The Fragile X Syndrome market report also offers comprehensive insights into the Fragile X Syndrome market size, share, Fragile X Syndrome epidemiology, emerging therapies, market drivers and barriers, ongoing clinical trials, key collaboration in the space, market uptake by key therapies and companies actively pushing Fragile X Syndrome market size growth forward.
Some of the key highlights from the Fragile X Syndrome Market Insights Report:
Several key pharmaceutical companies, including Confluence Pharmaceuticals, Ovid Therapeutics, Neuren Pharmaceuticals, Zynerba Pharmaceuticals, Tetra Therapeutics, Autifony Therapeutics, Prilenia Therapeutics, Alcobra Ltd., Seaside Therapeutics, Inc., Marinus Pharmaceuticals, Novartis, Neuren Pharmaceuticals, Hoffmann-La Roche, Healx, Nova Mentis, Lysogene, Actinogen, Prilenia Therapeutics, Confluence Pharmaceuticals, Sentinel Oncology, and others, are developing novel products to improve the Fragile X Syndrome treatment outlook.
In March 2022, the European Commission granted orphan drug designation to Zygel, a cannabidiol (CBD) transdermal gel developed by Zynerba Pharmaceuticals, to treat behavioral symptoms associated with Fragile X syndrome.
In February 2022, Ovid entered into an agreement with Healx, granting them an exclusive option to license gaboxadol (OV101). Healx plans to explore this compound as a potential part of a combination therapy for Fragile X syndrome and other conditions.
In December 2021, Nova Mentis Life Science and Mycrodose Therapeutics announced the successful integration of NOVA’s psilocybin-based drug development program with Mycrodose’s transdermal technology to address Fragile X syndrome.
In September 2021, The McQuade Center for Strategic Research and Development, part of Otsuka’s global pharmaceutical network, entered into a collaboration with Sentinel Oncology to support clinical development of a first-in-class treatment for Fragile X syndrome.
In October 2021, Nova Mentis’ psilocybin drug received orphan drug designation from the European Medicines Agency for its potential use in treating Fragile X syndrome.
According to DelveInsight’s estimates, in 2023, approximately 122,000 prevalent cases of Fragile X syndrome were diagnosed across the 7MM. The United States accounted for 58% of these cases, while the EU4 and the UK represented 31%, and Japan accounted for 11%.
The market for Fragile X syndrome therapies is expected to experience steady growth, with an estimated compound annual growth rate (CAGR) of about 7% from 2024 to 2034. This growth is driven by the introduction of new treatments like Zygel and BPN14770, along with advancements in diagnostic technologies and ongoing research focused on genetic mechanisms of the disorder.
Despite progress in understanding Fragile X syndrome, there remains a notable gap in effective, approved treatments targeting the core symptoms. Timely and accurate diagnosis is still a challenge, limiting early intervention.
The market faces several obstacles, including high clinical trial failure rates due to the varied behavioral phenotypes among patients. Additionally, the lack of approved treatments, regulatory hurdles, and the need for extensive research hinder market growth.
The competitive landscape for Fragile X syndrome treatments is intensifying, with several companies vying for market share by leveraging orphan drug designations. Ongoing clinical trials and strategic partnerships are focused on bringing new treatment options to the forefront. Notable therapies under development include Zygel and BPN14770.
In July 2024, Zatolmilast received fast-track designation from the US FDA for the treatment of Fragile X syndrome, expediting its development and review process to increase patient access.
As per DelveInsight analysis, the Fragile X Syndrome market is anticipated to witness growth at a considerable CAGR
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Fragile X Syndrome Overview
Fragile X Syndrome is a genetic condition caused by a mutation in the FMR1 gene on the X chromosome, which leads to intellectual disabilities, developmental delays, and behavioral issues. This mutation causes a deficiency in the fragile X mental retardation protein (FMRP), which is crucial for proper brain development. The syndrome is marked by intellectual disabilities, typically mild to moderate in males and mild in females. Physical traits may include a long, narrow face, large ears, flexible fingers, and enlarged testicles in males. Behavioral symptoms often resemble those of autism spectrum disorder, along with hyperactivity and seizures.
The diagnosis of Fragile X syndrome is confirmed through genetic testing to measure the number of CGG repeats in the FMR1 gene. Normally, there are 5 to 40 repeats, but Fragile X syndrome occurs with more than 200 repeats. Molecular genetic tests are used to assess both the number of CGG repeats and the methylation status of the FMR1 gene. Prenatal testing methods, such as chorionic villus sampling and amniocentesis, can also be used to detect the syndrome in fetuses.
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Fragile X Syndrome Epidemiology Insights
In 2023, the United States saw about 42 thousand males and 29 thousand females diagnosed with Fragile X syndrome, showing a higher prevalence among males than females. In Japan, the distribution of cases in 2023 included approximately 5,700 cases in the 0–11 age group, 2,300 cases in the 12–17 age group, and 5,000 cases in those over 18, reflecting different age-related prevalence rates.
Fragile X Syndrome Epidemiology Segmentation
DelveInsight’s Fragile X Syndrome market report is prepared on the basis of epidemiology model. It offers comprehensive insights to the Fragile X Syndrome historical patient pools and forecasted Fragile X Syndrome patients. The report provides in-depth data of various subtypes and for the same epidemiology is segmented further. The Fragile X Syndrome Market report proffers epidemiological analysis for the study period 2020-32 in the 7MM segmented into:
Fragile X Syndrome Prevalence
Age-Specific Fragile X Syndrome Prevalence
Gender-Specific Fragile X Syndrome Prevalence
Diagnosed and Treatable Cases of Fragile X Syndrome
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Fragile X Syndrome Market Outlook
Treatment for Fragile X syndrome primarily focuses on managing its associated symptoms through pharmacological interventions. These may include stimulants for attention deficit and hyperactivity, selective serotonin reuptake inhibitors (SSRIs) for anxiety, antipsychotic medications for aggression and mood instability, and melatonin for sleep issues.
In boys with Fragile X syndrome, stimulants are commonly used to address hyperactivity, impulsivity, and distractibility, though their effectiveness and side effects can vary from person to person. In children under 5 years of age, stimulants may cause irritability and other behavioral issues, in which case non-stimulant medications may be considered.
SSRIs are often prescribed to manage mood disorders, anxiety, and obsessive-compulsive behaviors. These medications are particularly effective for reducing social anxiety, tantrums, and aggression. Fluoxetine can be particularly helpful for treating selective mutism.
If SSRIs are not tolerated due to side effects, other antidepressants such as Trazodone, Bupropion, and tricyclic antidepressants may be used. Clonidine and guanfacine are also sometimes prescribed for anxiety-like symptoms, especially those related to sensory sensitivities, although their effectiveness for other anxiety symptoms may be limited. Buspirone can reduce anxiety, either alone or alongside an SSRI.
Benzodiazepines are generally reserved for acute emotional crises with high anxiety but are not recommended for long-term use due to potential memory issues, increased confusion, and the risk of paradoxical effects like heightened hyperactivity and disinhibition.
Fragile X Syndrome Emerging Drugs
Zygel (ZYN002 Cannabidiol Gel): Harmony Biosciences
BPN14770: Tetra Therapeutics
Fragile X Syndrome Key Companies
Confluence Pharmaceuticals, Ovid Therapeutics, Neuren Pharmaceuticals, Zynerba Pharmaceuticals, Tetra Therapeutics, Autifony Therapeutics, Prilenia Therapeutics, Alcobra Ltd., Seaside Therapeutics, Inc., Marinus Pharmaceuticals, Novartis, Neuren Pharmaceuticals, Hoffmann-La Roche, Healx, Nova Mentis, Lysogene, Actinogen, Prilenia Therapeutics, Confluence Pharmaceuticals, Sentinel Oncology, and others
For more information, visit Fragile X Syndrome Market Analysis, Patient Pool, and Emerging Therapies
Scope of the Fragile X Syndrome Market Report:
11 Years Forecast
7MM Coverage
Descriptive overview of Fragile X Syndrome, causes, signs and symptoms, diagnosis, treatment
Comprehensive insight into Fragile X Syndrome epidemiology in the 7MM
Fragile X Syndrome marketed and emerging therapies
Fragile X Syndrome companies
Fragile X Syndrome market drivers and barriers
Table of Contents:
1 Fragile X Syndrome Market Key Comprehensive Insights
2 Fragile X Syndrome Market Report Introduction
3 Competitive Intelligence Analysis for Fragile X Syndrome
4 Fragile X Syndrome Market Analysis Overview at a Glance
5 Executive Summary of Fragile X Syndrome
6 Fragile X Syndrome Epidemiology and Market Methodology
7 Fragile X Syndrome Epidemiology and Patient Population
8 Fragile X Syndrome Patient Journey
9 Fragile X Syndrome Treatment Algorithm, Fragile X Syndrome Current Treatment, and Medical Practices
10 Key Endpoints in Fragile X Syndrome Clinical Trials
11 Fragile X Syndrome Marketed Therapies
12 Fragile X Syndrome Emerging Therapies
13 Fragile X Syndrome: 7 Major Market Analysis
14 Attribute analysis
15 Access and Reimbursement Overview of Fragile X Syndrome
16 Fragile X Syndrome Market Key Opinion Leaders Reviews
18 Fragile X Syndrome Market Drivers
19 Fragile X Syndrome Market Barriers
20 SWOT Analysis
21 Disclaimer
22 DelveInsight Capabilities
23 About DelveInsight
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