DUBLIN--(BUSINESS WIRE)--The "Fragile X Syndrome (FXS) - Market Insights, Epidemiology and Market Forecast - 2030" drug pipelines has been added to ResearchAndMarkets.com's offering.
This 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.
Epidemiology
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.
Key Findings
- The prevalent population of Fragile X Syndrome in 7MM is expected to increase at a CAGR of 0.62% for the study period, i.e. 2017-2030.
- Among the gender-specific prevalent cases, in 2017, there were 40,102 cases of Fragile X Syndrome (FXS) in males and 27,552 cases in females, in the US.
- The United States accounts for the highest diagnosed prevalent cases of Fragile X Syndrome with 67,654 cases in 2017.
Drug Chapters
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.
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.
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.
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.
OV101 (Gaboxadol): Ovid Therapeutics
OV101 also known as gaboxadol, is an oral, first-in-class, potent, -selective, extrasynaptic GABAA receptor agonist (SEGA). 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 -selective direct-acting GABAA receptor agonist that can bind to and activate this specific -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.
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-1, 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.
Market Outlook
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.
Key Findings
- According to the publisher estimates, the United States accounts for the largest market size of Fragile X Syndrome (FXS), in comparison to Japan and EU5 (the United Kingdom, Germany, Italy, France, and Spain).
- The market size of FXS in the seven major markets was USD 33.8 Million in 2017.
- Even with substantial support and therapies, individuals with FXS continue to present with significant impairments in their functioning throughout their life. There remains a great need for safe and effective treatments for FXS, particularly for targeted treatments that surpass symptom management and address the pathophysiologic abnormalities that underlie the most common manifestations.
Scope of the Report
- The report covers the descriptive overview of Fragile X Syndrome (FXS), explaining its causes, signs and symptoms, pathogenesis and currently available therapies.
- Comprehensive insight has been provided into the fragile X Syndrome (FXS) epidemiology and treatment.
- Additionally, an all-inclusive account of both the current and emerging therapies for Fragile X Syndrome (FXS) is provided, along with the assessment of new therapies, which will have an impact on the current treatment landscape.
- A detailed review of Fragile X Syndrome (FXS) market; historical and forecasted is included in the report, covering the 7MM drug outreach.
- The report provides an edge while developing business strategies, by understanding trends shaping and driving the 7MMFragile X Syndrome (FXS) market.
Report Highlights
- The market is expected to be driven positively due to funding and cooperation of various organizations, identification of potential targets by animal models, strengthening the molecular basis of FXS. However few factors such as failure of clinical trials, expensive diagnostic procedures might hamper the market.
- The 7MM market is anticipated to increase at a CAGR of 29.83% for the forecasted period (2020-2030).
- In the coming years, Fragile X Syndrome (FXS) market is set to change due to the rising awareness of the disease, and incremental healthcare spending across the world; which would expand the size of the market to enable the drug manufacturers to penetrate more into the market.
- The companies and academics are working to assess challenges and seek opportunities that could influence Fragile X Syndrome (FXS) R&D. The therapies under development are focused on novel approaches to treat/improve the disease condition.
- Major players are involved in developing therapies for Fragile X Syndrome (FXS). Launch of emerging therapies will significantly impact the Fragile X Syndrome (FXS) market. In the present scenario, several companies are actively working in this space while five companies are leading with the major products.
- The in-depth analysis of the pipeline assets across different stages of development (phase III and phase II), different emerging trends and comparative analysis of pipeline products with detailed clinical profiles, key competitors, launch date along with product development activities will support the clients in the decision-making process regarding their therapeutic portfolio by identifying the overall scenario of the research and development activities.
For more information about this drug pipelines report visit https://www.researchandmarkets.com/r/qnafoq
