period (2023-2032) - DelveInsight | Key Companies - Zynerba Pharmaceuticals, Prilenia, Alcobra, Marinus, Novartis, Roche, Healx, Nova Mentis, Lysogene, Actinogen, Prilenia

DelveInsight's "Fragile X Syndrome (FXS) Market Insights, Epidemiology, and Market Forecast 2032" report provides comprehensive insights into the disease landscape, encompassing historical and forecasted epidemiological data, along with an analysis of the Fragile X Syndrome market. The report offers valuable information regarding market size, share, emerging trends, and growth prospects across the seven major markets (7MM), including the United States, EU4 (Germany, Spain, Italy, France), the United Kingdom, and Japan.

Additionally, the report delves into emerging therapies for Fragile X Syndrome, providing insights into current treatment methodologies, individual therapy market shares, and a comprehensive evaluation of the market size, spanning from 2019 to 2032. It assesses the existing treatment practices and algorithms for Fragile X Syndrome, investigates the key factors influencing market growth, identifies barriers, and highlights unmet medical needs. This comprehensive analysis aims to pinpoint opportunities and gauge the market's inherent potential to address the critical aspects of Fragile X Syndrome management.

Learn How the Fragile X Syndrome Market is Evolving with the Ongoing Developments @ Fragile X Syndrome Market Outlook

Fragile X Syndrome (FXS): An Overview

Fragile X Syndrome (FXS) is a genetic disease due to a CGG trinucleotide expansion, named full mutation (greater than 200 CGG repeats), in the fragile X mental retardation 1 gene locus Xq27.3. It leads to a hypermethylated 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. Also, FXS is considered the most common inherited cause of intellectual disability and the second most prevalent cause after Down syndrome.

At birth, FXS neonates often exhibit no clinical signs of FXS with most parameters (e.g., head circumference, weight, and height) in the normal range. In early childhood FXS, physical and developmental features become more apparent, including developmental delays, psychomotor delays, intellectual disabilities, and a long face with prominent ears and flat feet. The behavioral phenotype of FXS includes significant anxiety, attention deficit hyperactivity disorder (ADHD), and hyperarousal to sensory stimuli.

Currently, there is no treatment for FXS, but some treatments help minimize the symptoms of the condition. The treatment regimen of FXS includes the use of stimulants, selective serotonin reuptake inhibitors (SSRIs), atypical antipsychotics, and alpha agonists.

The market is expected to grow due to factors like the expected entry of emerging therapies with novel targets and pricing. Furthermore, the impending products such as Zygel, Acamprosate, BPN14770, OV101, and Trofinetide are anticipated to expand the market with more penetration in the 7MM markets.

Fragile X Syndrome (FXS) Market Key Facts

• The total diagnosed prevalent population of Fragile X Syndrome in the 7MM was estimated to be around ~121,000 in 2021. Additionally, there was a diagnosed prevalent population of ~70,000 cases of FXS in the US.
• Among the gender-specific prevalent cases, in 2021, there were nearly ~41,000 cases of Fragile X Syndrome in males and nearly ~28,000 cases in females, in the US.
• The highest number of diagnosed prevalent cases of Fragile X Syndrome (FXS) were recorded in the United States.
• As per the analysis, Japan recorded ~13,000 cases of Fragile X Syndrome in 2021, which is expected to increase during the forecast period (2023-2032).
• Among the five European countries, Germany had the highest diagnosed prevalent population of Fragile X Syndrome (FXS), followed by France and the UK. On the other hand, Spain had the lowest diagnosed prevalent population of Fragile X Syndrome (FXS).

Get Exclusive Data and Key Insights Shaping the Market Dynamics @ Fragile X Syndrome Market Trends

Fragile X Syndrome (FXS) Therapeutics Analysis

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.

Selective serotonin reuptake inhibitors (SSRIs) are used to treat mood disorders, anxiety, and obsessive-compulsive behaviors associated with FXS. They are effective, particularly in alleviating social anxiety, tantrums, and aggression. Fluoxetine can be beneficial for selective mutism.

Other classes of antidepressants, such as Trazodone, Bupropion, Tricyclic antidepressants may be considered in instances in which SSRIs are not tolerated due to side effects, such as when there is behavioral activation that could not be managed by dose reduction. Clonidine and guanfacine have also been used to treat anxiety‐like symptoms, particularly the ones related to hypersensitivity to environmental sensory stimuli, but are probably not meaningfully effective for other forms of anxiety. Also, Buspirone may in some cases reduce anxiety, either alone or in combination with an SSRI.

To improve the treatment scenario, Several major pharma and biotech companies are developing therapies for Fragile X Syndrome. Companies like Zynerba Pharmaceuticals, Confluence Pharmaceuticals, Tetra Therapeutics, Ovid Therapeutics, and Neuren Pharmaceuticals are coming up with novel therapeutic approaches that can entirely change the treatment landscape of Fragile X Syndrome (FXS). Currently, Zynerba Pharmaceuticals is leading the therapeutics market with its Fragile X Syndrome drug candidates in the most advanced stage of clinical development.

Fragile X Syndrome (FXS) Therapeutic Advancements and Emerging Treatments:

• Fragile X Syndrome Clinical Trial Progression and Key Companies: The market is set to experience significant growth, driven by the progression of emerging therapies expected for launch between 2023 and 2032. Pioneering companies, including Zynerba Pharmaceuticals, Tetra Therapeutics, Autifony Therapeutics, Prilenia Therapeutics, Alcobra Ltd., Seaside Therapeutics, Inc., Marinus Pharmaceuticals, Novartis Pharmaceuticals, Neuren Pharmaceuticals, Hoffmann-La Roche, Healx, Nova Mentis, Lysogene, Actinogen, Prilenia Therapeutics, Confluence Pharmaceuticals, Sentinel Oncology, and many others, are actively engaged in developing novel drugs for potential market entry.
• Fragile X Syndrome Emerging and Marketed Therapies: Ongoing research and development activities are fostering the introduction of innovative therapies designed to address the signs and symptoms of Fragile X Syndrome. Some of the emerging therapies in the pipeline include Zatolmilast (Tetra Discovery Partners), Zygel (Zynerba Pharmaceuticals), Pridopidine (Prilenia Therapeutics), and many more.

Recent Developments in the Fragile X Syndrome (FXS) Market

• On September 27, 2023, Shionogi & Co., Ltd. (Head Office: Osaka, Japan; Chief Executive Officer: Isao Teshirogi, Ph.D.; hereafter "Shionogi") announced the U.S. Food and Drug Administration (FDA) granted Tetra Therapeutics Inc. (hereafter "Tetra"), a Shionogi Group Company, Rare Pediatric Disease Designation for zatolmilast (BPN14770), an investigational treatment being studied for Fragile X syndrome (FXS), a leading cause of inherited intellectual disability and autism.
• On Aug. 14, 2023, Harmony Biosciences Holdings, Inc. ("Harmony") (Nasdaq: HRMY) announced a definitive agreement to acquire Zynerba Pharmaceuticals, Inc. ("Zynerba") (Nasdaq: ZYNE), a leader in innovative pharmaceutically-produced transdermal cannabinoid therapies for orphan neuropsychiatric disorders, including Fragile X syndrome (FXS).
• On August 30, 2023, Nova Mentis Life Science Corp. (CSE: NOVA) (FSE: HN3Q) (OTCQB: NMLSF) ("NOVA" or the "Company"), a biotechnology company and global leader in first-in-class psilocybin-based therapeutics and complementary diagnostics for neuroinflammatory disorders is pleased to provide an update on its Phase IIA clinical trial testing psilocybin for the treatment of fragile X syndrome (FXS), the leading genetic cause of autism spectrum disorder (ASD).

Discover Significant Progress and Start Making Informed Decisions @ Clinical, Commercial, and Regulatory Activities in the Fragile X Syndrome Market

Fragile X Syndrome (FXS) Market Growth Factors and Associated Trends

The Fragile X Syndrome (FXS) market encompasses a range of drivers and barriers that intricately influence its evolution. Drivers fostering market growth include advances in genetic research, leading to a deeper understanding of the molecular mechanisms underlying FXS. These insights drive the development of targeted therapies aimed at mitigating symptoms and addressing the root cause of the syndrome, such as medications targeting specific genetic pathways or neurodevelopmental processes. Moreover, increased awareness and early diagnosis through improved screening methods contribute to earlier intervention and management, potentially ameliorating the impact of FXS on affected individuals and their families. Additionally, the growing emphasis on personalized medicine and precision therapies augments research efforts to tailor treatments to the specific genetic profiles and symptoms of individuals with FXS. 

However, barriers persist, notably the complexity of FXS due to its wide-ranging symptoms and variable severity, posing challenges in developing universally effective treatments. Limited availability and accessibility of specialized healthcare services for FXS, especially in underserved regions, hinder timely diagnosis and comprehensive care. Furthermore, the high cost of research and development for rare diseases like FXS, coupled with relatively small patient populations, can impede investment and slow down therapeutic innovation. Overcoming these barriers requires collaborative efforts among healthcare providers, researchers, pharmaceutical companies, and advocacy groups to enhance awareness, improve access to specialized care, and advance research for effective targeted therapies tailored to the diverse needs of individuals affected by FXS.

Fragile X Syndrome (FXS) Market

The report's market outlook section offers a comprehensive understanding of the historical, present, and projected market size. It scrutinizes the influence of existing and upcoming pipeline therapies for Fragile X Syndrome. Moreover, it conducts a thorough evaluation of the factors driving and hindering the Fragile X Syndrome market, highlighting unmet needs and emerging technologies poised to shape the market's dynamics in the foreseeable future.

The report meticulously outlines the market trends concerning each marketed Fragile X Syndrome drug and mid to late-stage pipeline therapies. This evaluation gauges their impact by considering various aspects such as the annual cost of therapy, Mechanism of Action (MOA), Route of Administration (ROA), molecule types, competitive landscape among therapies, brand value, and their collective influence on the market dynamics.

Discover the Anticipated Evolution and Growth of the Market @ Fragile X Syndrome Therapeutics Market Forecast

Fragile X Syndrome (FXS) Epidemiology Assessment

The epidemiology section offers valuable insights into the historical, present, and anticipated trends in Fragile X Syndrome epidemiology across the seven major countries (7MM) from 2019 to 2032. This section aims to identify the factors contributing to current and projected Fragile X Syndrome epidemiological patterns by examining diverse studies and research findings. Additionally, it furnishes a comprehensive analysis of diagnosed and prevalent patient populations, and future projections, and incorporates perspectives from key opinion leaders to enrich the understanding of this disease landscape.

The Report Covers the Fragile X Syndrome (FXS) Epidemiology, Segmented as -

• Gender-specific Cases of Fragile X Syndrome (FXS) [2019–2032]
• Age-specific Cases of Fragile X Syndrome (FXS) [2019–2032]
• Total Diagnosed Prevalent Cases of Fragile X Syndrome (FXS) [2019–2032]

Fragile X Syndrome (FXS) Drugs Uptake and Pipeline Development Activities

The drug uptake section concentrates on evaluating the adoption rate of newly launched or upcoming potential drugs within the Fragile X Syndrome market during the study period. This analysis encompasses the assessment of drug uptake in the Fragile X Syndrome market, patient adoption across different therapies, and the sales performance of each drug. Furthermore, the therapeutics assessment section facilitates a comprehensive comprehension of market dynamics by examining drug sales trends, identifying drugs with the highest adoption rates, and elucidating the reasons contributing to the widespread use of specific medications. Additionally, it conducts comparative analyses among Fragile X Syndrome drugs, considering their sales performance and respective market shares.

The report encompasses comprehensive coverage of ongoing Fragile X Syndrome pipeline development initiatives. It furnishes valuable insights into diverse therapeutic candidates at various developmental stages, elucidating the significant companies actively engaged in creating targeted treatments for Fragile X Syndrome. Moreover, it conducts an in-depth analysis of recent advancements, including collaborations, acquisitions, mergers, licensing details, and patent information, providing a holistic view of emerging therapies within the field.

Reports Key Features and Analysis

• Study Period - 2019-32
• Forecast Period - 2023-32
• Geography Coverage - 7MM (United States, EU4 [Germany, Spain, Italy, France], the United Kingdom, and Japan)
• Market Size (by 2032) and Expected CAGR - Download the Sample PDF
• Major Players Shaping the Market Outlook - Zynerba Pharmaceuticals, Tetra Therapeutics, Autifony Therapeutics, Prilenia Therapeutics, Alcobra Ltd., Seaside Therapeutics, Inc., Marinus Pharmaceuticals, Novartis Pharmaceuticals, Neuren Pharmaceuticals, Hoffmann-La Roche, Healx, Nova Mentis, Lysogene, Actinogen, Prilenia Therapeutics, Confluence Pharmaceuticals, Sentinel Oncology, and many others.
• Emerging and Marketed Therapies Analyzed in the Report - Zatolmilast (Tetra Discovery Partners), Zygel (Zynerba Pharmaceuticals), Pridopidine (Prilenia Therapeutics), and many more.

For In-depth Insights, key Report Offerings TOC, Access the Sample PDF File @ Fragile X Syndrome Therapeutics Market Outlook and Forecast

Related Reports:

• Fragile X Syndrome Epidemiology Forecast

Fragile X Syndrome Epidemiology Forecast report delivers an in-depth understanding of the Fragile X Syndrome, historical and forecasted epidemiology in the United States, EU5 (Germany, Spain, Italy, France, and the United Kingdom), and Japan.

• Fragile X Syndrome Pipeline Insight

Fragile X Syndrome Pipeline Insight report, provides comprehensive insights about major pharma and biotech companies and emerging drugs in the Fragile X Syndrome pipeline landscape. It covers the pipeline drug profiles, including clinical and nonclinical stage products. It also covers the therapeutics assessment by product type, stage, route of administration, and molecule type.

Healthcare Consulting and Market Research Services by DelveInsight

Unlock key insights and innovation in the healthcare industry with our cutting-edge market research services, paving the way for informed decisions and competitive advantage in this rapidly evolving industry @ Medical Device Consulting Services

Explore Our Success Story on Building an Interactive Competitive Intelligence Dashboard for HR+ HER2- Breast Cancer Market Landscape. Download our Oncology Competitive Intelligence Case Study!

About DelveInsight:

DelveInsight is a prominent business consultant and market research firm specializing in the life sciences sector. With a focus on supporting pharmaceutical companies, DelveInsight provides end-to-end solutions to enhance their performance.

Contact Information:

Shruti Thakur (Manager, Marketing and IB)

info@delveinsight.com

www.delveinsight.com

Connect with DelveInsight: LinkedIn | Facebook | Twitter