Market Research Report
Fragile X Syndrome (FXS) - Market Insights, Epidemiology and Market Forecast - 2030
|Fragile X Syndrome (FXS) - Market Insights, Epidemiology and Market Forecast - 2030|
DelveInsight Business Research LLP
Content info: 167 Pages
Delivery time: 2-10 business days
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.
Study Period: 2017-2030
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.
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.
This section provides glimpses of the Fragile X Syndrome (FXS) epidemiology in the 7MM.
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.
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
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.
This section includes a glimpse of the Fragile X Syndrome (FXS) 7MM market.
This section provides the totalFragile X Syndrome (FXS) market size and market size by therapies in the United States.
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.
The total Fragile X Syndrome (FXS) market size and market size by therapies in Japan are provided.
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.
Current Treatment Scenario, Marketed Drugs and Emerging Therapies:
List of Companies